Children with sickle cell disease (SCD) require specific perioperative care, and clinical practic... more Children with sickle cell disease (SCD) require specific perioperative care, and clinical practice in this area remains poorly defined. We aimed to conduct a systematic, PRISMA-based review of the literature, available clinical guidelines and practice recommendations. We also aimed to extract any valuable information for the "best of available-evidence"-based prevention of perioperative adverse events in children with SCD, and highlight the most urgent priorities in clinical research. As data sources, US
Methods This retrospective analysis included a cohort of patients < 30 years (y) on chronic HD tr... more Methods This retrospective analysis included a cohort of patients < 30 years (y) on chronic HD treatment since childhood, having received thrice-weekly HD between 2004 and 2016 in outpatient DaVita dialysis centers. Survival while on HD (death from any cause) was investigated using Kaplan-Meier analysis stratified by age at start of HD (0-2, >2-6, >6-12, and >12-18 y), and three mean delivered dialysis dose levels (spKt/V < 1.4, 1.4-1.6, >1.6). Survival curves between subgroups were compared using the Log-rank test. Results 1773 patients were included in the analysis, among n=34 having started HD at age of 0-2y, n=57 at >2-6y, n=244 at >6-12y, and n=1438 at >12-18y. Median followup on HD ranged between 1.5 (>2-6y) to 4.7 years (>6-12y) with maximal follow-up of 23 years. Death while on HD occurred in 1/34, 6/57, 26/244, and 101/1438 patients during recorded follow-up (p=0.075, n.s.). Patients with mean spKt/V < 1.4 had lower survival on HD than those with spKt/V >1.4-1.6 (p=0.019) and those with spKt/V >1.6 (p=0.035), with 10-year survival estimated to 75% (65.2-86.2%) versus 84.5% (78.5-90.9%) and 85.0% (80.8-89.5%), respectively. Conclusions This is the first study to report long term survival and its relationship with delivered HD dose in patients starting HD in childhood. Our results support targeting spKt/V (urea)>1.4 in children on chronic HD treatment.
Trilateral retinoblastoma (TRb) is a well-known syndrome associating hereditary retinoblastoma (R... more Trilateral retinoblastoma (TRb) is a well-known syndrome associating hereditary retinoblastoma (Rb) with an intracranial neuroblastic tumor arising usually in the pineal region, rarely at the suprasellar or parasellar site. It develops in most cases after diagnosis of Rb. The outcome is usually fatal because of secondary spinal dissemination. Pineal cysts have recently been reported as a benign variant of TRb. We report the unusual presentation of a TRb in a 12-month-old boy with extensive bilateral Rb, a voluminous suprasellar tumor, pineal cyst, and leptomeningeal disease. The special features of this &amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;quot;quadrilateral&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;quot; Rb are discussed.
This study evaluates our institution&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;... more This study evaluates our institution&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;#39;s target trough cyclosporine (CSA) concentrations as predictors of severe acute graft versus host disease (aGvHD) in children receiving either matched related or unrelated hematopoietic stem cell transplantation (HSCT). The outcomes of 87 consecutive children who underwent allogeneic HSCT and received CSA and methotrexate as prophylaxis against aGvHD between October 1, 1999 and September 30, 2002 were retrospectively evaluated. The proportion of time that each patient maintained a whole blood CSA concentration within or above the initial target range (105-155 ng/mL or 155-210 ng/mL) was calculated for each of the following time periods: in each week after HSCT from day 0 to +28; in the week preceding engraftment; and in the week preceding the onset of aGvHD. Patients were prospectively evaluated twice weekly for the presence and severity of aGvHD by senior attending physicians. The relationship between potential predictors and the development of severe aGvHD was examined using univariate logistic regression. The main variables of interest were the proportion of time that therapeutic or supratherapeutic CSA concentrations were maintained; median CSA concentrations; the number of methotrexate doses received; and the use of folinic acid rescue. Mean follow-up time was 3.0+/-1.9 years among children who survived beyond day +100. Three variables were significantly associated with the development of severe aGvHD on univariate analysis: initial CSA target concentration [odds ratio (OR), 0.24; P=0.03], proportion of time the target CSA concentration was achieved during the second week after transplant (OR, 0.16; P=0.02), and proportion of time the target CSA concentration was achieved during the week before engraftment (OR, 0.22; P=0.0489). Multivariable analysis demonstrated an inverse relationship between the median CSA concentration during the week before engraftment and the development of severe aGvHD (OR, 0.99; P=0.045). These results suggest that achievement of our CSA target concentrations is important to aGvHD outcomes.
Sevelamer is a phosphate-binder used effectively for the treatment of hyperphosphatemia in patien... more Sevelamer is a phosphate-binder used effectively for the treatment of hyperphosphatemia in patients treated with dialysis. To describe the safety of sevelamer in children with hyperphosphatemia secondary to tumor lysis syndrome and the serum phosphate concentrations observed following its administration. A retrospective chart review of all children with leukemia/lymphoma diagnosed between November 2002 and April 2004 who received sevelamer during their initial admission was conducted. We monitored the effects of sevelamer on serum phosphate concentration, calcium/phosphate product and renal function at hours 24, 48, and 72 from sevelamer initiation. Thirteen patients received sevelamer during the study period. Their median age was 13 years (range 2.7-17.9) and eight were boys. Nine children had acute lymphoblastic leukemia, one had acute myeloid leukemia and 3 had non-Hodgkin&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;#39;s lymphoma. The most frequently used dose of sevelamer was 400 mg orally twice daily. The median duration of sevelamer therapy was 2 days (range 1-7). Two children were excluded from the efficacy analysis due to concurrent use of dialysis. Mean serum phosphate levels decreased after sevelamer administration, in eleven patients, from a baseline 2.2 mmol/L +/- 0.4 (95% CI, 1.7-3.1) to 1.1 mmol/L +/- 0.2 at hour 72 (95%CI, 0.6-1.5). The only toxicity attributed to sevelamer was mild vomiting in three patients. Sevelamer appears to be effective and tolerable for the treatment of hyperphosphatemia associated with tumor lysis syndrome.
Children with sickle cell disease (SCD) require specific perioperative care, and clinical practic... more Children with sickle cell disease (SCD) require specific perioperative care, and clinical practice in this area remains poorly defined. We aimed to conduct a systematic, PRISMA-based review of the literature, available clinical guidelines and practice recommendations. We also aimed to extract any valuable information for the "best of available-evidence"-based prevention of perioperative adverse events in children with SCD, and highlight the most urgent priorities in clinical research. As data sources, US
Methods This retrospective analysis included a cohort of patients < 30 years (y) on chronic HD tr... more Methods This retrospective analysis included a cohort of patients < 30 years (y) on chronic HD treatment since childhood, having received thrice-weekly HD between 2004 and 2016 in outpatient DaVita dialysis centers. Survival while on HD (death from any cause) was investigated using Kaplan-Meier analysis stratified by age at start of HD (0-2, >2-6, >6-12, and >12-18 y), and three mean delivered dialysis dose levels (spKt/V < 1.4, 1.4-1.6, >1.6). Survival curves between subgroups were compared using the Log-rank test. Results 1773 patients were included in the analysis, among n=34 having started HD at age of 0-2y, n=57 at >2-6y, n=244 at >6-12y, and n=1438 at >12-18y. Median followup on HD ranged between 1.5 (>2-6y) to 4.7 years (>6-12y) with maximal follow-up of 23 years. Death while on HD occurred in 1/34, 6/57, 26/244, and 101/1438 patients during recorded follow-up (p=0.075, n.s.). Patients with mean spKt/V < 1.4 had lower survival on HD than those with spKt/V >1.4-1.6 (p=0.019) and those with spKt/V >1.6 (p=0.035), with 10-year survival estimated to 75% (65.2-86.2%) versus 84.5% (78.5-90.9%) and 85.0% (80.8-89.5%), respectively. Conclusions This is the first study to report long term survival and its relationship with delivered HD dose in patients starting HD in childhood. Our results support targeting spKt/V (urea)>1.4 in children on chronic HD treatment.
Trilateral retinoblastoma (TRb) is a well-known syndrome associating hereditary retinoblastoma (R... more Trilateral retinoblastoma (TRb) is a well-known syndrome associating hereditary retinoblastoma (Rb) with an intracranial neuroblastic tumor arising usually in the pineal region, rarely at the suprasellar or parasellar site. It develops in most cases after diagnosis of Rb. The outcome is usually fatal because of secondary spinal dissemination. Pineal cysts have recently been reported as a benign variant of TRb. We report the unusual presentation of a TRb in a 12-month-old boy with extensive bilateral Rb, a voluminous suprasellar tumor, pineal cyst, and leptomeningeal disease. The special features of this &amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;quot;quadrilateral&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;quot; Rb are discussed.
This study evaluates our institution&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;... more This study evaluates our institution&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;#39;s target trough cyclosporine (CSA) concentrations as predictors of severe acute graft versus host disease (aGvHD) in children receiving either matched related or unrelated hematopoietic stem cell transplantation (HSCT). The outcomes of 87 consecutive children who underwent allogeneic HSCT and received CSA and methotrexate as prophylaxis against aGvHD between October 1, 1999 and September 30, 2002 were retrospectively evaluated. The proportion of time that each patient maintained a whole blood CSA concentration within or above the initial target range (105-155 ng/mL or 155-210 ng/mL) was calculated for each of the following time periods: in each week after HSCT from day 0 to +28; in the week preceding engraftment; and in the week preceding the onset of aGvHD. Patients were prospectively evaluated twice weekly for the presence and severity of aGvHD by senior attending physicians. The relationship between potential predictors and the development of severe aGvHD was examined using univariate logistic regression. The main variables of interest were the proportion of time that therapeutic or supratherapeutic CSA concentrations were maintained; median CSA concentrations; the number of methotrexate doses received; and the use of folinic acid rescue. Mean follow-up time was 3.0+/-1.9 years among children who survived beyond day +100. Three variables were significantly associated with the development of severe aGvHD on univariate analysis: initial CSA target concentration [odds ratio (OR), 0.24; P=0.03], proportion of time the target CSA concentration was achieved during the second week after transplant (OR, 0.16; P=0.02), and proportion of time the target CSA concentration was achieved during the week before engraftment (OR, 0.22; P=0.0489). Multivariable analysis demonstrated an inverse relationship between the median CSA concentration during the week before engraftment and the development of severe aGvHD (OR, 0.99; P=0.045). These results suggest that achievement of our CSA target concentrations is important to aGvHD outcomes.
Sevelamer is a phosphate-binder used effectively for the treatment of hyperphosphatemia in patien... more Sevelamer is a phosphate-binder used effectively for the treatment of hyperphosphatemia in patients treated with dialysis. To describe the safety of sevelamer in children with hyperphosphatemia secondary to tumor lysis syndrome and the serum phosphate concentrations observed following its administration. A retrospective chart review of all children with leukemia/lymphoma diagnosed between November 2002 and April 2004 who received sevelamer during their initial admission was conducted. We monitored the effects of sevelamer on serum phosphate concentration, calcium/phosphate product and renal function at hours 24, 48, and 72 from sevelamer initiation. Thirteen patients received sevelamer during the study period. Their median age was 13 years (range 2.7-17.9) and eight were boys. Nine children had acute lymphoblastic leukemia, one had acute myeloid leukemia and 3 had non-Hodgkin&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;#39;s lymphoma. The most frequently used dose of sevelamer was 400 mg orally twice daily. The median duration of sevelamer therapy was 2 days (range 1-7). Two children were excluded from the efficacy analysis due to concurrent use of dialysis. Mean serum phosphate levels decreased after sevelamer administration, in eleven patients, from a baseline 2.2 mmol/L +/- 0.4 (95% CI, 1.7-3.1) to 1.1 mmol/L +/- 0.2 at hour 72 (95%CI, 0.6-1.5). The only toxicity attributed to sevelamer was mild vomiting in three patients. Sevelamer appears to be effective and tolerable for the treatment of hyperphosphatemia associated with tumor lysis syndrome.
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