Transplantation for Severe Combined Immunodeficiency
My Research Interests are divided into 3 areas of focus: 1. Supportive Care (especially Invasive Fungal Infections) following Hematopoietic Stem Cell Transplantation 2. Transplantation for Severe Combined Immunodeficiency 3. Transplantation for Rare Leukemias (JMML and APL)
Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, Cowan MJ, Dvorak CC, Craddock JA, Prockop SE, Chandrakasan S, Kapoor N, Buckley RH, Parikh S, Chellapandian D, Oshrine BR, Bednarski JJ, Cooper MA, Shenoy S, Davila Saldana BJ, Forbes LR, Martinez C, Haddad E, Shyr DC, Chen K, Sullivan KE, Heimall J, Wright N, Bhatia M, Cuvelier GDE, Goldman FD, Meyts I, Miller HK, Seidel MG, Vander Lugt MT, Bacchetta R, Weinacht KG, Andolina JR, Caywood E, Chong H, de la Morena MT, Aquino VM, Shereck E, Walter JE, Dorsey MJ, Seroogy CM, Griffith LM, Kohn DB, Puck JM, Pulsipher MA, Torgerson TR
Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Front Immunol. 2020; 11:239.
Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.
Papanicolaou GA, Dvorak CC, Dadwal S, Maron G, Prasad VK, Giller R, Abdel-Azim H, Sadanand A, Casciano R, Chandak A, Huang S, Nichols G, Brundage T, Vainorius E, Mozaffari E, Hutcheson R
Transplant infectious disease : an official journal of the Transplantation Society
Practice patterns and incidence of adenovirus infection in allogeneic hematopoietic cell transplant recipients: Multicenter survey of transplant centers in the United States. Transpl Infect Dis. 2020 Aug; 22(4):e13283.
Practice patterns and incidence of adenovirus infection in allogeneic hematopoietic cell transplant recipients: Multicenter survey of transplant centers in the United States.
Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Dávila Saldaña BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 06 04; 135(23):2094-2105.
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.
Contreras CF, Long-Boyle JR, Shimano KA, Melton A, Kharbanda S, Dara J, Higham C, Huang JN, Cowan MJ, Dvorak CC
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Reduced Toxicity Conditioning for Nonmalignant Hematopoietic Cell Transplants. Biol Blood Marrow Transplant. 2020 09; 26(9):1646-1654.
Reduced Toxicity Conditioning for Nonmalignant Hematopoietic Cell Transplants.
Primary Immune Deficiency Treatment Consortium
Dvorak CC, Gracia CR, Sanders JE, Cheng EY, Baker KS, Pulsipher MA, Petryk A
NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: endocrine challenges-thyroid dysfunction, growth impairment, bone health, & reproductive risks. Biol Blood Marrow Transplant. 2011 Dec; 17(12):1725-38.
NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: endocrine challenges-thyroid dysfunction, growth impairment, bone health, & reproductive risks.
Dvorak CC, Fisher BT, Sung L, Steinbach WJ, Nieder M, Alexander S, Zaoutis TE
Antifungal prophylaxis in pediatric hematology/oncology: new choices & new data. Pediatr Blood Cancer. 2012 Jul 15; 59(1):21-6.
Antifungal prophylaxis in pediatric hematology/oncology: new choices & new data.
Alexander S, Nieder M, Zerr DM, Fisher BT, Dvorak CC, Sung L
Prevention of bacterial infection in pediatric oncology: what do we know, what can we learn? Pediatr Blood Cancer. 2012 Jul 15; 59(1):16-20.
Prevention of bacterial infection in pediatric oncology: what do we know, what can we learn?
Fisher BT, Alexander S, Dvorak CC, Zaoutis TE, Zerr DM, Sung L
Epidemiology and potential preventative measures for viral infections in children with malignancy and those undergoing hematopoietic cell transplantation. Pediatr Blood Cancer. 2012 Jul 15; 59(1):11-5.
Epidemiology and potential preventative measures for viral infections in children with malignancy and those undergoing hematopoietic cell transplantation.
Dvorak CC, Bollard CM, El-Bietar J, Filipovich A
Complications of transplant for nonmalignant disorders: autoimmune cytopenias, opportunistic infections, and PTLD. Biol Blood Marrow Transplant. 2012 Jan; 18(1 Suppl):S101-10.
Complications of transplant for nonmalignant disorders: autoimmune cytopenias, opportunistic infections, and PTLD.
Pulsipher MA, Skinner R, McDonald GB, Hingorani S, Armenian SH, Cooke KR, Gracia C, Petryk A, Bhatia S, Bunin N, Nieder ML, Dvorak CC, Sung L, Sanders JE, Kurtzberg J, Baker KS
National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on late effects after pediatric hematopoietic cell transplantation: the need for pediatric-specific long-term follow-up guidelines. Biol Blood Marrow Transplant. 2012 Mar; 18(3):334-47.
National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on late effects after pediatric hematopoietic cell transplantation: the need for pediatric-specific long-term follow-up guidelines.
Mehling WE, Lown EA, Dvorak CC, Cowan MJ, Horn BN, Dunn EA, Acree M, Abrams DI, Hecht FM
Evidence-based complementary and alternative medicine : eCAM
Hematopoietic cell transplant and use of massage for improved symptom management: results from a pilot randomized control trial. Evid Based Complement Alternat Med. 2012; 2012:450150.
Hematopoietic cell transplant and use of massage for improved symptom management: results from a pilot randomized control trial.
Ackerman SL, Lown EA, Dvorak CC, Dunn EA, Abrams DI, Horn BN, Degelman M, Cowan MJ, Mehling WE
Massage for children undergoing hematopoietic cell transplantation: a qualitative report. Evid Based Complement Alternat Med. 2012; 2012:792042.
Massage for children undergoing hematopoietic cell transplantation: a qualitative report.
Veys P, Wynn RF, Ahn KW, Samarasinghe S, He W, Bonney D, Craddock J, Cornish J, Davies SM, Dvorak CC, Duerst RE, Gross TG, Kapoor N, Kitko C, Krance RA, Leung W, Lewis VA, Steward C, Wagner JE, Carpenter PA, Eapen M
Impact of immune modulation with in vivo T-cell depletion and myleoablative total body irradiation conditioning on outcomes after unrelated donor transplantation for childhood acute lymphoblastic leukemia. Blood. 2012 Jun 21; 119(25):6155-61.
Impact of immune modulation with in vivo T-cell depletion and myleoablative total body irradiation conditioning on outcomes after unrelated donor transplantation for childhood acute lymphoblastic leukemia.
Law J, Cowan MJ, Dvorak CC, Musick L, Long-Boyle JR, Baxter-Lowe LA, Horn B
Busulfan, fludarabine, and alemtuzumab as a reduced toxicity regimen for children with malignant and nonmalignant diseases improves engraftment and graft-versus-host disease without delaying immune reconstitution. Biol Blood Marrow Transplant. 2012 Nov; 18(11):1656-63.
Busulfan, fludarabine, and alemtuzumab as a reduced toxicity regimen for children with malignant and nonmalignant diseases improves engraftment and graft-versus-host disease without delaying immune reconstitution.
Steinbach WJ, Roilides E, Berman D, Hoffman JA, Groll AH, Bin-Hussain I, Palazzi DL, Castagnola E, Halasa N, Velegraki A, Dvorak CC, Charkabarti A, Sung L, Danziger-Isakov L, Lachenauer C, Arrieta A, Knapp K, Abzug MJ, Ziebold C, Lehrnbecher T, Klingspor L, Warris A, Leckerman K, Martling T, Walsh TJ, Benjamin DK, Zaoutis TE, International Pediatric Fungal Network
The Pediatric infectious disease journal
Results from a prospective, international, epidemiologic study of invasive candidiasis in children and neonates. Pediatr Infect Dis J. 2012 Dec; 31(12):1252-7.
Results from a prospective, international, epidemiologic study of invasive candidiasis in children and neonates.
Engelhardt KR, Shah N, Faizura-Yeop I, Kocacik Uygun DF, Frede N, Muise AM, Shteyer E, Filiz S, Chee R, Elawad M, Hartmann B, Arkwright PD, Dvorak C, Klein C, Puck JM, Grimbacher B, Glocker EO
The Journal of allergy and clinical immunology
Clinical outcome in IL-10- and IL-10 receptor-deficient patients with or without hematopoietic stem cell transplantation. J Allergy Clin Immunol. 2013 Mar; 131(3):825-30.
Clinical outcome in IL-10- and IL-10 receptor-deficient patients with or without hematopoietic stem cell transplantation.
Dvorak CC, Gilman AL, Horn B, Oon CY, Dunn EA, Baxter-Lowe LA, Cowan MJ
Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34(+) cells and a fixed CD3(+) dose. Bone Marrow Transplant. 2013 Apr; 48(4):508-13.
Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34(+) cells and a fixed CD3(+) dose.
Grupp SA, Dvorak CC, Nieder ML, Levine JE, Wall DA, Langholz B, Pulsipher MA, COG Stem Cell Transplant Scientific Committee
Children's Oncology Group's 2013 blueprint for research: stem cell transplantation. Pediatr Blood Cancer. 2013 06; 60(6):1044-7.
Children's Oncology Group's 2013 blueprint for research: stem cell transplantation.
Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH
The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct; 33(7):1156-64.
The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901.
Savic RM, Cowan MJ, Dvorak CC, Pai SY, Pereira L, Bartelink IH, Boelens JJ, Bredius RG, Wynn RF, Cuvelier GD, Shaw PJ, Slatter MA, Long-Boyle J
Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2013 Nov; 19(11):1608-14.
Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation.
Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC, workshop participants
Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb; 133(2):335-47.
Primary Immune Deficiency Treatment Consortium (PIDTC) report.
Schuetz C, Neven B, Dvorak CC, Leroy S, Ege MJ, Pannicke U, Schwarz K, Schulz AS, Hoenig M, Sparber-Sauer M, Gatz SA, Denzer C, Blanche S, Moshous D, Picard C, Horn BN, de Villartay JP, Cavazzana M, Debatin KM, Friedrich W, Fischer A, Cowan MJ
SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. Blood. 2014 Jan 09; 123(2):281-9.
SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID.
Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ
Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr; 133(4):1092-8.
Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience.
Aplenc R, Zhang MJ, Sung L, Zhu X, Ho VT, Cooke K, Dvorak C, Hale G, Isola LM, Lazarus HM, McCarthy PL, Olsson R, Pulsipher M, Pasquini MC, Bunin N, Regimen-Related Toxicity Working Committee, Center for International Blood and Marrow Transplant Res
Effect of body mass in children with hematologic malignancies undergoing allogeneic bone marrow transplantation. Blood. 2014 May 29; 123(22):3504-11.
Effect of body mass in children with hematologic malignancies undergoing allogeneic bone marrow transplantation.
Karski EE, Dvorak CC, Leung W, Miller W, Shaw PJ, Qayed M, Katsanis E, Feusner JH
Journal of pediatric hematology/oncology
Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue: the pediatric blood and marrow transplant consortium experience and review of the literature. J Pediatr Hematol Oncol. 2014 Jul; 36(5):362-8.
Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue: the pediatric blood and marrow transplant consortium experience and review of the literature.
Juvenile myelomonocytic leukemia: molecular pathogenesis informs current approaches to therapy and hematopoietic cell transplantation. Front Pediatr. 2014; 2:25.
Juvenile myelomonocytic leukemia: molecular pathogenesis informs current approaches to therapy and hematopoietic cell transplantation.
Huang L, Lizak P, Dvorak CC, Aweeka F, Long-Boyle J
Journal of chromatography. B, Analytical technologies in the biomedical and life sciences
Simultaneous determination of fludarabine and clofarabine in human plasma by LC-MS/MS. J Chromatogr B Analyt Technol Biomed Life Sci. 2014 Jun 01; 960:194-9.
J Chromatogr B Analyt Technol Biomed Life Sci
Simultaneous determination of fludarabine and clofarabine in human plasma by LC-MS/MS.
Dvorak CC, Horn BN, Puck JM, Czechowicz A, Shizuru JA, Ko RM, Cowan MJ
A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant. 2014 Sep; 18(6):602-8.
A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.
Dvorak CC, Horn BN, Puck JM, Adams S, Veys P, Czechowicz A, Cowan MJ
A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant. 2014 Sep; 18(6):609-16.
A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.
Lund TC, Cathey SS, Miller WP, Eapen M, Andreansky M, Dvorak CC, Davis JH, Dalal JD, Devine SM, Eames GM, Ferguson WS, Giller RH, He W, Kurtzberg J, Krance R, Katsanis E, Lewis VA, Sahdev I, Orchard PJ
Outcomes after hematopoietic stem cell transplantation for children with I-cell disease. Biol Blood Marrow Transplant. 2014 Nov; 20(11):1847-51.
Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.
Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ
Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31; 371(5):434-46.
Transplantation outcomes for severe combined immunodeficiency, 2000-2009.
Long-Boyle JR, Savic R, Yan S, Bartelink I, Musick L, French D, Law J, Horn B, Cowan MJ, Dvorak CC
Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use. Ther Drug Monit. 2015 Apr; 37(2):236-45.
Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use.
Bedside to bench in juvenile myelomonocytic leukemia: insights into leukemogenesis from a rare pediatric leukemia. Blood. 2014 Oct 16; 124(16):2487-97.
Bedside to bench in juvenile myelomonocytic leukemia: insights into leukemogenesis from a rare pediatric leukemia.
Yanik GA, Grupp SA, Pulsipher MA, Levine JE, Schultz KR, Wall DA, Langholz B, Dvorak CC, Alangaden K, Goyal RK, White ES, Collura JM, Skeens MA, Eid S, Pierce EM, Cooke KR
TNF-receptor inhibitor therapy for the treatment of children with idiopathic pneumonia syndrome. A joint Pediatric Blood and Marrow Transplant Consortium and Children's Oncology Group Study (ASCT0521). Biol Blood Marrow Transplant. 2015 Jan; 21(1):67-73.
TNF-receptor inhibitor therapy for the treatment of children with idiopathic pneumonia syndrome. A joint Pediatric Blood and Marrow Transplant Consortium and Children's Oncology Group Study (ASCT0521).
Dvorak CC, Hassan A, Slatter MA, Hönig M, Lankester AC, Buckley RH, Pulsipher MA, Davis JH, Güngör T, Gabriel M, Bleesing JH, Bunin N, Sedlacek P, Connelly JA, Crawford DF, Notarangelo LD, Pai SY, Hassid J, Veys P, Gennery AR, Cowan MJ
Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Oct; 134(4):935-943.e15.
Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency.
Flank J, Sung L, Dvorak CC, Spettigue W, Dupuis LL
The safety of olanzapine in young children: a systematic review and meta-analysis. Drug Saf. 2014 Oct; 37(10):791-804.
The safety of olanzapine in young children: a systematic review and meta-analysis.
Juvenile myelomonocytic leukemia. Pediatr Clin North Am. 2015 Feb; 62(1):95-106.
Niemeyer CM, Loh ML, Cseh A, Cooper T, Dvorak CC, Chan R, Xicoy B, Germing U, Kojima S, Manabe A, Dworzak M, De Moerloose B, Starý J, Smith OP, Masetti R, Catala A, Bergstraesser E, Ussowicz M, Fabri O, Baruchel A, Cavé H, Zwaan M, Locatelli F, Hasle H, van den Heuvel-Eibrink MM, Flotho C, Yoshimi A
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. Haematologica. 2015 Jan; 100(1):17-22.
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.
Horn B, Petrovic A, Wahlstrom J, Dvorak CC, Kong D, Hwang J, Expose-Spencer J, Gates M, Cowan MJ
Chimerism-based pre-emptive immunotherapy with fast withdrawal of immunosuppression and donor lymphocyte infusions after allogeneic stem cell transplantation for pediatric hematologic malignancies. Biol Blood Marrow Transplant. 2015 Apr; 21(4):729-37.
Chimerism-based pre-emptive immunotherapy with fast withdrawal of immunosuppression and donor lymphocyte infusions after allogeneic stem cell transplantation for pediatric hematologic malignancies.
Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OT, Pasquini MC, Schriber JR, Cooke KR
Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies. Leukemia. 2015 Aug; 29(8):1754-62.
Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies.
Holter-Chakrabarty JL, Pierson N, Zhang MJ, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL
The Sequence of Cyclophosphamide and Myeloablative Total Body Irradiation in Hematopoietic Cell Transplantation for Patients with Acute Leukemia. Biol Blood Marrow Transplant. 2015 Jul; 21(7):1251-7.
The Sequence of Cyclophosphamide and Myeloablative Total Body Irradiation in Hematopoietic Cell Transplantation for Patients with Acute Leukemia.
Wattier RL, Dvorak CC, Auerbach AD, Weintrub PS
Repeat blood cultures in children with persistent fever and neutropenia: Diagnostic and clinical implications. Pediatr Blood Cancer. 2015 Aug; 62(8):1421-6.
Repeat blood cultures in children with persistent fever and neutropenia: Diagnostic and clinical implications.
Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency. Curr Pediatr Rep. 2015 Mar 01; 3(1):1-10.
Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency.
Mehta PA, Zhang MJ, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel-Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM
Transplantation Outcomes for Children with Hypodiploid Acute Lymphoblastic Leukemia. Biol Blood Marrow Transplant. 2015 Jul; 21(7):1273-7.
Transplantation Outcomes for Children with Hypodiploid Acute Lymphoblastic Leukemia.
Bode SF, Ammann S, Al-Herz W, Bataneant M, Dvorak CC, Gehring S, Gennery A, Gilmour KC, Gonzalez-Granado LI, Groß-Wieltsch U, Ifversen M, Lingman-Framme J, Matthes-Martin S, Mesters R, Meyts I, van Montfrans JM, Pachlopnik Schmid J, Pai SY, Soler-Palacin P, Schuermann U, Schuster V, Seidel MG, Speckmann C, Stepensky P, Sykora KW, Tesi B, Vraetz T, Waruiru C, Bryceson YT, Moshous D, Lehmberg K, Jordan MB, Ehl S, Inborn Errors Working Party of the EBMT
The syndrome of hemophagocytic lymphohistiocytosis in primary immunodeficiencies: implications for differential diagnosis and pathogenesis. Haematologica. 2015 Jul; 100(7):978-88.
The syndrome of hemophagocytic lymphohistiocytosis in primary immunodeficiencies: implications for differential diagnosis and pathogenesis.
Zinter MS, Dvorak CC, Spicer A, Cowan MJ, Sapru A
New Insights Into Multicenter PICU Mortality Among Pediatric Hematopoietic Stem Cell Transplant Patients. Crit Care Med. 2015 Sep; 43(9):1986-94.
New Insights Into Multicenter PICU Mortality Among Pediatric Hematopoietic Stem Cell Transplant Patients.
Levy ER, Musick L, Zinter MS, Lang T, Cowan MJ, Weintrub PS, Dvorak CC
Safe and Effective Prophylaxis with Bimonthly Intravenous Pentamidine in the Pediatric Hematopoietic Stem Cell Transplant Population. Pediatr Infect Dis J. 2016 Feb; 35(2):135-41.
Safe and Effective Prophylaxis with Bimonthly Intravenous Pentamidine in the Pediatric Hematopoietic Stem Cell Transplant Population.
Burke MJ, Verneris MR, Le Rademacher J, He W, Abdel-Azim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL
Transplant Outcomes for Children with T Cell Acute Lymphoblastic Leukemia in Second Remission: A Report from the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant. 2015 Dec; 21(12):2154-2159.
Transplant Outcomes for Children with T Cell Acute Lymphoblastic Leukemia in Second Remission: A Report from the Center for International Blood and Marrow Transplant Research.
Wattier RL, Dvorak CC, Hoffman JA, Brozovich AA, Bin-Hussain I, Groll AH, Castagnola E, Knapp KM, Zaoutis TE, Gustafsson B, Sung L, Berman D, Halasa NB, Abzug MJ, Velegraki A, Sharma TS, Fisher BT, Steinbach WJ
Journal of the Pediatric Infectious Diseases Society
A Prospective, International Cohort Study of Invasive Mold Infections in Children. J Pediatric Infect Dis Soc. 2015 Dec; 4(4):313-22.
A Prospective, International Cohort Study of Invasive Mold Infections in Children.
Zinter MS, Spicer A, Orwoll BO, Alkhouli M, Dvorak CC, Calfee CS, Matthay MA, Sapru A
American journal of physiology. Lung cellular and molecular physiology
Plasma angiopoietin-2 outperforms other markers of endothelial injury in prognosticating pediatric ARDS mortality. Am J Physiol Lung Cell Mol Physiol. 2016 Feb 01; 310(3):L224-31.
Plasma angiopoietin-2 outperforms other markers of endothelial injury in prognosticating pediatric ARDS mortality.
Williams KM, Ahn KW, Chen M, Aljurf MD, Agwu AL, Chen AR, Walsh TJ, Szabolcs P, Boeckh MJ, Auletta JJ, Lindemans CA, Zanis-Neto J, Malvezzi M, Lister J, de Toledo Codina JS, Sackey K, Chakrabarty JL, Ljungman P, Wingard JR, Seftel MD, Seo S, Hale GA, Wirk B, Smith MS, Savani BN, Lazarus HM, Marks DI, Ustun C, Abdel-Azim H, Dvorak CC, Szer J, Storek J, Yong A, Riches MR
The incidence, mortality and timing of Pneumocystis jiroveci pneumonia after hematopoietic cell transplantation: a CIBMTR analysis. Bone Marrow Transplant. 2016 Apr; 51(4):573-80.
The incidence, mortality and timing of Pneumocystis jiroveci pneumonia after hematopoietic cell transplantation: a CIBMTR analysis.
Alexander S, Pole JD, Gibson P, Lee M, Hesser T, Chi SN, Dvorak CC, Fisher B, Hasle H, Kanerva J, Möricke A, Phillips B, Raetz E, Rodriguez-Galindo C, Samarasinghe S, Schmiegelow K, Tissing W, Lehrnbecher T, Sung L, International Pediatric Oncology Mortality Classification Group
Classification of treatment-related mortality in children with cancer: a systematic assessment. Lancet Oncol. 2015 Dec; 16(16):e604-10.
Classification of treatment-related mortality in children with cancer: a systematic assessment.
Bailey-Olson M, Cowan M, Dvorak C, Mueller S, Owens A, Wahlstrom J, Horn B
Journal of pediatric oncology nursing : official journal of the Association of Pediatric Oncology Nurses
Evaluation of Pre-Hematopoietic Cell Transplantation (HCT) Brain MRI and Neurologic Complications of Pediatric Patients Undergoing HCT for Hematologic Malignancies. J Pediatr Oncol Nurs. 2017 Jan/Feb; 34(1):65-73.
Evaluation of Pre-Hematopoietic Cell Transplantation (HCT) Brain MRI and Neurologic Complications of Pediatric Patients Undergoing HCT for Hematologic Malignancies.
Horn B, O'Kane S, Wattier RL, Wahlstrom JT, Melton A, Cowan MJ, Dvorak CC
Risk of serious bloodstream infections is low in pediatric hematopoietic stem cell transplant (HSCT) recipients with fevers due to antithymocyte globulins and alemtuzumab. Bone Marrow Transplant. 2016 11; 51(11):1510-1512.
Risk of serious bloodstream infections is low in pediatric hematopoietic stem cell transplant (HSCT) recipients with fevers due to antithymocyte globulins and alemtuzumab.
Geerlinks AV, Issekutz T, Wahlstrom JT, Sullivan KE, Cowan MJ, Dvorak CC, Fernandez CV
Severe, persistent, and fatal T-cell immunodeficiency following therapy for infantile leukemia. Pediatr Blood Cancer. 2016 11; 63(11):2046-9.
Severe, persistent, and fatal T-cell immunodeficiency following therapy for infantile leukemia.
Wahlstrom J, Patel K, Eckhert E, Kong D, Horn B, Cowan MJ, Dvorak CC
Transplacental maternal engraftment and posttransplantation graft-versus-host disease in children with severe combined immunodeficiency. J Allergy Clin Immunol. 2017 02; 139(2):628-633.e10.
Transplacental maternal engraftment and posttransplantation graft-versus-host disease in children with severe combined immunodeficiency.
de la Morena MT, Leonard D, Torgerson TR, Cabral-Marques O, Slatter M, Aghamohammadi A, Chandra S, Murguia-Favela L, Bonilla FA, Kanariou M, Damrongwatanasuk R, Kuo CY, Dvorak CC, Meyts I, Chen K, Kobrynski L, Kapoor N, Richter D, DiGiovanni D, Dhalla F, Farmaki E, Speckmann C, Español T, Shcherbina A, Hanson IC, Litzman J, Routes JM, Wong M, Fuleihan R, Seneviratne SL, Small TN, Janda A, Bezrodnik L, Seger R, Raccio AG, Edgar JD, Chou J, Abbott JK, van Montfrans J, González-Granado LI, Bunin N, Kutukculer N, Gray P, Seminario G, Pasic S, Aquino V, Wysocki C, Abolhassani H, Dorsey M, Cunningham-Rundles C, Knutsen AP, Sleasman J, Costa Carvalho BT, Condino-Neto A, Grunebaum E, Chapel H, Ochs HD, Filipovich A, Cowan M, Gennery A, Cant A, Notarangelo LD, Roifman CM
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation. J Allergy Clin Immunol. 2017 Apr; 139(4):1282-1292.
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation.
Bartelink IH, Lalmohamed A, van Reij EM, Dvorak CC, Savic RM, Zwaveling J, Bredius RG, Egberts AC, Bierings M, Kletzel M, Shaw PJ, Nath CE, Hempel G, Ansari M, Krajinovic M, Théorêt Y, Duval M, Keizer RJ, Bittencourt H, Hassan M, Güngör T, Wynn RF, Veys P, Cuvelier GD, Marktel S, Chiesa R, Cowan MJ, Slatter MA, Stricherz MK, Jennissen C, Long-Boyle JR, Boelens JJ
Association of busulfan exposure with survival and toxicity after haemopoietic cell transplantation in children and young adults: a multicentre, retrospective cohort analysis. Lancet Haematol. 2016 Nov; 3(11):e526-e536.
Association of busulfan exposure with survival and toxicity after haemopoietic cell transplantation in children and young adults: a multicentre, retrospective cohort analysis.
Nademi Z, Slatter MA, Dvorak CC, Neven B, Fischer A, Suarez F, Booth C, Rao K, Laberko A, Rodina J, Bertrand Y, Koltan S, Debski R, Flood T, Abinun M, Gennery AR, Hambleton S, Ehl S, Cant AJ
Hematopoietic stem cell transplant in patients with activated PI3K delta syndrome. J Allergy Clin Immunol. 2017 03; 139(3):1046-1049.
Hematopoietic stem cell transplant in patients with activated PI3K delta syndrome.
Musical chairs: in utero HCT via mobilization. Blood. 2016 11 17; 128(20):2378-2380.
Musical chairs: in utero HCT via mobilization.
Maziarz RT, Brazauskas R, Chen M, McLeod AA, Martino R, Wingard JR, Aljurf M, Battiwalla M, Dvorak CC, Geroge B, Guinan EC, Hale GA, Lazarus HM, Lee JW, Liesveld JL, Ramanathan M, Reddy V, Savani BN, Smith FO, Strasfeld L, Taplitz RA, Ustun C, Boeckh MJ, Gea-Banacloche J, Lindemans CA, Auletta JJ, Riches ML
Pre-existing invasive fungal infection is not a contraindication for allogeneic HSCT for patients with hematologic malignancies: a CIBMTR study. Bone Marrow Transplant. 2017 Feb; 52(2):270-278.
Pre-existing invasive fungal infection is not a contraindication for allogeneic HSCT for patients with hematologic malignancies: a CIBMTR study.
Satwani P, Freedman JL, Chaudhury S, Jin Z, Levinson A, Foca MD, Krajewski J, Sahdev I, Talekar MK, Gardenswartz A, Silverman J, Hayes M, Dvorak CC
A Multicenter Study of Bacterial Blood Stream Infections in Pediatric Allogeneic Hematopoietic Cell Transplantation Recipients: The Role of Acute Gastrointestinal Graft-versus-Host Disease. Biol Blood Marrow Transplant. 2017 Apr; 23(4):642-647.
A Multicenter Study of Bacterial Blood Stream Infections in Pediatric Allogeneic Hematopoietic Cell Transplantation Recipients: The Role of Acute Gastrointestinal Graft-versus-Host Disease.
Horn B, Wahlstrom JT, Melton A, Liou A, Ouachee-Chardin M, Sunkersett G, Willert J, Hwang J, Expose-Spencer J, Cowan MC, Facchino J, Dvorak CC
Early mixed chimerism-based preemptive immunotherapy in children undergoing allogeneic hematopoietic stem cell transplantation for acute leukemia. Pediatr Blood Cancer. 2017 Aug; 64(8).
Early mixed chimerism-based preemptive immunotherapy in children undergoing allogeneic hematopoietic stem cell transplantation for acute leukemia.
Treatment of infants identified as having severe combined immunodeficiency by means of newborn screening. J Allergy Clin Immunol. 2017 Mar; 139(3):733-742.
Treatment of infants identified as having severe combined immunodeficiency by means of newborn screening.
Theiler M, Oza VS, Mathes EF, Dvorak CC, McCalmont TH, Yeh I, Sidbury R, Cordoro KM
Eosinophilic Pustular Folliculitis in Children after Stem Cell Transplantation: An Eruption Distinct from Graft-Versus-Host Disease. Pediatr Dermatol. 2017 May; 34(3):326-330.
Eosinophilic Pustular Folliculitis in Children after Stem Cell Transplantation: An Eruption Distinct from Graft-Versus-Host Disease.
Liou A, Wahlstrom JT, Dvorak CC, Horn BN
Safety of pre-emptive donor lymphocyte infusions (DLI) based on mixed chimerism (MC) in peripheral blood or bone marrow subsets in children undergoing hematopoietic stem cell transplant (HSCT) for hematologic malignancies. Bone Marrow Transplant. 2017 07; 52(7):1057-1059.
Safety of pre-emptive donor lymphocyte infusions (DLI) based on mixed chimerism (MC) in peripheral blood or bone marrow subsets in children undergoing hematopoietic stem cell transplant (HSCT) for hematologic malignancies.
Dvorak CC, Patel K, Puck JM, Wahlstrom J, Dorsey MJ, Adams R, Facchino J, Cowan MJ
Unconditioned unrelated donor bone marrow transplantation for IL7Rα- and Artemis-deficient SCID. Bone Marrow Transplant. 2017 07; 52(7):1036-1038.
Unconditioned unrelated donor bone marrow transplantation for IL7Rα- and Artemis-deficient SCID.
Cao Z, Villa KF, Lipkin CB, Robinson SB, Nejadnik B, Dvorak CC
Burden of illness associated with sinusoidal obstruction syndrome/veno-occlusive disease in patients with hematopoietic stem cell transplantation. J Med Econ. 2017 Aug; 20(8):871-883.
Burden of illness associated with sinusoidal obstruction syndrome/veno-occlusive disease in patients with hematopoietic stem cell transplantation.
Zinter MS, Barrows BD, Ursell PC, Kowalek K, Kalantar K, Cambronero N, DeRisi JL, Oishi P, Dvorak CC
Extracorporeal life support survival in a pediatric hematopoietic cellular transplant recipient with presumed GvHD-related fulminant myocarditis. Bone Marrow Transplant. 2017 09; 52(9):1330-1333.
Extracorporeal life support survival in a pediatric hematopoietic cellular transplant recipient with presumed GvHD-related fulminant myocarditis.
Ivaturi V, Dvorak CC, Chan D, Liu T, Cowan MJ, Wahlstrom J, Stricherz M, Jennissen C, Orchard PJ, Tolar J, Pai SY, Huang L, Aweeka F, Long-Boyle J
Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients. Biol Blood Marrow Transplant. 2017 Oct; 23(10):1701-1713.
Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients.
Langelier C, Zinter MS, Kalantar K, Yanik GA, Christenson S, O'Donovan B, White C, Wilson M, Sapru A, Dvorak CC, Miller S, Chiu CY, DeRisi JL
American journal of respiratory and critical care medicine
Metagenomic Sequencing Detects Respiratory Pathogens in Hematopoietic Cellular Transplant Patients. Am J Respir Crit Care Med. 2018 02 15; 197(4):524-528.
Metagenomic Sequencing Detects Respiratory Pathogens in Hematopoietic Cellular Transplant Patients.
Esbenshade AJ, Zhao Z, Aftandilian C, Saab R, Wattier RL, Beauchemin M, Miller TP, Wilkes JJ, Kelly MJ, Fernbach A, Jeng M, Schwartz CL, Dvorak CC, Shyr Y, Moons KGM, Sulis ML, Friedman DL
Multisite external validation of a risk prediction model for the diagnosis of blood stream infections in febrile pediatric oncology patients without severe neutropenia. Cancer. 2017 Oct 01; 123(19):3781-3790.
Multisite external validation of a risk prediction model for the diagnosis of blood stream infections in febrile pediatric oncology patients without severe neutropenia.
Bajwa RPS, Mahadeo KM, Taragin BH, Dvorak CC, McArthur J, Jeyapalan A, Duncan CN, Tamburro R, Gehred A, Lehmann L, Richardson P, Auletta JJ, Woolfrey AE
Consensus Report by Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees: Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 1: Focus on Investigations, Prophylaxis, and Specific Treatment. Biol Blood Marrow Transplant. 2017 Nov; 23(11):1817-1825.
Consensus Report by Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees: Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 1: Focus on Investigations, Prophylaxis, and Specific Treatment.
Wattier RL, Levy ER, Sabnis AJ, Dvorak CC, Auerbach AD
Infection control and hospital epidemiology
Reducing Second Gram-Negative Antibiotic Therapy on Pediatric Oncology and Hematopoietic Stem Cell Transplantation Services. Infect Control Hosp Epidemiol. 2017 09; 38(9):1039-1047.
Reducing Second Gram-Negative Antibiotic Therapy on Pediatric Oncology and Hematopoietic Stem Cell Transplantation Services.
Hematology/oncology clinics of North America
Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy. Hematol Oncol Clin North Am. 2017 10; 31(5):809-822.
Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy.
Zinter MS, Melton A, Sabnis AJ, Dvorak CC, Elicker BM, Nawaytou HM, Kameny RJ, Fineman JR
Pulmonary veno-occlusive disease in a pediatric hematopoietic stem cell transplant patient: a cautionary tale. Leuk Lymphoma. 2018 06; 59(6):1494-1497.
Pulmonary veno-occlusive disease in a pediatric hematopoietic stem cell transplant patient: a cautionary tale.
Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC
Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study. Blood. 2017 12 21; 130(25):2718-2727.
Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.
Mahadeo KM, McArthur J, Adams RH, Radhi M, Angelo J, Jeyapalan A, Nicol K, Su L, Rabi H, Auletta JJ, Pai V, Duncan CN, Tamburro R, Dvorak CC, Bajwa RPS
Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplant Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents: Part 2-Focus on Ascites, Fluid and Electrolytes, Renal, and Transfusion Issues. Biol Blood Marrow Transplant. 2017 Dec; 23(12):2023-2033.
Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplant Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents: Part 2-Focus on Ascites, Fluid and Electrolytes, Renal, and Transfusion Issues.
Ovchinsky N, Frazier W, Auletta JJ, Dvorak CC, Ardura M, Song E, McArthur J, Jeyapalan A, Tamburro R, Mahadeo KM, Traube C, Duncan CN, Bajwa RPS
Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 3: Focus on Cardiorespiratory Dysfunction, Infections, Liver Dysfunction, and Delirium. Biol Blood Marrow Transplant. 2018 02; 24(2):207-218.
Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 3: Focus on Cardiorespiratory Dysfunction, Infections, Liver Dysfunction, and Delirium.
Zinter MS, Holubkov R, Steurer MA, Dvorak CC, Duncan CN, Sapru A, Tamburro RF, McQuillen PS, Pollack MM
Pediatric Hematopoietic Cell Transplant Patients Who Survive Critical Illness Frequently Have Significant but Recoverable Decline in Functional Status. Biol Blood Marrow Transplant. 2018 02; 24(2):330-336.
Pediatric Hematopoietic Cell Transplant Patients Who Survive Critical Illness Frequently Have Significant but Recoverable Decline in Functional Status.
Shukla P, Goswami S, Keizer RJ, Winger BA, Kharbanda S, Dvorak CC, Long-Boyle J
Assessment of a Model-Informed Precision Dosing Platform Use in Routine Clinical Care for Personalized Busulfan Therapy in the Pediatric Hematopoietic Cell Transplantation (HCT) Population. Front Pharmacol. 2020; 11:888.
Assessment of a Model-Informed Precision Dosing Platform Use in Routine Clinical Care for Personalized Busulfan Therapy in the Pediatric Hematopoietic Cell Transplantation (HCT) Population.
Shukla P, Dvorak CC, Long-Boyle J, Kharbanda S
International journal of molecular sciences
Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients. Int J Mol Sci. 2020 Aug 06; 21(16).
Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients.
Behnert A, Lee AG, Young EP, Breese MR, Leung SG, Behroozfard I, Maruffi M, Sweet-Cordero EA, Dvorak CC, Chu J, Stieglitz E
NUP98-NSD1 Driven MDS/MPN in Childhood Masquerading as JMML. J Pediatr Hematol Oncol. 2021 08 01; 43(6):e808-e811.
NUP98-NSD1 Driven MDS/MPN in Childhood Masquerading as JMML.
Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Martinez C, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium
Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2020 Nov; 40(8):1211-1213.
Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.
Orgel E, Freyer DR, Ullrich NJ, Hardy KK, Thomas SM, Dvorak CC, Esbenshade AJ
Assessment of provider perspectives on otoprotection research for children and adolescents: A Children's Oncology Group Cancer Control and Supportive Care Committee survey. Pediatr Blood Cancer. 2020 11; 67(11):e28647.
Assessment of provider perspectives on otoprotection research for children and adolescents: A Children's Oncology Group Cancer Control and Supportive Care Committee survey.
Barzaghi F, Amaya Hernandez LC, Neven B, Ricci S, Kucuk ZY, Bleesing JJ, Nademi Z, Slatter MA, Ulloa ER, Shcherbina A, Roppelt A, Worth A, Silva J, Aiuti A, Murguia-Favela L, Speckmann C, Carneiro-Sampaio M, Fernandes JF, Baris S, Ozen A, Karakoc-Aydiner E, Kiykim A, Schulz A, Steinmann S, Notarangelo LD, Gambineri E, Lionetti P, Shearer WT, Forbes LR, Martinez C, Moshous D, Blanche S, Fisher A, Ruemmele FM, Tissandier C, Ouachee-Chardin M, Rieux-Laucat F, Cavazzana M, Qasim W, Lucarelli B, Albert MH, Kobayashi I, Alonso L, Diaz De Heredia C, Kanegane H, Lawitschka A, Seo JJ, Gonzalez-Vicent M, Diaz MA, Goyal RK, Sauer MG, Yesilipek A, Kim M, Yilmaz-Demirdag Y, Bhatia M, Khlevner J, Richmond Padilla EJ, Martino S, Montin D, Neth O, Molinos-Quintana A, Valverde-Fernandez J, Broides A, Pinsk V, Ballauf A, Haerynck F, Bordon V, Dhooge C, Garcia-Lloret ML, Bredius RG, Kalwak K, Haddad E, Seidel MG, Duckers G, Pai SY, Dvorak CC, Ehl S, Locatelli F, Goldman F, Gennery AR, Cowan MJ, Roncarolo MG, Bacchetta R, Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. J Allergy Clin Immunol. 2018 03; 141(3):1036-1049.e5.
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.
Dvorak CC, Puck JM, Wahlstrom JT, Dorsey M, Melton A, Cowan MJ
Neurologic event-free survival demonstrates a benefit for SCID patients diagnosed by newborn screening. Blood Adv. 2017 Sep 12; 1(20):1694-1698.
Neurologic event-free survival demonstrates a benefit for SCID patients diagnosed by newborn screening.
Dorsey MJ, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier GDE, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J
Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 01; 41(1):38-50.
Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.
How We Treat Fever and Hypotension in Pediatric Hematopoietic Cell Transplant Patients. Front Oncol. 2020; 10:581447.
How We Treat Fever and Hypotension in Pediatric Hematopoietic Cell Transplant Patients.
Journal of health care for the poor and underserved
Hospital resource utilization among patients with sickle cell disease. J Health Care Poor Underserved. 2003 Feb; 14(1):122-35.
Hospital resource utilization among patients with sickle cell disease.
Harris AC, Boelens JJ, Ahn KW, Fei M, Abraham A, Artz A, Dvorak C, Frangoul H, Freytes C, Gale RP, Hong S, Lazarus HM, Loren A, Mineishi S, Nishihori T, O'Brien T, Williams K, Pasquini MC, Levine JE
Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine. Blood Adv. 2018 06 12; 2(11):1198-1206.
Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine.
Dvorak CC, Steinbach WJ, Brown JM, Agarwal R
Risks and outcomes of invasive fungal infections in pediatric patients undergoing allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2005 Oct; 36(7):621-9.
Risks and outcomes of invasive fungal infections in pediatric patients undergoing allogeneic hematopoietic cell transplantation.
Dvorak CC, Sanders RP, Dahl GV, Donaldson SS, Razzouk BI
Reinduction of relapsed acute promyelocytic leukemia with ATRA and low dose antimetabolite-based chemotherapy. Pediatr Blood Cancer. 2007 May; 48(5):582-5.
Reinduction of relapsed acute promyelocytic leukemia with ATRA and low dose antimetabolite-based chemotherapy.
Use of intravenous mycophenolate mofetil for graft-versus-host disease prophylaxis in an allogeneic hematopoietic stem cell transplant recipient with an allergic reaction to cyclosporine and tacrolimus. Bone Marrow Transplant. 2006 Aug; 38(3):253-4.
Use of intravenous mycophenolate mofetil for graft-versus-host disease prophylaxis in an allogeneic hematopoietic stem cell transplant recipient with an allergic reaction to cyclosporine and tacrolimus.
Optimizing care for GI disorders in children after hematopoietic stem cell transplantation. Gastrointest Endosc. 2006 Sep; 64(3):386-8.
Optimizing care for GI disorders in children after hematopoietic stem cell transplantation.
Hematopoietic stem cell transplantation for primary immunodeficiency disease. Bone Marrow Transplant. 2008 Jan; 41(2):119-26.
Hematopoietic stem cell transplantation for primary immunodeficiency disease.
Sutherland SM, Hong DK, Balagtas J, Gutierrez K, Dvorak CC, Sarwal M
Liposomal amphotericin B associated with severe hyperphosphatemia. Pediatr Infect Dis J. 2008 Jan; 27(1):77-9.
Liposomal amphotericin B associated with severe hyperphosphatemia.
Ozyurek E, Cowan MJ, Koerper MA, Baxter-Lowe LA, Dvorak CC, Horn BN
Increasing mixed chimerism and the risk of graft loss in children undergoing allogeneic hematopoietic stem cell transplantation for non-malignant disorders. Bone Marrow Transplant. 2008 Jul; 42(2):83-91.
Increasing mixed chimerism and the risk of graft loss in children undergoing allogeneic hematopoietic stem cell transplantation for non-malignant disorders.
Dvorak CC, Sandford A, Fong A, Cowan MJ, George TI, Lewis DB
Maternal T-cell engraftment associated with severe hemophagocytosis of the bone marrow in untreated X-linked severe combined immunodeficiency. J Pediatr Hematol Oncol. 2008 May; 30(5):396-400.
Maternal T-cell engraftment associated with severe hemophagocytosis of the bone marrow in untreated X-linked severe combined immunodeficiency.
Burgos A, Zaoutis TE, Dvorak CC, Hoffman JA, Knapp KM, Nania JJ, Prasad P, Steinbach WJ
Pediatric invasive aspergillosis: a multicenter retrospective analysis of 139 contemporary cases. Pediatrics. 2008 May; 121(5):e1286-94.
Pediatric invasive aspergillosis: a multicenter retrospective analysis of 139 contemporary cases.
Dvorak CC, Agarwal R, Dahl GV, Gregory JJ, Feusner JH
Hematopoietic stem cell transplant for pediatric acute promyelocytic leukemia. Biol Blood Marrow Transplant. 2008 Jul; 14(7):824-30.
Hematopoietic stem cell transplant for pediatric acute promyelocytic leukemia.
Dvorak CC, Hung GY, Horn B, Dunn E, Oon CY, Cowan MJ
Megadose CD34(+) cell grafts improve recovery of T cell engraftment but not B cell immunity in patients with severe combined immunodeficiency disease undergoing haplocompatible nonmyeloablative transplantation. Biol Blood Marrow Transplant. 2008 Oct; 14(10):1125-1133.
Megadose CD34(+) cell grafts improve recovery of T cell engraftment but not B cell immunity in patients with severe combined immunodeficiency disease undergoing haplocompatible nonmyeloablative transplantation.
Agarwal R, Dvorak CC, Stockerl-Goldstein KE, Johnston L, Srinivas S
High-dose chemotherapy followed by stem cell rescue for high-risk germ cell tumors: the Stanford experience. Bone Marrow Transplant. 2009 Apr; 43(7):547-52.
High-dose chemotherapy followed by stem cell rescue for high-risk germ cell tumors: the Stanford experience.
Dvorak CC, Wright NB, Wong WB, Kristovich KM, Matthews EW, Weinberg KI, Amylon MD, Agarwal R
Safety of hematopoietic stem cell transplantation in children less than three years of age. Pediatr Hematol Oncol. 2008 Dec; 25(8):705-22.
Safety of hematopoietic stem cell transplantation in children less than three years of age.
Gramatges MM, Dvorak CC, Regula DP, Enns GM, Weinberg K, Agarwal R
Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity. Bone Marrow Transplant. 2009 Oct; 44(7):449-50.
Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity.
Primary graft failure after umbilical cord blood transplant rescued by parental haplocompatible stem cell transplantation. J Pediatr Hematol Oncol. 2009 Apr; 31(4):300-3.
Primary graft failure after umbilical cord blood transplant rescued by parental haplocompatible stem cell transplantation.
Dvorak CC, Gilman AL, Horn B, Jaroscak J, Dunn EA, Baxter-Lowe LA, Cowan MJ
Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions. Bone Marrow Transplant. 2009 Dec; 44(12):805-12.
Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions.
Dvorak CC, Cowan MJ, Horn B, Weintrub PS
Clinical infectious diseases : an official publication of the Infectious Diseases Society of America
Development of herpes simplex virus stomatitis during receipt of cidofovir therapy. Clin Infect Dis. 2009 Oct 15; 49(8):e92-5.
Development of herpes simplex virus stomatitis during receipt of cidofovir therapy.
Zerr DM, Milstone AM, Dvorak CC, Adler AL, Chen L, Villaluna D, Dang H, Qin X, Addetia A, Yu LC, Conway Keller M, Esbenshade AJ, August KJ, Fisher BT, Sung L
Chlorhexidine gluconate bathing in children with cancer or those undergoing hematopoietic stem cell transplantation: A double-blinded randomized controlled trial from the Children's Oncology Group. Cancer. 2021 01 01; 127(1):56-66.
Chlorhexidine gluconate bathing in children with cancer or those undergoing hematopoietic stem cell transplantation: A double-blinded randomized controlled trial from the Children's Oncology Group.
The tortoise and the hare: slow and steady stem cells win the race. Leuk Res. 2010 May; 34(5):572-3.
The tortoise and the hare: slow and steady stem cells win the race.
Immunology and allergy clinics of North America
Radiosensitive severe combined immunodeficiency disease. Immunol Allergy Clin North Am. 2010 Feb; 30(1):125-42.
Radiosensitive severe combined immunodeficiency disease.
Kussmaul SC, Horn BN, Dvorak CC, Abramovitz L, Cowan MJ, Weintrub PS
Safety of the live, attenuated varicella vaccine in pediatric recipients of hematopoietic SCTs. Bone Marrow Transplant. 2010 Nov; 45(11):1602-6.
Safety of the live, attenuated varicella vaccine in pediatric recipients of hematopoietic SCTs.
Hogeling M, Nakano T, Dvorak CC, Maguiness S, Frieden IJ
Severe neonatal congenital erythropoietic porphyria. Pediatr Dermatol. 2011 Jul-Aug; 28(4):416-20.
Severe neonatal congenital erythropoietic porphyria.
Nemecek ER, Ellis K, He W, Bunin NJ, Bajwa RS, Cheerva A, Cairo MS, Dvorak C, Duval M, Davies S, Eapen M, Gross TG, Hussein AA, MacMillan ML, Mehta PA, Pulsipher MA, Seber A, Woolfrey AE, Frangoul HA, Carpenter PA
Outcome of myeloablative conditioning and unrelated donor hematopoietic cell transplantation for childhood acute lymphoblastic leukemia in third remission. Biol Blood Marrow Transplant. 2011 Dec; 17(12):1833-40.
Outcome of myeloablative conditioning and unrelated donor hematopoietic cell transplantation for childhood acute lymphoblastic leukemia in third remission.
Pereira S, Vayntrub T, Hiraki DD, Cherry AM, Arai S, Dvorak CC, Grumet FC
Short tandem repeat and human leukocyte antigen mutations or losses confound engraftment and typing analysis in hematopoietic stem cell transplants. Hum Immunol. 2011 Jun; 72(6):503-9.
Short tandem repeat and human leukocyte antigen mutations or losses confound engraftment and typing analysis in hematopoietic stem cell transplants.
Baker KS, Bhatia S, Bunin N, Nieder M, Dvorak CC, Sung L, Sanders JE, Kurtzberg J, Pulsipher MA
NCI, NHLBI first international consensus conference on late effects after pediatric hematopoietic cell transplantation: state of the science, future directions. Biol Blood Marrow Transplant. 2011 Oct; 17(10):1424-7.
NCI, NHLBI first international consensus conference on late effects after pediatric hematopoietic cell transplantation: state of the science, future directions.
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Outcomes of Measurable Residual Disease in Pediatric Acute Myeloid Leukemia before and after Hematopoietic Stem Cell Transplant: Validation of Difference from Normal Flow Cytometry with Chimerism Studies and Wilms Tumor 1 Gene Expression. Biol Blood Marrow Transplant. 2018 10; 24(10):2040-2046.
Outcomes of Measurable Residual Disease in Pediatric Acute Myeloid Leukemia before and after Hematopoietic Stem Cell Transplant: Validation of Difference from Normal Flow Cytometry with Chimerism Studies and Wilms Tumor 1 Gene Expression.
Dvorak CC, Fisher BT, Esbenshade AJ, Nieder ML, Alexander S, Steinbach WJ, Dang H, Villaluna D, Chen L, Skeens M, Zaoutis TE, Sung L
A Randomized Trial of Caspofungin vs Triazoles Prophylaxis for Invasive Fungal Disease in Pediatric Allogeneic Hematopoietic Cell Transplant. J Pediatric Infect Dis Soc. 2021 Apr 30; 10(4):417-425.
A Randomized Trial of Caspofungin vs Triazoles Prophylaxis for Invasive Fungal Disease in Pediatric Allogeneic Hematopoietic Cell Transplant.
Forlanini F, Dara J, Dvorak CC, Cowan MJ, Puck JM, Dorsey MJ
Unknown cytomegalovirus serostatus in primary immunodeficiency disorders: A new category of transplant recipients. Transpl Infect Dis. 2021 Apr; 23(2):e13504.
Unknown cytomegalovirus serostatus in primary immunodeficiency disorders: A new category of transplant recipients.
Qayed M, Ahn KW, Kitko CL, Johnson MH, Shah NN, Dvorak C, Mellgren K, Friend BD, Verneris MR, Leung W, Toporski J, Levine J, Chewning J, Wayne A, Kapoor U, Triplett B, Schultz KR, Yanik GA, Eapen M
A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation. Blood. 2021 02 18; 137(7):983-993.
A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation.
Dorsey M, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Burroughs LM, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier G, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J
Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 Feb; 41(2):498-500.
Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers.
Zinter MS, Lindemans CA, Versluys BA, Mayday MY, Sunshine S, Reyes G, Sirota M, Sapru A, Matthay MA, Kharbanda S, Dvorak CC, Boelens JJ, DeRisi JL
The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury. Blood. 2021 03 25; 137(12):1679-1689.
The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury.
Dandoy CE, Rotz S, Alonso PB, Klunk A, Desmond C, Huber J, Ingraham H, Higham C, Dvorak CC, Duncan C, Schoettler M, Lehmann L, Cancio M, Killinger J, Davila B, Phelan R, Mahadeo KM, Khazal S, Lalefar N, Vissa M, Myers K, Wallace G, Nelson A, Khandelwal P, Bhatla D, Gloude N, Anderson E, Huo J, Roehrs P, Auletta JJ, Chima R, Lane A, Davies SM, Jodele S
A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant. Blood Adv. 2021 01 12; 5(1):1-11.
A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant.
Bajwa RPS, Taylor K, Hoyt A, Kamboj MK, Stanek J, Mahadeo KM, Alsaedi H, Abdel-Azim H, O'Kane S, Martin PL, Stafford LA, Dvorak CC
Vitamin D has no impact on outcomes after HSCT in children-A retrospective study. Pediatr Transplant. 2021 Jun; 25(4):e14008.
Vitamin D has no impact on outcomes after HSCT in children-A retrospective study.
Apsel Winger B, Shukla P, Kharbanda S, Keizer RJ, Goswami S, Cowan MJ, Dvorak CC, Long-Boyle J
The Relationship Between Busulfan Exposure and Achievement of Sustained Donor Myeloid Chimerism in Patients with Non-Malignant Disorders. Transplant Cell Ther. 2021 03; 27(3):258.e1-258.e6.
The Relationship Between Busulfan Exposure and Achievement of Sustained Donor Myeloid Chimerism in Patients with Non-Malignant Disorders.
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Musculoskeletal impairments in children receiving intensive therapy for acute leukemia or undergoing hematopoietic stem cell transplant: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2021 08; 68(8):e29053.
Musculoskeletal impairments in children receiving intensive therapy for acute leukemia or undergoing hematopoietic stem cell transplant: A report from the Children's Oncology Group.
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Transplant-associated thrombotic microangiopathy in pediatric patients: pre-HSCT risk stratification and prophylaxis. Blood Adv. 2021 04 27; 5(8):2106-2114.
Transplant-associated thrombotic microangiopathy in pediatric patients: pre-HSCT risk stratification and prophylaxis.
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Reply to Are we certain that chlorhexidine gluconate bathing is not beneficial in deducing central line associated blood stream infections among children with cancer or undergoing hematopoietic stem cell transplantation? Cancer. 2021 08 01; 127(15):2813-2814.
Reply to Are we certain that chlorhexidine gluconate bathing is not beneficial in deducing central line associated blood stream infections among children with cancer or undergoing hematopoietic stem cell transplantation?
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Outcome of domino hematopoietic stem cell transplantation in human subjects: An international case series.
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International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome. J Allergy Clin Immunol. 2022 01; 149(1):410-421.e7.
International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome.
Clinical Profile at UCSF Benioff Children's Hospital
Fisher BT, Westling T, Boge CLK, Zaoutis TE, Dvorak CC, Nieder M, Zerr DM, Wingard JR, Villaluna D, Esbenshade AJ, Alexander S, Gunn S, Wheat LJ, Sung L
Prospective Evaluation of Galactomannan and (1→3) β-d-Glucan Assays as Diagnostic Tools for Invasive Fungal Disease in Children, Adolescents, and Young Adults With Acute Myeloid Leukemia Receiving Fungal Prophylaxis. J Pediatric Infect Dis Soc. 2021 Sep 23; 10(8):864-871.
Prospective Evaluation of Galactomannan and (1→3) β-d-Glucan Assays as Diagnostic Tools for Invasive Fungal Disease in Children, Adolescents, and Young Adults With Acute Myeloid Leukemia Receiving Fungal Prophylaxis.
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A Guidance for Concomitant Drug Reconciliation Prior to Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults. Front Pediatr. 2021; 9:713091.
A Guidance for Concomitant Drug Reconciliation Prior to Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults.
Marsh RA, Hebert K, Kim S, Dvorak CC, Aquino VM, Baker KS, Chellapandian D, Dávila Saldaña B, Duncan CN, Eckrich MJ, Georges GE, Olson TS, Pulsipher MA, Shenoy S, Stenger E, Lugt MV, Yu LC, Gennery AR, Eapen M
Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders. J Allergy Clin Immunol. 2022 03; 149(3):1097-1104.e2.
Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders.
Delmonte OM, Castagnoli R, Yu J, Dvorak CC, Cowan MJ, Dávila Saldaña BJ, De Ravin SS, Mamcarz E, Chang CK, Daley SR, Griffith LM, Notarangelo LD, Puck JM
Poor T-cell receptor β repertoire diversity early posttransplant for severe combined immunodeficiency predicts failure of immune reconstitution. J Allergy Clin Immunol. 2022 03; 149(3):1113-1119.
Poor T-cell receptor β repertoire diversity early posttransplant for severe combined immunodeficiency predicts failure of immune reconstitution.
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Juvenile myelomonocytic leukemia in the molecular era: a clinician's guide to diagnosis, risk stratification, and treatment. Blood Adv. 2021 11 23; 5(22):4783-4793.
Juvenile myelomonocytic leukemia in the molecular era: a clinician's guide to diagnosis, risk stratification, and treatment.
Miller TP, Marx MZ, Henchen C, DeGroote NP, Jones S, Weiland J, Fisher B, Esbenshade AJ, Aplenc R, Dvorak CC, Fisher BT
Challenges and Barriers to Adverse Event Reporting in Clinical Trials: A Children's Oncology Group Report. J Patient Saf. 2022 Apr 01; 18(3):e672-e679.
Challenges and Barriers to Adverse Event Reporting in Clinical Trials: A Children's Oncology Group Report.
Zinter MS, Delucchi KL, Kong MY, Orwoll BE, Spicer AS, Lim MJ, Alkhouli MF, Ratiu AE, McKenzie AV, McQuillen PS, Dvorak CC, Calfee CS, Matthay MA, Sapru A
Early Plasma Matrix Metalloproteinase Profiles. A Novel Pathway in Pediatric Acute Respiratory Distress Syndrome. Am J Respir Crit Care Med. 2019 01 15; 199(2):181-189.
Early Plasma Matrix Metalloproteinase Profiles. A Novel Pathway in Pediatric Acute Respiratory Distress Syndrome.
Frederick NN, Klosky JL, Meacham LR, Quinn GP, Kelvin JF, Cherven B, Freyer DR, Dvorak CC, Brackett J, Ahmed-Winston S, Bryson E, Chow EJ, Levine J
Infrastructure of Fertility Preservation Services for Pediatric Cancer Patients: A Report From the Children's Oncology Group. JCO Oncol Pract. 2022 03; 18(3):e325-e333.
Infrastructure of Fertility Preservation Services for Pediatric Cancer Patients: A Report From the Children's Oncology Group.
The Beginning of the End of Allogeneic Transplantation for Hurler Syndrome? N Engl J Med. 2021 11 18; 385(21):2003-2004.
The Beginning of the End of Allogeneic Transplantation for Hurler Syndrome?
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Hematopoietic Cell Transplantation-Comorbidity Index Score Is Correlated with Treatment-Related Mortality and Overall Survival following Second Allogeneic Hematopoietic Cell Transplantation in Children. Transplant Cell Ther. 2022 03; 28(3):155.e1-155.e8.
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Population Pharmacokinetic Model Development of Tacrolimus in Pediatric and Young Adult Patients Undergoing Hematopoietic Cell Transplantation. Front Pharmacol. 2021; 12:750672.
Population Pharmacokinetic Model Development of Tacrolimus in Pediatric and Young Adult Patients Undergoing Hematopoietic Cell Transplantation.
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Therapy-related myeloid neoplasms resembling juvenile myelomonocytic leukemia: a case series and review of the literature. Pediatr Blood Cancer. 2022 05; 69(5):e29499.
Therapy-related myeloid neoplasms resembling juvenile myelomonocytic leukemia: a case series and review of the literature.
Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood. 2018 10 25; 132(17):1737-1749.
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.
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Population Pharmacokinetics of Melphalan for Pediatric Patients Undergoing Hematopoietic Cell Transplantation. J Clin Pharmacol. 2022 07; 62(7):873-882.
Population Pharmacokinetics of Melphalan for Pediatric Patients Undergoing Hematopoietic Cell Transplantation.
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Association Between the Magnitude of Intravenous Busulfan Exposure and Development of Hepatic Veno-Occlusive Disease in Children and Young Adults Undergoing Myeloablative Allogeneic Hematopoietic Cell Transplantation. Transplant Cell Ther. 2022 04; 28(4):196-202.
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The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018). J Allergy Clin Immunol. 2019 01; 143(1):405-407.
The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018).
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A pilot trial of prophylactic defibrotide to prevent serious thrombotic microangiopathy in high-risk pediatric patients. Pediatr Blood Cancer. 2022 05; 69(5):e29641.
A pilot trial of prophylactic defibrotide to prevent serious thrombotic microangiopathy in high-risk pediatric patients.
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Pulmonary Metagenomic Sequencing Suggests Missed Infections in Immunocompromised Children. Clin Infect Dis. 2019 05 17; 68(11):1847-1855.
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Comment on: Olanzapine for chemotherapy-induced nausea: Lessons learned from child and adolescent psychiatry. Pediatr Blood Cancer. 2019 01; 66(1):e27456.
Comment on: Olanzapine for chemotherapy-induced nausea: Lessons learned from child and adolescent psychiatry.
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Effect of Levofloxacin Prophylaxis on Bacteremia in Children With Acute Leukemia or Undergoing Hematopoietic Stem Cell Transplantation: A Randomized Clinical Trial. JAMA. 2018 09 11; 320(10):995-1004.
Effect of Levofloxacin Prophylaxis on Bacteremia in Children With Acute Leukemia or Undergoing Hematopoietic Stem Cell Transplantation: A Randomized Clinical Trial.
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Pulmonary microbiome and gene expression signatures differentiate lung function in pediatric hematopoietic cell transplant candidates. Sci Transl Med. 2022 03 09; 14(635):eabm8646.
Pulmonary microbiome and gene expression signatures differentiate lung function in pediatric hematopoietic cell transplant candidates.
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Identifying clinical practice guidelines for the supportive care of children with cancer: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2019 01; 66(1):e27471.
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Daratumumab in life-threatening autoimmune hemolytic anemia following hematopoietic stem cell transplantation.
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Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 08 18; 140(7):685-705.
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
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Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2019 08; 54(8):1254-1265.
Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation.
Pulsipher MA, Logan BR, Kiefer DM, Chitphakdithai P, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Varni JW, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Ball ED, Bolwell BJ, Bunin NJ, Cheerva A, Delgado DC, Dvorak CC, Gillio AP, Hahn TE, Hale GA, Haight AE, Hayes-Lattin BM, Kasow KA, Linenberger M, Magalhaes-Silverman M, Mori S, Prasad VK, Quigg TC, Sahdev I, Schriber JR, Shenoy S, Tse WT, Yanik GA, Navarro WH, Horowitz MM, Confer DL, Shaw BE, Switzer GE
Higher Risks of Toxicity and Incomplete Recovery in 13- to 17-Year-Old Females after Marrow Donation: RDSafe Peds Results. Biol Blood Marrow Transplant. 2019 05; 25(5):955-964.
Higher Risks of Toxicity and Incomplete Recovery in 13- to 17-Year-Old Females after Marrow Donation: RDSafe Peds Results.
Forlanini F, Chan A, Dara J, Dvorak CC, Cowan MJ, Puck JM, Dorsey MJ
Impact of Genetic Diagnosis on the Outcome of Hematopoietic Stem Cell Transplant in Primary Immunodeficiency Disorders. J Clin Immunol. 2023 04; 43(3):636-646.
Impact of Genetic Diagnosis on the Outcome of Hematopoietic Stem Cell Transplant in Primary Immunodeficiency Disorders.
Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Dávila Saldaña BJ, Eissa H, Goldman FD, Heimall J, O'Reilly R, Chaudhury S, Kolb EA, Shenoy S, Griffith LM, Pulsipher M, Kohn DB, Notarangelo LD, Pai SY, Cowan MJ, Dvorak CC, Haddad É, Puck JM, Barreiro LB, Decaluwe H
Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation. J Allergy Clin Immunol. 2023 01; 151(1):260-271.
Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation.
Otto WR, Dvorak CC, Boge CLK, Ostrosky-Zeichner L, Esbenshade AJ, Nieder ML, Alexander S, Steinbach WJ, Dang H, Villaluna D, Chen L, Skeens M, Zaoutis TE, Sung L, Fisher BT
Prospective Evaluation of the Fungitell® (1→3) Beta-D-Glucan Assay as a Diagnostic Tool for Invasive Fungal Disease in Pediatric Allogeneic Hematopoietic Cell Transplantation: A Report from the Children's Oncology Group. Pediatr Transplant. 2023 02; 27(1):e14399.
Prospective Evaluation of the Fungitell® (1→3) Beta-D-Glucan Assay as a Diagnostic Tool for Invasive Fungal Disease in Pediatric Allogeneic Hematopoietic Cell Transplantation: A Report from the Children's Oncology Group.
Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, Abraham RS, Satter LF, Notarangelo LD, Puck JM
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):539-546.
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions.
Cowan MJ, Yu J, Facchino J, Fraser-Browne C, Sanford U, Kawahara M, Dara J, Long-Boyle J, Oh J, Chan W, Chag S, Broderick L, Chellapandian D, Decaluwe H, Golski C, Hu D, Kuo CY, Miller HK, Petrovic A, Currier R, Hilton JF, Punwani D, Dvorak CC, Malech HL, McIvor RS, Puck JM
Lentiviral Gene Therapy for Artemis-Deficient SCID. N Engl J Med. 2022 12 22; 387(25):2344-2355.
Lentiviral Gene Therapy for Artemis-Deficient SCID.
Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):547-555.e5.
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.
Brooks JT, Solans BP, Lu Y, Kharbanda S, Dvorak CC, Lalefar N, Long S, Gupta AO, Horn B, Lamba JK, Huang L, Apsel-Winger B, Keizer RJ, Savic R, Long-Boyle J
Prospective Validation and Refinement of a Population Pharmacokinetic Model of Fludarabine in Children and Young Adults Undergoing Hematopoietic Cell Transplantation. Pharmaceutics. 2022 Nov 15; 14(11).
Prospective Validation and Refinement of a Population Pharmacokinetic Model of Fludarabine in Children and Young Adults Undergoing Hematopoietic Cell Transplantation.
Frederick NN, Klosky JL, Meacham L, Quinn GP, Kelvin JF, Cherven B, Freyer DR, Dvorak CC, Brackett J, Ahmed-Winston S, Bryson E, Su HI, Chow EJ, Levine J
Fertility Preservation Practices at Pediatric Oncology Institutions in the United States: A Report From the Children's Oncology Group. JCO Oncol Pract. 2023 04; 19(4):e550-e558.
Fertility Preservation Practices at Pediatric Oncology Institutions in the United States: A Report From the Children's Oncology Group.
Gale R, Hinterberger W, Young NS, Gennery A, Dvorak C, Hebert K, Heim M, Broglie L, Eapen M
What Causes Aplastic Anaemia: Results of Transplants from Genetically-Identical Twins. Res Sq. 2023 Feb 03.
What Causes Aplastic Anaemia: Results of Transplants from Genetically-Identical Twins.
Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, Moore TB, Naides SJ, Sciortino S, Feuchtbaum L, Koupaei RA, Puck JM
Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017. Pediatrics. 2019 02; 143(2).
Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017.
Ferrua F, Galimberti S, Courteille V, Slatter MA, Booth C, Moshous D, Neven B, Blanche S, Cavazzana M, Laberko A, Shcherbina A, Balashov D, Soncini E, Porta F, Al-Mousa H, Al-Saud B, Al-Dhekri H, Arnaout R, Formankova R, Bertrand Y, Lange A, Smart J, Wolska-Kusnierz B, Aquino VM, Dvorak CC, Fasth A, Fouyssac F, Heilmann C, Hoenig M, Schuetz C, Kelecic J, Bredius RGM, Lankester AC, Lindemans CA, Suarez F, Sullivan KE, Albert MH, Kalwak K, Barlogis V, Bhatia M, Bordon V, Czogala W, Alonso L, Dogu F, Gozdzik J, Ikinciogullari A, Kriván G, Ljungman P, Meyts I, Mustillo P, Smith AR, Speckmann C, Sundin M, Keogh SJ, Shaw PJ, Boelens JJ, Schulz AS, Sedlacek P, Veys P, Mahlaoui N, Janda A, Davies EG, Fischer A, Cowan MJ, Gennery AR, SCETIDE, PIDTC, EBMT & ESID IEWP
Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study. J Allergy Clin Immunol. 2019 06; 143(6):2238-2253.
Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study.
Knight TE, Ahn KW, Hebert KM, Atshan R, Wall DA, Chiengthong K, Rotz SJ, Fraint E, Rangarajan HG, Auletta JJ, Sharma A, Kitko CL, Hashem H, Williams KM, Wirk B, Dvorak CC, Myers KC, Pulsipher MA, Warwick AB, Lalefar NR, Schultz KR, Qayed M, Broglie L, Eapen M, Yanik GA
Effect of Autograft CD34+ Dose on Outcome in Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors. Transplant Cell Ther. 2023 06; 29(6):380.e1-380.e9.
Effect of Autograft CD34+ Dose on Outcome in Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors.
Gale RP, Hinterberger W, Young NS, Gennery AR, Dvorak CC, Hebert KM, Heim M, Broglie L, Eapen M
What causes aplastic anaemia? Leukemia. 2023 06; 37(6):1191-1193.
Esbenshade AJ, Sung L, Brackett J, Dupuis LL, Fisher BT, Grimes A, Miller TP, Ullrich NJ, Dvorak CC, Cancer Control and Supportive Care Committee
Children's Oncology Group's 2023 blueprint for research: Cancer control and supportive care. Pediatr Blood Cancer. 2023 09; 70 Suppl 6:e30568.
Children's Oncology Group's 2023 blueprint for research: Cancer control and supportive care.
Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium. Lancet. 2023 07 08; 402(10396):129-140.
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.
Huang L, Cheah V, Chan D, Marzan F, Dvorak CC, Aweeka FT, Long-Boyle J
Determination of melphalan in human plasma by UPLC-UV method. Cancer Chemother Pharmacol. 2019 05; 83(5):905-910.
Determination of melphalan in human plasma by UPLC-UV method.
Dvorak CC, Long-Boyle J, Dara J, Melton A, Shimano KA, Huang JN, Puck JM, Dorsey MJ, Facchino J, Chang CK, Cowan MJ
Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency. Biol Blood Marrow Transplant. 2019 07; 25(7):1355-1362.
Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency.
Parikh SH, Satwani P, Ahn KW, Sahr NA, Fretham C, Abraham AA, Agrawal V, Auletta JJ, Abdel-Azim H, Copelan E, Diaz MA, Dvorak CC, Frangoul HA, Freytes CO, Gadalla SM, Gale RP, George B, Gergis U, Hashmi S, Hematti P, Hildebrandt GC, Keating AK, Lazarus HM, Myers KC, Olsson RF, Prestidge T, Rotz SJ, Savani BN, Shereck EB, Williams KM, Wirk B, Pasquini MC, Loren AW
Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant. JAMA Pediatr. 2019 05 01; 173(5):e190081.
Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant.
Leiding JW, Arnold DE, Parikh S, Logan B, Marsh RA, Griffith LM, Wu R, Kidd S, Mallhi K, Chellapandian D, Si Lim SJ, Grunebaum E, Falcone EL, Murguia-Favela L, Grossman D, Prasad VK, Heimall JR, Touzot F, Burroughs LM, Bleesing J, Kapoor N, Dara J, Williams O, Kapadia M, Oshrine BR, Bednarski JJ, Rayes A, Chong H, Cuvelier GDE, Forbes Satter LR, Martinez C, Vander Lugt MT, Yu LC, Chandrakasan S, Joshi A, Prockop SE, Dávila Saldaña BJ, Aquino V, Broglie LA, Ebens CL, Madden LM, DeSantes K, Milner J, Rangarajan HG, Shah AJ, Gillio AP, Knutsen AP, Miller HK, Moore TB, Graham P, Bauchat A, Bunin NJ, Teira P, Petrovic A, Chandra S, Abdel-Azim H, Dorsey MJ, Birbrayer O, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM
Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD. Blood. 2023 12 14; 142(24):2105-2118.
Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD.
Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. J Allergy Clin Immunol. 2023 12; 152(6):1619-1633.e11.
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease.
Schoettler ML, Dandoy CE, Harris A, Chan M, Tarquinio KM, Jodele S, Qayed M, Watkins B, Kamat P, Petrillo T, Obordo J, Higham CS, Dvorak CC, Westbrook A, Zinter MS, Williams KM
Diffuse alveolar hemorrhage after hematopoietic cell transplantation- response to treatments and risk factors for mortality. Front Oncol. 2023; 13:1232621.
Diffuse alveolar hemorrhage after hematopoietic cell transplantation- response to treatments and risk factors for mortality.
Zinter MS, Brazauskas R, Strom J, Chen S, Bo-Subait S, Sharma A, Beitinjaneh A, Dimitrova D, Guilcher G, Preussler J, Myers K, Bhatt NS, Ringden O, Hematti P, Hayashi RJ, Patel S, De Oliveira SN, Rotz S, Badawy SM, Nishihori T, Buchbinder D, Hamilton B, Savani B, Schoemans H, Sorror M, Winestone L, Duncan C, Phelan R, Dvorak CC
medRxiv : the preprint server for health sciences
Critical Illness Risk and Long-Term Outcomes Following Intensive Care in Pediatric Hematopoietic Cell Transplant Recipients. medRxiv. 2023 Aug 05.
Critical Illness Risk and Long-Term Outcomes Following Intensive Care in Pediatric Hematopoietic Cell Transplant Recipients.
Knight TE, Ahn KW, Hebert KM, Atshan R, Wall DA, Chiengthong K, Lund TC, Prestidge T, Rangarajan HG, Dvorak CC, Auletta JJ, Kent M, Hashem H, Talano JA, Rotz SJ, Fraint E, Myers KC, Leung W, Sharma A, Bhatt NS, Driscoll TA, Yu LC, Schultz KR, Qayed M, Broglie L, Eapen M, Yanik GA
No impact of CD34+ cell dose on outcome among children undergoing autologous hematopoietic stem cell transplant for high-risk neuroblastoma. Bone Marrow Transplant. 2023 12; 58(12):1390-1393.
No impact of CD34+ cell dose on outcome among children undergoing autologous hematopoietic stem cell transplant for high-risk neuroblastoma.
Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O'Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Dávila Saldaña BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 01; 153(1):287-296.
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.
Zinter MS, Dvorak CC, Mayday MY, Reyes G, Simon MR, Pearce EM, Kim H, Shaw PJ, Rowan CM, Auletta JJ, Martin PL, Godder K, Duncan CN, Lalefar NR, Kreml EM, Hume JR, Abdel-Azim H, Hurley C, Cuvelier GDE, Keating AK, Qayed M, Killinger JS, Fitzgerald JC, Hanna R, Mahadeo KM, Quigg TC, Satwani P, Castillo P, Gertz SJ, Moore TB, Hanisch B, Abdel-Mageed A, Phelan R, Davis DB, Hudspeth MP, Yanik GA, Pulsipher MA, Sulaiman I, Segal LN, Versluys BA, Lindemans CA, Boelens JJ, DeRisi JL, Pediatric Transplantation and Cell Therapy Consortium
Pulmonary microbiome and transcriptome signatures reveal distinct pathobiologic states associated with mortality in two cohorts of pediatric stem cell transplant patients. medRxiv. 2023 Nov 29.
Pulmonary microbiome and transcriptome signatures reveal distinct pathobiologic states associated with mortality in two cohorts of pediatric stem cell transplant patients.
Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant recipients. Blood Adv. 2024 02 27; 8(4):1002-1017.
Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant recipients.
Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguía-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Dávila Saldaña BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study. J Allergy Clin Immunol. 2024 May; 153(5):1423-1431.e2.
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study.
Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Dávila Saldaña BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E
Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clin Immunol. 2024 04; 261:109942.
Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia.
Amarin JZ, Dulek DE, Simmons J, Hayek H, Chappell JD, Nochowicz CH, Kitko CL, Schuster JE, Muñoz FM, Bocchini CE, Moulton EA, Coffin SE, Freedman JL, Ardura MI, Wattier RL, Maron G, Grimley M, Paulsen G, Danziger-Isakov L, Carpenter PA, Englund JA, Halasa NB, Spieker AJ, Kalams SA
Immunophenotypic predictors of influenza vaccine immunogenicity in pediatric hematopoietic cell transplant recipients. Blood Adv. 2024 04 23; 8(8):1880-1892.
Immunophenotypic predictors of influenza vaccine immunogenicity in pediatric hematopoietic cell transplant recipients.
Salinas Cisneros G, Dvorak CC, Long-Boyle J, Kharbanda S, Shimano KA, Melton A, Chu J, Winestone LE, Dara J, Huang JN, Hermiston ML, Zinter M, Higham CS
Diagnosing and Grading of Sinusoidal Obstructive Syndrome after Hematopoietic Stem Cell Transplant of Children, Adolescent and Young Adults treated in a Pediatric Institution with Pediatric Protocols. Transplant Cell Ther. 2024 07; 30(7):690.e1-690.e16.
Diagnosing and Grading of Sinusoidal Obstructive Syndrome after Hematopoietic Stem Cell Transplant of Children, Adolescent and Young Adults treated in a Pediatric Institution with Pediatric Protocols.
Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation. Nat Med. 2024 Jul; 30(7):1982-1993.
Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation.
Neely JA, Dvorak CC, Pantell MS, Melton A, Huang JN, Shimano KA
Autoimmune Cytopenias in Pediatric Hematopoietic Cell Transplant Patients. Front Pediatr. 2019; 7:171.
Autoimmune Cytopenias in Pediatric Hematopoietic Cell Transplant Patients.
Higham CS, Shimano KA, Kharbanda S, Chu J, Cisneros GS, Winestone LE, Dara J, Huang JN, Hermiston ML, Long-Boyle JR, Dvorak CC
Cyclophosphamide and Thiotepa Increases Risk of Transplant-Associated Thrombotic Microangiopathy. Transplant Cell Ther. 2024 09; 30(9):931.e1-931.e10.
Cyclophosphamide and Thiotepa Increases Risk of Transplant-Associated Thrombotic Microangiopathy.
Keller MD, Hanley PJ, Chi YY, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai SY, Dávila Saldaña BJ, Hanisch B, Quigg TC, Adams RH, Dahlberg A, Chandrakasan S, Hasan H, Malvar J, Jensen-Wachspress MA, Lazarski CA, Sani G, Idso JM, Lang H, Chansky P, McCann CD, Tanna J, Abraham AA, Webb JL, Shibli A, Keating AK, Satwani P, Muranski P, Hall E, Eckrich MJ, Shereck E, Miller H, Mamcarz E, Agarwal R, De Oliveira SN, Vander Lugt MT, Ebens CL, Aquino VM, Bednarski JJ, Chu J, Parikh S, Whangbo J, Lionakis M, Zambidis ET, Gourdine E, Bollard CM, Pulsipher MA
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. Nat Commun. 2024 Apr 18; 15(1):3258.
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment.
Smith MA, Cheng G, Phelan R, Brazauskas R, Strom J, Ahn KW, Hamilton BK, Peterson A, Savani B, Schoemans H, Schoettler ML, Sorror M, Keller RL, Higham CS, Dvorak CC, Fineman JR, Zinter MS
Pulmonary hypertension in the intensive care unit after pediatric allogeneic hematopoietic stem cell transplant: incidence, risk factors, and outcomes. Front Oncol. 2024; 14:1415984.
Pulmonary hypertension in the intensive care unit after pediatric allogeneic hematopoietic stem cell transplant: incidence, risk factors, and outcomes.
Cheng G, Smith MA, Phelan R, Brazauskas R, Strom J, Ahn KW, Hamilton B, Peterson A, Savani B, Schoemans H, Schoettler M, Sorror M, Higham C, Kharbanda S, Dvorak CC, Zinter MS
Epidemiology of Diffuse Alveolar Hemorrhage in Pediatric Allogeneic Hematopoietic Cell Transplantation Recipients. Transplant Cell Ther. 2024 10; 30(10):1017.e1-1017.e12.
Epidemiology of Diffuse Alveolar Hemorrhage in Pediatric Allogeneic Hematopoietic Cell Transplantation Recipients.
Dvorak CC, Long-Boyle JR, Holbrook-Brown L, Abdel-Azim H, Bertaina A, Vatsayan A, Talano JA, Bunin N, Anderson E, Flower A, Lalefar N, Higham CS, Kapoor N, Klein O, Odinakachukwu MC, Cho S, Jacobsohn DA, Collier W, Pulsipher MA
Effect of rabbit ATG PK on outcomes after TCR-αβ/CD19-depleted pediatric haploidentical HCT for hematologic malignancy. Blood Adv. 2024 Dec 10; 8(23):6003-6014.
Effect of rabbit ATG PK on outcomes after TCR-αβ/CD19-depleted pediatric haploidentical HCT for hematologic malignancy.
Bognàr T, Garcia-Rosa M, Lalmohamed A, Güngör T, Hauri-Hohl M, Prockop S, Oram L, Pai SY, Brooks J, Savic RM, Dvorak CC, Long-Boyle JR, Krajinovic M, Bittencourt H, Teyssier AC, Théorêt Y, Martinez C, Egberts TCG, Morales E, Slatter M, Cuvelier GDE, Chiesa R, Wynn RF, Coussons M, Cicalese MP, Ansari M, Long SE, Ebens CL, Lust H, Chaudhury S, Nath CE, Shaw PJ, Keogh SJ, van der Stoep MYEC, Bredius R, Lindemans CA, Boelens JJ, Bartelink IH
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity. Blood Adv. 2024 10 08; 8(19):5137-5145.
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity.
Bahakel H, Spieker AJ, Hayek H, Schuster JE, Hamdan L, Dulek DE, Kitko CL, Stopczynski T, Batarseh E, Haddadin Z, Stewart LS, Stahl A, Potter M, Rahman H, Amarin J, Kalams SA, Bocchini CE, Moulton EA, Coffin SE, Ardura MI, Wattier RL, Maron G, Grimley M, Paulsen G, Harrison CJ, Freedman J, Carpenter PA, Englund JA, Munoz FM, Danziger-Isakov L, Halasa N, Pediatric HCT Flu Study
Immunogenicity and Reactogenicity of High- or Standard-Dose Influenza Vaccine in a Second Consecutive Influenza Season. J Infect Dis. 2025 Feb 04; 231(1):e123-e131.
Immunogenicity and Reactogenicity of High- or Standard-Dose Influenza Vaccine in a Second Consecutive Influenza Season.
Miranda MA, Tsalatsanis A, Trotter JR, Arnold DE, Squire JD, Kidd S, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Grunebaum E, Sullivan KE, Newburger PE, Dinauer MC, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Morton FB, Leiding JW
High symptom burden in female X-linked chronic granulomatous disease carriers. Clin Immunol. 2024 Nov; 268:110364.
High symptom burden in female X-linked chronic granulomatous disease carriers.
Buso H, Adam E, Arkwright PD, Bhattad S, Hamidieh AA, Behfar M, Belot A, Benezech S, Chan AY, Crow YJ, Dvorak CC, Flinn AM, Kapoor U, Lankester A, Kobayashi M, Matsumura R, Mottaghipisheh H, Okada S, Ouachee M, Parvaneh N, Ramprakash S, Satwani P, Sharafian S, Triaille C, Wynn RF, Movahedi N, Ziaee V, Williams E, Slatter M, Gennery AR
Hematopoietic Stem Cell Transplantation for C1q Deficiency: A Study on Behalf of the EBMT Inborn Errors Working Party. J Clin Immunol. 2024 10 29; 45(1):35.
Hematopoietic Stem Cell Transplantation for C1q Deficiency: A Study on Behalf of the EBMT Inborn Errors Working Party.
Tolbert VP, Dvorak CC, Golden C, Vissa M, El-Haj N, Perwad F, Matthay KK, Vo KT
Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma. Biol Blood Marrow Transplant. 2019 10; 25(10):2031-2039.
Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma.
Slatnick LR, Hoogstra D, Fisher BT, Wolf J, Orgel E, Nessle CN, Patel PA, Miller TP, Wilkes J, Dupuis LL, Goode E, Jackson K, Willis DN, Elgarten C, Aftandilian C, Thompson J, Alexander S, Beauchemin MP, Belsky JA, Hess J, Prudowsky ZD, Guinipero T, Rossoff J, Demedis J, Walsh AM, Richards R, Choi DK, Dvorak CC, Esbenshade AJ
Prevention and Management of Infectious Complications in Pediatric Patients With Cancer: A Survey Assessment of Current Practices Across Children's Oncology Group Institutions. Pediatr Blood Cancer. 2025 Mar; 72(3):e31532.
Prevention and Management of Infectious Complications in Pediatric Patients With Cancer: A Survey Assessment of Current Practices Across Children's Oncology Group Institutions.
In ALD, I feel the need for speed. Blood. 2019 03 21; 133(12):1275-1277.
Transplant-Associated Thrombotic Microangiopathy in Pediatric Hematopoietic Cell Transplant Recipients: A Practical Approach to Diagnosis and Management. Front Pediatr. 2019; 7:133.
Transplant-Associated Thrombotic Microangiopathy in Pediatric Hematopoietic Cell Transplant Recipients: A Practical Approach to Diagnosis and Management.
Rolfes MC, Sriaroon P, Dávila Saldaña BJ, Dvorak CC, Chapdelaine H, Ferdman RM, Chen K, Jolles S, Patel NC, Kim YJ, Tarrant TK, Martelius T, Seppanen M, Joshi AY
Diagnostic microbiology and infectious disease
Chronic norovirus infection in primary immune deficiency disorders: an international case series. Diagn Microbiol Infect Dis. 2019 Jan; 93(1):69-73.
Chronic norovirus infection in primary immune deficiency disorders: an international case series.
Wang H, Jones AK, Dvorak CC, Huang L, Orchard P, Ivaturi V, Long-Boyle J
Population Pharmacokinetics of Clofarabine as Part of Pretransplantation Conditioning in Pediatric Subjects before Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant. 2019 08; 25(8):1603-1610.
Population Pharmacokinetics of Clofarabine as Part of Pretransplantation Conditioning in Pediatric Subjects before Hematopoietic Cell Transplantation.
Primary Immune Deficiency Treatment Consortium (PIDTC): A resource to drive scientific and clinical advances related to the treatment of inborn errors of immunity
Dvorak CC, Temple WC, Cisneros GS, Chu J, Winestone LE, Higham CS, Shimano KA, Kharbanda S, Avagyan S, Pauerstein P, Huang JN, Cheng G, Waters E, Winger BA, Cowan MJ, Long-Boyle JR
Younger children with nonmalignant disease have increased incidence of mixed myeloid chimerism after allogeneic hematopoietic cell transplantation with busulfan-based conditioning. Transplant Cell Ther. 2025 Apr; 31(4):259.e1-259.e15.
Younger children with nonmalignant disease have increased incidence of mixed myeloid chimerism after allogeneic hematopoietic cell transplantation with busulfan-based conditioning.
Dvorak CC, Cho S, Salinas Cisneros G, Higham CS, Chu J, Winestone LE, Temple WC, Kharbanda S, Shimano KA, Avagyan S, Pauerstein PT, Huang JN, Cheng G, Lalefar N, Aguayo-Hiraldo P, Keizer RJ, Pulsipher MA, Long-Boyle JR
High Melphalan Exposure Increases the Risk of Graft-Versus-Host Disease in Pediatric Patients Undergoing Alpha-Beta T-Cell Depleted Haploidentical Transplantation. Transplant Cell Ther. 2025 Jun; 31(6):384.e1-384.e15.
High Melphalan Exposure Increases the Risk of Graft-Versus-Host Disease in Pediatric Patients Undergoing Alpha-Beta T-Cell Depleted Haploidentical Transplantation.
University of California, San Diego, Molecular Biology
Vanderbilt University, School of Medicine
Ladas EJ, Collier W, Park H, Auletta JJ, Dvorak CC, August A, Esbenshade AJ, Bhatia M, Fisher BT, Levine JE, Pollock BH, Uhlemann AC, Verneris MR, Walters M, Yu L, Nieder M
A Randomized Clinical Trial Evaluating Lactiplantibacillus Plantarum for the Prevention of GI aGvHD: A Report From the Children's Oncology Group (ACCL1633). Transplant Cell Ther. 2025 Jul; 31(7):452.e1-452.e12.
A Randomized Clinical Trial Evaluating Lactiplantibacillus Plantarum for the Prevention of GI aGvHD: A Report From the Children's Oncology Group (ACCL1633).
Pearce EM, Evans E, Mayday MY, Reyes G, Simon MR, Blum J, Kim H, Mu J, Shaw PJ, Rowan CM, Auletta JJ, Martin PL, Hurley C, Kreml EM, Qayed M, Abdel-Azim H, Keating AK, Cuvelier GDE, Hume JR, Killinger JS, Godder K, Hanna R, Duncan CN, Quigg TC, Castillo P, Lalefar NR, Fitzgerald JC, Mahadeo KM, Satwani P, Moore TB, Hanisch B, Abdel-Mageed A, Davis DB, Hudspeth MP, Yanik GA, Pulsipher MA, Dvorak CCJL, Zinter MS
Integrating Pulmonary and Systemic Transcriptomic Profiles to Characterize Lung Injury after Pediatric Hematopoietic Stem Cell Transplant. medRxiv. 2025 Apr 01.
Integrating Pulmonary and Systemic Transcriptomic Profiles to Characterize Lung Injury after Pediatric Hematopoietic Stem Cell Transplant.
Hecht A, Meyer J, Chehab FF, White KL, Magruder K, Dvorak CC, Loh ML, Stieglitz E
Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia. Pediatr Blood Cancer. 2019 11; 66(11):e27948.
Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia.
Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium
Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2019 10; 39(7):653-667.
Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.
Pearce EM, Evans E, Mayday MY, Reyes G, Simon MR, Blum J, Kim H, Mu J, Shaw PJ, Rowan CM, Auletta JJ, Martin PL, Hurley C, Kreml EM, Qayed M, Abdel-Azim H, Keating AK, Cuvelier G, Hume JR, Killinger JS, Godder K, Hanna R, Duncan CN, Quigg TC, Castillo P, Lalefar NR, Fitzgerald JC, Mahadeo KM, Satwani P, Moore TB, Hanisch B, Abdel-Mageed A, Davis DB, Hudspeth MP, Yanik GA, Pulsipher MA, Dvorak CC, DeRisi JL, Zinter MS
Integrating pulmonary and systemic transcriptomes to characterize lung injury after pediatric hematopoietic stem cell transplant. JCI Insight. 2025 Sep 09; 10(17).
Integrating pulmonary and systemic transcriptomes to characterize lung injury after pediatric hematopoietic stem cell transplant.
Li C, Mathews V, Kim S, George B, Hebert K, Jiang H, Li C, Zhu Y, Keesler DA, Boelens JJ, Dvorak CC, Agarwal R, Auletta JJ, Goyal RK, Hanna R, Kasow K, Shenoy S, Smith AR, Walters MC, Eapen M
Related and unrelated donor transplantation for β-thalassemia major: results of an international survey. Blood Adv. 2019 09 10; 3(17):2562-2570.
Related and unrelated donor transplantation for β-thalassemia major: results of an international survey.
Lown EA, Banerjee A, Vittinghoff E, Dvorak CC, Hartogensis W, Melton A, Mangurian C, Hu H, Shear D, Adcock R, Morgan M, Golden C, Hecht FM
Acupressure to Reduce Treatment-Related Symptoms for Children With Cancer and Recipients of Hematopoietic Stem Cell Transplant: Protocol for a Randomized Controlled Trial. Glob Adv Health Med. 2019; 8:2164956119870444.
Acupressure to Reduce Treatment-Related Symptoms for Children With Cancer and Recipients of Hematopoietic Stem Cell Transplant: Protocol for a Randomized Controlled Trial.
Severe Combined Immunodeficiency in the Newborn Period. Neoreviews. 2025 Sep 01; 26(9):e600-e614.
Severe Combined Immunodeficiency in the Newborn Period.
Gadomski TE, Bolton M, Alfadhel M, Dvorak C, Ogunsakin OA, Nelson SL, Morava E
American journal of medical genetics. Part A
ALG13-CDG in a male with seizures, normal cognitive development, and normal transferrin isoelectric focusing. Am J Med Genet A. 2017 Oct; 173(10):2772-2775.
ALG13-CDG in a male with seizures, normal cognitive development, and normal transferrin isoelectric focusing.
van Asbeck E, Ramalingam A, Dvorak C, Chen TJ, Morava E
Duplication at Xq28 involving IKBKG is associated with progressive macrocephaly, recurrent infections, ectodermal dysplasia, benign tumors, and neuropathy. Clin Dysmorphol. 2014 Jul; 23(3):77-82.
Duplication at Xq28 involving IKBKG is associated with progressive macrocephaly, recurrent infections, ectodermal dysplasia, benign tumors, and neuropathy.
Chao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E
Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia. 2020 02; 34(2):662-666.
Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing.
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{"ucsfid":"024274755","first_name":"Janel","middle_name":"","last_name":"Long-Boyle","email":"janel.long-boyle@ucsf.edu","committees":[{"id":46,"title":"Academic Space for Clinicians Policy Task Force","abbreviation":"CPTF","category":"ucsf","type":"Task Force","webpage":"","service":[{"role":"Member","additional_role":"","start_date":"2018-02-01","end_date":"2018-06-30"}]},{"id":21,"title":"Pharmacy Faculty Council","abbreviation":"SOP FC","category":"ucsf","type":"Faculty Council","webpage":"https://senate.ucsf.edu/committee/sop+fc","service":[{"role":"Member","additional_role":"","start_date":"2014-09-01","end_date":"2016-08-31"}]},{"id":12,"title":"Research","abbreviation":"COR","category":"ucsf","type":"Standing Committee","webpage":"https://senate.ucsf.edu/committee/cor","service":[{"role":"Member","additional_role":"","start_date":"2016-09-01","end_date":"2019-08-31"}]}],"awards":[]}
Zinter MS, Logan BR, Fretham C, Sapru A, Abraham A, Aljurf MD, Arnold SD, Artz A, Auletta JJ, Chhabra S, Copelan E, Duncan C, Gale RP, Guinan E, Hematti P, Keating AK, Marks DI, Olsson R, Savani BN, Ustun C, Williams KM, Pasquini MC, Dvorak CC
Comprehensive Prognostication in Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results from Merging the Center for International Blood and Marrow Transplant Research (CIBMTR) and Virtual Pediatric Systems (VPS) Registries. Biol Blood Marrow Transplant. 2020 02; 26(2):333-342.
Comprehensive Prognostication in Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results from Merging the Center for International Blood and Marrow Transplant Research (CIBMTR) and Virtual Pediatric Systems (VPS) Registries.
Dvorak CC, Waters E, Temple WC, Salinas Cisneros G, Chu J, Winestone LE, Higham CS, Kharbanda S, Shimano KA, Avagyan S, Pauerstein P, Huang JN, Brooks JT, Long-Boyle JR
Fludarabine Pharmacokinetic Model-Based Dosing Minimizes the Risk of Graft Rejection in Pediatric Patients Undergoing Allogeneic Hematopoietic Cell Transplantation. Transplant Cell Ther. 2026 Apr; 32(4):476.e1-476.e14.
Fludarabine Pharmacokinetic Model-Based Dosing Minimizes the Risk of Graft Rejection in Pediatric Patients Undergoing Allogeneic Hematopoietic Cell Transplantation.
Dvorak CC, Satwani P, Stieglitz E, Cairo MS, Dang H, Pei Q, Gao Y, Wall D, Mazor T, Olshen AB, Parker JS, Kahwash S, Hirsch B, Raimondi S, Patel N, Skeens M, Cooper T, Mehta PA, Grupp SA, Loh ML
Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatr Blood Cancer. 2018 07; 65(7):e27034.
Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study.
Duncan CN, Brazauskas R, Huang J, Shaw BE, Majhail NS, Savani BN, Flowers MED, Battiwalla M, Beebe K, Dietz AC, Dvorak CC, Giller R, Jacobsohn DA, Kletzel M, Martin PL, Nemecek ER, Nuechterlein B, Talano JA, Pulsipher MA, Baker KS
Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2018 10; 53(10):1278-1287.
Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation.
Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY
B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation. Blood. 2018 06 28; 131(26):2967-2977.
B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation.
Smith MA, Hutchinson Z, Colglazier E, Parker C, Higham CS, Fineman JR, Zinter MS, Dvorak CC, Nawaytou HM
Early Echocardiographic and Serum Biomarkers Predict Thrombotic Microangiopathy, Endotheliopathy, and Survival After Pediatric Hematopoietic Stem Cell Transplant. Transplant Cell Ther. 2026 Mar; 32(3):306.e1-306.e13.
Early Echocardiographic and Serum Biomarkers Predict Thrombotic Microangiopathy, Endotheliopathy, and Survival After Pediatric Hematopoietic Stem Cell Transplant.
Rayes A, Logan BR, Liu X, Dara J, Buckley RH, Oved JH, Kapoor N, Kapadia M, Chandra S, Martinez CA, Bunin NJ, Chandrakasan S, Chen K, Bednarski JJ, Haines HL, Eissa H, Talano JM, Keller MD, Ebens CL, Chaudhury S, Shereck EB, Aquino VM, Knutsen AP, Alexander JL, Gillio AP, Chellapandian D, Shah AJ, Miller HK, Vander Lugt MT, Seroogy CM, Dorsey MJ, Mousallem T, Parrott RE, O'Reilly RJ, Aguayo-Hiraldo PI, Prockop SE, Dávila Saldaña BJ, Thakar MS, Burroughs LM, Torgerson TR, Leiding JW, Marsh RA, Griffith LM, Pulsipher MA, Kohn DB, Notarangelo L, Cowan MJ, Puck JM, Cuvelier GDE, Heimall J, Haddad E, Pai SY, Dvorak CC
Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC. Blood Adv. 2026 Feb 10; 10(3):887-900.
Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC.
Brumm VL, Kidd SA, Logan BR, Chunara F, Heimall J, Griffith LM, Kohn DB, Sanchez L, Bednarski JJ, Martinez C, Lugt MV, Kapoor N, Wright N, Spitzer B, Oved JH, Chandra S, Chellapandian D, Ebens CL, Petrovic A, Rayes A, Haines HL, Lust H, Schofield HT, Christopher L, Harris LL, Satter LF, Burroughs L, Dvorak CC, Haddad E, Leiding JW, Marsh RA, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Cowan MJ, Shah AJ
Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study. J Hum Immun. 2026 Jan 05; 2(1):e20250163.
Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study.
Kharbanda S, Cho E, Chen J, Zhang M, Yalcin K, Yesilipek A, Yabe H, Lindemans C, van Hasselt P, Alghasi A, Fong N, Bhattacharya R, Sheikh I, Guilcher GMT, Grimard P, Tomatsu S, Dvorak CC
Allogeneic Hematopoietic Cell Transplantation for Morquio A Syndrome: An International Retrospective Study. Transplant Cell Ther. 2026 Jan 28.
Allogeneic Hematopoietic Cell Transplantation for Morquio A Syndrome: An International Retrospective Study.
Arnold DE, Leiding JW, Logan B, Marsh RA, Griffith LM, Grunebaum E, Murguía-Favela L, Mallhi K, Chellapandian D, Deal CL, Lim SS, Prasad V, Heimall J, Chandrakasan S, Chen K, Yu LC, Seroogy CM, Gillio A, Bednarski JJ, Kapoor N, Moore TB, Cuvelier GDE, Touzot F, Rayes A, Ebens CL, Schaefer E, Bauchat A, Chopek A, Burroughs L, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Malech HL, Kang EM, Parikh S
Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report. Transplant Cell Ther. 2026 Jan; 32(1):89.e1-89.e10.
Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report.
Chandrakasan S, Westbrook A, Griffith LM, Hagin D, Iyengar S, Mangurian C, Scalchunes C, Sullivan KE, Zablocki A, Bakshi N, Sinha C, Burroughs LM, Chan AY, Dvorak CC, Haddad E, Heimall J, Kohn DB, Leiding JW, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Marsh RA, Torgerson T, Cowan MJ, Ochs HD, Parikh S
Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers. Clin Immunol. 2026 Feb; 283:110658.
Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers.
Hudda Z, Webster A, Anderson E, Badia P, Bagatell R, Bhar S, Bidgoli A, Boyd K, Chaudhury S, Cisneros GS, Craddock J, Davies SM, Desai AV, Drobatz L, Dvorak CC, Elkus H, Fabrizio VA, Foster JH, Fraint E, Freedman JL, Galvez J, Garcia M, Haight A, Higham C, Jodele S, Kamat A, Kaviany S, Lehmann L, Leung K, Lind KT, Llaurador G, Kapadia M, Khazal S, Massi K, Mormann M, Pater L, Rangarajan HG, Rotz S, Sabulski A, Sartain SE, Schoettler ML, Sokol E, Streby KA, Stein M, Verneris MR, Weiss BD, Yazdani S, Zheng D, Dandoy CE
Acute Toxicities and Early Outcomes of Tandem Autologous Stem Cell Transplantation in Pediatric High-Risk Neuroblastoma: A Multicenter Study. Transplant Cell Ther. 2026 Feb; 32(2):199.e1-199.e12.
Acute Toxicities and Early Outcomes of Tandem Autologous Stem Cell Transplantation in Pediatric High-Risk Neuroblastoma: A Multicenter Study.
Liberio N, Allbee-Johnson M, Ahn KW, Moskop A, Phelan R, Shaw BE, Page KM, Schultz KR, Phillips CL, Mehta PA, Qayed M, Sharma A, Lipsitt AE, Symons H, Bolaños-Meade J, Kharbanda S, Dvorak CC, Kapoor N, Doherty EE, Chavan RS, Broglie L
Optimizing Haploidentical Donor Selection for Pediatric Hematopoietic Cell Transplant. J Clin Oncol. 2026 Feb 25; JCO2501591.
Optimizing Haploidentical Donor Selection for Pediatric Hematopoietic Cell Transplant.
Evans EM, Mayday MY, Pearce EM, Iwanaga K, Ly NP, Church GD, Reyes G, Simon MR, Blum J, Kim H, Mu J, Baez-Maidana J, Auletta JJ, Shaw PJ, Kreml EM, Martin PL, Duncan CN, Rowan CM, Godder K, Hurley C, Cuvelier GDE, Qayed M, Abdel-Azim H, Keating AK, Fitzgerald JC, Hanna R, Killinger JS, Hume JR, Quigg TC, Castillo P, Satwani P, Moore TB, Dvorak CC, Zinter MS
Upper and Lower Respiratory Tract Compartmentalization in Pediatric Stem Cell Transplantation. medRxiv. 2025 Oct 17.
Upper and Lower Respiratory Tract Compartmentalization in Pediatric Stem Cell Transplantation.
[{"Id":"NCT03538899","Title":"Autologous Gene Therapy for Artemis-Deficient SCID","StartDate":"2018-05-31T00:00:00","EstimatedCompletionDate":"2038-06-30T00:00:00","CompletionDate":null,"Conditions":"Immunodeficiency,Severe Combined Immunodeficiency","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT03538899"},{"Id":"NCT01346150","Title":"Patients Treated for SCID (1968-Present)","StartDate":"2011-05-15T00:00:00","EstimatedCompletionDate":"2023-08-31T00:00:00","CompletionDate":null,"Conditions":"Severe Combined Immunodeficiency,X-Linked Severe Combined Immunodeficiency,Reticular Dysgenesis,ADA Deficiency,Immunodeficiency","Status":"Unknown status","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT01346150"},{"Id":"NCT01186913","Title":"Natural History Study of SCID Disorders","StartDate":"2010-09-02T00:00:00","EstimatedCompletionDate":"2028-09-30T00:00:00","CompletionDate":null,"Conditions":"Immunodeficiency,Severe Combined Immunodeficiency,Reticular Dysgenesis,X-Linked Severe Combined Immunodeficiency,ADA Deficiency","Status":"Enrolling by invitation","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT01186913"},{"Id":"NCT00295971","Title":"Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease","StartDate":"2005-04-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2011-12-31T00:00:00","Conditions":"Congenital Amegakaryocytic Thrombocytopenia,Leukemia,Myelodysplastic Syndrome,Congenital Neutropenia,Bone Disease,Bone Marrow Transplant,Immunodeficiency,Stem Cell Transplant,Transplants,Anemia","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT00295971"},{"Id":"NCT00301834","Title":"Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders","StartDate":"2005-01-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2011-09-30T00:00:00","Conditions":"Congenital Amegakaryocytic Thrombocytopenia,Anemia,Leukemia,Myelodysplastic Syndrome,Congenital Neutropenia,Bone Marrow Transplant,Hematologic Disease,Stem Cell Transplant,Transplants","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT00301834"},{"Id":"NCT00305708","Title":"Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission","StartDate":"2000-08-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2004-07-31T00:00:00","Conditions":"Congenital Amegakaryocytic Thrombocytopenia,Anemia,Fanconi Anemia,Leukemia,Congenital Neutropenia,Thrombocytopenia,Acute Myeloid Leukemia,Bone Marrow Transplant,Chronic Myeloid Leukemia,Stem Cell Transplant,Transplants","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT00305708"}]
[{"Id":"NCT01512888","Title":"Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants","StartDate":"2016-08-17T00:00:00","EstimatedCompletionDate":"2034-08-31T00:00:00","CompletionDate":null,"Conditions":"Immunodeficiency,Severe Combined Immunodeficiency,X-Linked Severe Combined Immunodeficiency","Status":"Suspended","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT01512888"},{"Id":"NCT02082353","Title":"Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995","StartDate":"2014-06-01T00:00:00","EstimatedCompletionDate":"2021-11-30T00:00:00","CompletionDate":null,"Conditions":"Chronic Granulomatous Disease","Status":"Unknown status","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02082353"}]
[{"Id":"NCT05267821","Title":"Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS","StartDate":"2022-06-14T00:00:00","EstimatedCompletionDate":"2028-08-31T00:00:00","CompletionDate":null,"Conditions":"Multiple Organ Dysfunction Syndrome","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT05267821"},{"Id":"NCT05266001","Title":"GM-CSF for Reversal of Immunoparalysis in Pediatric Sepsis-induced MODS","StartDate":"2022-06-14T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2024-12-01T00:00:00","Conditions":"Multiple Organ Dysfunction Syndrome","Status":"Terminated","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT05266001"},{"Id":"NCT05026749","Title":"Azithromycin Treatment for Respiratory Syncytial Virus-induced Respiratory Failure in Children","StartDate":"2022-02-27T00:00:00","EstimatedCompletionDate":"2028-07-31T00:00:00","CompletionDate":null,"Conditions":"RSV","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT05026749"},{"Id":"NCT04098445","Title":"TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients","StartDate":"2021-09-08T00:00:00","EstimatedCompletionDate":"2036-09-30T00:00:00","CompletionDate":null,"Conditions":"Bone Marrow Transplant,Hematopoietic Cell Transplantation,Stem Cell Transplant,Transplants,Diffuse Alveolar Hemorrhage,Thrombotic Microangiopathies,Interstitial Lung Disease,Bronchiolitis Obliterans","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT04098445"},{"Id":"NCT03401398","Title":"Stress Hydrocortisone In Pediatric Septic Shock","StartDate":"2019-03-11T00:00:00","EstimatedCompletionDate":"2026-12-31T00:00:00","CompletionDate":null,"Conditions":"Septic Shock,Sepsis","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT03401398"},{"Id":"NCT02926612","Title":"Pathogen Identification in Pediatric Hematopoietic Stem Cell Transplant Patients With Suspected Lower Respiratory Tract Infection","StartDate":"2016-06-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":null,"Conditions":"Lower Respiratory Tract Infection,Bone Marrow Transplant,Hematopoietic Cell Transplantation,Stem Cell Transplant,Transplants","Status":"Unknown status","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02926612"}]
[{"Id":"NCT01351545","Title":"A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)","StartDate":"2011-10-01T00:00:00","EstimatedCompletionDate":"2041-10-31T00:00:00","CompletionDate":null,"Conditions":"Hematologic Malignancy,Disorders of Metabolism,Abnormalities of Platelets,Histiocytic Disorders,Acute Myeloid Leukemia,Leukemia,Acute Lymphoblastic Leukemia,Chronic Myeloid Leukemia,Hodgkin's Lymphoma,Lymphoma,Non-Hodgkin Lymphoma,Multiple Myeloma,Abnormalities of Erythrocyte Differentiation or Function,Autoimmune Disease,Anemia,Hematologic Neoplasms","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT01351545"}]
[{"Id":"NCT03266653","Title":"EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection","StartDate":"2020-07-07T00:00:00","EstimatedCompletionDate":"2027-12-31T00:00:00","CompletionDate":null,"Conditions":"Epstein Barr Virus,Immunodeficiency,Primary Immunodeficiency Diseases","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT03266653"},{"Id":"NCT04337515","Title":"A Feasibility Study Using CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem Cell Transplant","StartDate":"2019-12-19T00:00:00","EstimatedCompletionDate":"2035-01-01T00:00:00","CompletionDate":null,"Conditions":"Graft Versus Host Disease,Bone Marrow Transplant,Stem Cell Transplant,Transplants","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT04337515"},{"Id":"NCT02963064","Title":"JSP191 Antibody Targeting Conditioning in SCID Patients","StartDate":"2017-03-20T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2025-07-07T00:00:00","Conditions":"Severe Combined Immunodeficiency,Hematopoietic Cell Transplantation","Status":"Terminated","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02963064"},{"Id":"NCT02458235","Title":"Donor Lymphocyte Infusion With Azacitidine to Prevent Hematologic Malignancy Relapse After Stem Cell Transplantation","StartDate":"2015-06-02T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2019-03-15T00:00:00","Conditions":"Acute Myeloid Leukemia,Leukemia,Juvenile Myelomonocytic Leukemia,Myelodysplastic Syndrome,Bone Marrow Transplant,Hematologic Malignancy,Stem Cell Transplant,Lymphoma","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02458235"},{"Id":"NCT01596699","Title":"Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation","StartDate":"2012-05-24T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2019-06-30T00:00:00","Conditions":"Myeloid Malignancy,Bone Disease,Immunodeficiency,Metabolic Disease,Immune Dysregulation,Allogeneic Hematopoietic Cell Transplantation,Hematopoietic Cell Transplantation","Status":"Terminated","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT01596699"},{"Id":"NCT01200017","Title":"Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant","StartDate":"2010-09-09T00:00:00","EstimatedCompletionDate":null,"CompletionDate":null,"Conditions":"Acute Lymphoblastic Leukemia,Leukemia,Acute Myeloid Leukemia,Chronic Myeloid Leukemia,Myelodysplastic Syndrome,Lymphoma,Bone Disease,Hemoglobinopathy,Immunodeficiency,Osteopetrosis,Cytopenia,Leukocyte Disorders,Anemia,Bone Marrow Transplant,Stem Cell Transplant,Transplants,Graft Versus Host Disease,Hematologic Disease,Neoplasms,Lymphoproliferative Disorders","Status":"No longer available","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT01200017"},{"Id":"NCT01186913","Title":"Natural History Study of SCID Disorders","StartDate":"2010-09-02T00:00:00","EstimatedCompletionDate":"2028-09-30T00:00:00","CompletionDate":null,"Conditions":"Immunodeficiency,Severe Combined Immunodeficiency,Reticular Dysgenesis,X-Linked Severe Combined Immunodeficiency,ADA Deficiency","Status":"Enrolling by invitation","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.ucsf.edu/trial/NCT01186913"},{"Id":"NCT01182675","Title":"Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim","StartDate":"2010-08-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2013-09-30T00:00:00","Conditions":"Immunodeficiency,Severe Combined Immunodeficiency,Bone Marrow Transplant,Hematopoietic Cell Transplantation,Stem Cell Transplant","Status":"Terminated","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT01182675"}]
[{"Id":"NCT06876363","Title":"Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease","StartDate":"2025-08-05T00:00:00","EstimatedCompletionDate":"2027-12-31T00:00:00","CompletionDate":null,"Conditions":"Chronic Granulomatous Disease","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://ucla.clinicaltrials.researcherprofiles.org/trial/NCT06876363"},{"Id":"NCT06282432","Title":"Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)","StartDate":"2022-03-09T00:00:00","EstimatedCompletionDate":"2036-10-04T00:00:00","CompletionDate":null,"Conditions":"Leukocyte Adhesion Deficiency","Status":"Active, not recruiting","IsMultiSite":false,"SourceUrl":"https://ucla.clinicaltrials.researcherprofiles.org/trial/NCT06282432"},{"Id":"NCT04525352","Title":"A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis","StartDate":"2020-11-19T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2021-05-21T00:00:00","Conditions":"Infantile Malignant Osteopetrosis,Bone Disease","Status":"Terminated","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT04525352"},{"Id":"NCT04140539","Title":"A Clinical Study to Enable Process Validation of Commercial Grade OTL-101","StartDate":"2019-10-15T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2021-08-30T00:00:00","Conditions":"ADA Deficiency,Immunodeficiency,Severe Combined Immunodeficiency","Status":"Withdrawn","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT04140539"},{"Id":"NCT04049084","Title":"An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID","StartDate":"2019-09-26T00:00:00","EstimatedCompletionDate":"2040-08-31T00:00:00","CompletionDate":null,"Conditions":"ADA Deficiency,Severe Combined Immunodeficiency,Immunodeficiency","Status":"Enrolling by invitation","IsMultiSite":false,"SourceUrl":"https://ucla.clinicaltrials.researcherprofiles.org/trial/NCT04049084"},{"Id":"NCT03812263","Title":"A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I","StartDate":"2019-08-30T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2023-09-12T00:00:00","Conditions":"Leukocyte Adhesion Defect - Type I","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT03812263"},{"Id":"NCT02999984","Title":"Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID","StartDate":"2016-12-16T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2019-09-26T00:00:00","Conditions":"ADA Deficiency,Immunodeficiency,Severe Combined Immunodeficiency","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02999984"},{"Id":"NCT02234934","Title":"Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease","StartDate":"2015-10-29T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2024-12-01T00:00:00","Conditions":"Granulomatous Disease, Chronic, X-linked,Chronic Granulomatous Disease","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02234934"},{"Id":"NCT02247843","Title":"Stem Cell Gene Therapy for Sickle Cell Disease","StartDate":"2014-12-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2025-09-24T00:00:00","Conditions":"Sickle Cell Anemia,Bone Marrow Transplant,Stem Cell Transplant,Anemia","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02247843"},{"Id":"NCT02082353","Title":"Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995","StartDate":"2014-06-01T00:00:00","EstimatedCompletionDate":"2021-11-30T00:00:00","CompletionDate":null,"Conditions":"Chronic Granulomatous Disease","Status":"Unknown status","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT02082353"},{"Id":"NCT01852071","Title":"Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene","StartDate":"2013-08-02T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2018-08-27T00:00:00","Conditions":"Severe Combined Immunodeficiency,ADA Deficiency,Immunodeficiency","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT01852071"},{"Id":"NCT00794508","Title":"MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID","StartDate":"2008-11-01T00:00:00","EstimatedCompletionDate":null,"CompletionDate":"2015-01-31T00:00:00","Conditions":"Immunodeficiency,Severe Combined Immunodeficiency,ADA Deficiency","Status":"Completed","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.gov/ct2/show/NCT00794508"}]
[{"Id":"NCT05905328","Title":"Study of CTO1681 for the Prevention and Treatment of CRS in DLBCL Patients Receiving CAR T-Cell Therapy","StartDate":"2023-12-28T00:00:00","EstimatedCompletionDate":"2027-06-30T00:00:00","CompletionDate":null,"Conditions":"Cytokine Release Syndrome,CAR T-Cell Therapy,Diffuse Large B-Cell Lymphoma,Lymphoma,Non-Hodgkin Lymphoma","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.icts.uci.edu/trial/NCT05905328"},{"Id":"NCT06028828","Title":"Risk-ADAPTed Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation","StartDate":"2023-09-11T00:00:00","EstimatedCompletionDate":"2027-09-30T00:00:00","CompletionDate":null,"Conditions":"Allogeneic Hematopoietic Cell Transplantation,Bone Marrow Transplant,Hematopoietic Cell Transplantation,Stem Cell Transplant,Hematologic Malignancy,Hematologic Neoplasms","Status":"Recruiting","IsMultiSite":false,"SourceUrl":"https://clinicaltrials.icts.uci.edu/trial/NCT06028828"}]
Bejanyan N, Kim S, Hebert KM, Kekre N, Abdel-Azim H, Ahmed I, Aljurf M, Badawy SM, Beitinjaneh A, Boelens JJ, Diaz MA, Dvorak CC, Gadalla S, Gajewski J, Gale RP, Ganguly S, Gennery AR, George B, Gergis U, Gómez-Almaguer D, Vicent MG, Hashem H, Kamble RT, Kasow KA, Lazarus HM, Mathews V, Orchard PJ, Pulsipher M, Ringden O, Schultz K, Teira P, Woolfrey AE, Saldaña BD, Savani B, Winiarski J, Yared J, Weisdorf DJ, Antin JH, Eapen M
Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Adv. 2019 10 22; 3(20):3123-3131.
Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia.
Fisher BT, Zaoutis T, Dvorak CC, Nieder M, Zerr D, Wingard JR, Callahan C, Villaluna D, Chen L, Dang H, Esbenshade AJ, Alexander S, Wiley JM, Sung L
Effect of Caspofungin vs Fluconazole Prophylaxis on Invasive Fungal Disease Among Children and Young Adults With Acute Myeloid Leukemia: A Randomized Clinical Trial. JAMA. 2019 11 05; 322(17):1673-1681.
Effect of Caspofungin vs Fluconazole Prophylaxis on Invasive Fungal Disease Among Children and Young Adults With Acute Myeloid Leukemia: A Randomized Clinical Trial.
Friend BD, Bailey-Olson M, Melton A, Shimano KA, Kharbanda S, Higham C, Winestone LE, Huang J, Stieglitz E, Dvorak CC
The impact of total body irradiation-based regimens on outcomes in children and young adults with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation. Pediatr Blood Cancer. 2020 02; 67(2):e28079.
The impact of total body irradiation-based regimens on outcomes in children and young adults with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation.
Seghezzo SP, Dvorak CC, Cowan MJ, Puck JM, Dorsey MJ
Extended Follow-up After Hematopoietic Cell Transplantation for IκBα Deficiency with Disseminated Mycobacterium avium Infection. J Clin Immunol. 2020 01; 40(1):248-250.
Extended Follow-up After Hematopoietic Cell Transplantation for IκBα Deficiency with Disseminated Mycobacterium avium Infection.
Dandoy CE, Kim S, Chen M, Ahn KW, Ardura MI, Brown V, Chhabra S, Diaz MA, Dvorak C, Farhadfar N, Flagg A, Ganguly S, Hale GA, Hashmi SK, Hematti P, Martino R, Nishihori T, Nusrat R, Olsson RF, Rotz SJ, Sung AD, Perales MA, Lindemans CA, Komanduri KV, Riches ML
Incidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury-Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant. JAMA Netw Open. 2020 01 03; 3(1):e1918668.
Incidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury-Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant.
TA-TMA: state of the art for diagnosis and treatment. Blood Adv. 2020 01 14; 4(1):217.
TA-TMA: state of the art for diagnosis and treatment.