Firezer Haregu, R. Jerome Dixon, Michael Porter, Michael McCulloch
BackgroundOrgan procurement organizations (OPOs) are responsible for the medical management of organ donors. Given the variability in pediatric donor heart utilization among OPOs, we examined factors that may explain this variability, including differences in donor medical management, organ quality, and candidate factors.MethodsThe Organ Procurement and Transplant Network database was queried for pediatric (<18 years) heart donors and candidates receiving pediatric donor heart offers from 2010 to 2019. OPOs were stratified by pediatric donor heart utilization rate, and the top and bottom quintiles were compared based on donor management strategies and outcomes. A machine learning algorithm, combining 11 OPO, donor, candidate, and offer variables, was used to determine factors most predictive of whether a heart offer is accepted.ResultsThere was no clinically significant difference between the top and bottom quintile OPOs in baseline donor characteristics, distance between donor and listing center, management strategies, or organ quality. Machine learning modeling suggested neither OPO donor management nor cardiac function is the primary driver of whether an organ is accepted. Instead, number of prior donor offer refusals and individual listing center receiving the offer were two of the most predictive variables of organ acceptance.ConclusionsOPO clinical practice variation does not seem to account for the discrepancy in pediatric donor heart utilization rates among OPOs. Listing center acceptance practice and prior number of donor refusals seem to be the important drivers of heart utilization and may at least partially account for the variation in OPO heart utilization rates given the regional association between OPOs and listing centers.
{"title":"Pediatric donor heart utilization variability among organ procurement organizations","authors":"Firezer Haregu, R. Jerome Dixon, Michael Porter, Michael McCulloch","doi":"10.1111/petr.14747","DOIUrl":"https://doi.org/10.1111/petr.14747","url":null,"abstract":"BackgroundOrgan procurement organizations (OPOs) are responsible for the medical management of organ donors. Given the variability in pediatric donor heart utilization among OPOs, we examined factors that may explain this variability, including differences in donor medical management, organ quality, and candidate factors.MethodsThe Organ Procurement and Transplant Network database was queried for pediatric (<18 years) heart donors and candidates receiving pediatric donor heart offers from 2010 to 2019. OPOs were stratified by pediatric donor heart utilization rate, and the top and bottom quintiles were compared based on donor management strategies and outcomes. A machine learning algorithm, combining 11 OPO, donor, candidate, and offer variables, was used to determine factors most predictive of whether a heart offer is accepted.ResultsThere was no clinically significant difference between the top and bottom quintile OPOs in baseline donor characteristics, distance between donor and listing center, management strategies, or organ quality. Machine learning modeling suggested neither OPO donor management nor cardiac function is the primary driver of whether an organ is accepted. Instead, number of prior donor offer refusals and individual listing center receiving the offer were two of the most predictive variables of organ acceptance.ConclusionsOPO clinical practice variation does not seem to account for the discrepancy in pediatric donor heart utilization rates among OPOs. Listing center acceptance practice and prior number of donor refusals seem to be the important drivers of heart utilization and may at least partially account for the variation in OPO heart utilization rates given the regional association between OPOs and listing centers.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jin Jieyang, Liao Mei, Liu Kunying, Wu Tao, Jin Ling, Yang Yang, Yi Shuhong, Lv Yan, Ren Jie
BackgroundVenous complications after pediatric liver transplantation seriously affect the survival rate of patients and grafts. At present, the diagnostic indicators have not been unified. Venous complications may cause portal hypertension, which may lead to splenomegaly and splenic vein dilatation. Therefore, the changes in spleen may be closely related to the venous complications. The purpose of this study was to explore the relationship between ultrasonic splenic parameters and venous complications and to study whether these splenic parameters can be used for the diagnosis of venous complications.MethodsWe retrospectively included pediatric patients who underwent liver transplantation and collected ultrasonic spleen parameters before, and then 1–3 days, 1–3 weeks, 1–3 months, and 4–12 months after liver transplantation. We observed whether there were portal vein or hepatic vein complications within 1 year after liver transplantation.ResultsAmong 109 pediatric patients after liver transplantation included in our study, 11 of them suffered from portal vein complications and nine hepatic vein complications. Spleen transverse diameter, spleen longitudinal diameter, spleen portal vein diameter, spleen index, spleen transverse diameter ratio, spleen longitudinal diameter ratio, and spleen index ratio were independent risk factors of venous complications. The accuracy of spleen transverse diameter (AUROC: 0.73), spleen index (AUROC: 0.70), spleen transverse diameter ratio (AUROC: 0.71), and spleen index ratio (AUROC: 0.72) in predicting venous complications were higher than other ones.ConclusionsUltrasonic examination is a common follow‐up method for pediatric patients after liver transplantation and the application of ultrasonic spleen parameters may be helpful to monitor venous complications.
{"title":"A new method to predict venous complications in pediatric liver transplantation: Evaluation of splenic parameters by ultrasonography","authors":"Jin Jieyang, Liao Mei, Liu Kunying, Wu Tao, Jin Ling, Yang Yang, Yi Shuhong, Lv Yan, Ren Jie","doi":"10.1111/petr.14737","DOIUrl":"https://doi.org/10.1111/petr.14737","url":null,"abstract":"BackgroundVenous complications after pediatric liver transplantation seriously affect the survival rate of patients and grafts. At present, the diagnostic indicators have not been unified. Venous complications may cause portal hypertension, which may lead to splenomegaly and splenic vein dilatation. Therefore, the changes in spleen may be closely related to the venous complications. The purpose of this study was to explore the relationship between ultrasonic splenic parameters and venous complications and to study whether these splenic parameters can be used for the diagnosis of venous complications.MethodsWe retrospectively included pediatric patients who underwent liver transplantation and collected ultrasonic spleen parameters before, and then 1–3 days, 1–3 weeks, 1–3 months, and 4–12 months after liver transplantation. We observed whether there were portal vein or hepatic vein complications within 1 year after liver transplantation.ResultsAmong 109 pediatric patients after liver transplantation included in our study, 11 of them suffered from portal vein complications and nine hepatic vein complications. Spleen transverse diameter, spleen longitudinal diameter, spleen portal vein diameter, spleen index, spleen transverse diameter ratio, spleen longitudinal diameter ratio, and spleen index ratio were independent risk factors of venous complications. The accuracy of spleen transverse diameter (AUROC: 0.73), spleen index (AUROC: 0.70), spleen transverse diameter ratio (AUROC: 0.71), and spleen index ratio (AUROC: 0.72) in predicting venous complications were higher than other ones.ConclusionsUltrasonic examination is a common follow‐up method for pediatric patients after liver transplantation and the application of ultrasonic spleen parameters may be helpful to monitor venous complications.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Buria Naeem, Adil Ayub, Jorge Coss‐Bu, Muhammad Umair M. Mian, Ruben Hernaez, Thomas P. Fogarty, Kirby Deshotels, Curt Kennedy, John Goss, Moreshwar S. Desai
IntroductionAcute‐on‐chronic liver failure (ACLF) is associated with increased mortality and morbidity in patients with biliary atresia (BA). Data on impact of ACLF on postoperative outcomes, however, are sparse.MethodWe performed a retrospective analysis of patients with BA aged <18 years who underwent LT between 2011 and 2021 at our institution. ACLF was defined using the pediatric ACLF criteria: ≥1 extra‐hepatic organ failure in children with decompensated cirrhosis.ResultsOf 107 patients (65% female; median age 14 [9–31] months) who received a LT, 13 (12%) had ACLF during the index admission prior to LT. Two (15%) had Grade 1; 4 (30%) had Grade 2; and 7 (55%) had Grade ≥3 ACLF. ACLF cohort was younger at time of listing (5 [4–8] vs. 9 [6–24] months; p < .001) and at LT (8 [8–11] vs. 16 [10–40] months, p < .001) compared to no‐ACLF group. Intraoperatively, ACLF patients had higher blood loss (40 [20–53] vs. 10 [6–19] mL/kg; p < .001) and blood transfusion requirements (33 [21–69] vs. 18 [7–25] mL/kg; p = .004). Postoperatively, they needed higher vasopressor support (31% vs. 10.6%; p = .04) and had higher total hospital length of stay (106 [45–151] vs. 13 [7–30] days; p = .023). Rate of return to the operating room, hospital readmission rates, and 1‐year post‐LT survival rates were comparable between the groups.ConclusionDespite higher perioperative complications, survival outcomes for ACLF in BA after LT are favorable and comparable to those without ACLF. These encouraging data reiterate prioritization during organ allocation of these critically ill children for LT.
{"title":"Postoperative outcomes of acute‐on‐chronic liver failure in infants and children with biliary atresia","authors":"Buria Naeem, Adil Ayub, Jorge Coss‐Bu, Muhammad Umair M. Mian, Ruben Hernaez, Thomas P. Fogarty, Kirby Deshotels, Curt Kennedy, John Goss, Moreshwar S. Desai","doi":"10.1111/petr.14736","DOIUrl":"https://doi.org/10.1111/petr.14736","url":null,"abstract":"IntroductionAcute‐on‐chronic liver failure (ACLF) is associated with increased mortality and morbidity in patients with biliary atresia (BA). Data on impact of ACLF on postoperative outcomes, however, are sparse.MethodWe performed a retrospective analysis of patients with BA aged <18 years who underwent LT between 2011 and 2021 at our institution. ACLF was defined using the pediatric ACLF criteria: ≥1 extra‐hepatic organ failure in children with decompensated cirrhosis.ResultsOf 107 patients (65% female; median age 14 [9–31] months) who received a LT, 13 (12%) had ACLF during the index admission prior to LT. Two (15%) had Grade 1; 4 (30%) had Grade 2; and 7 (55%) had Grade ≥3 ACLF. ACLF cohort was younger at time of listing (5 [4–8] vs. 9 [6–24] months; <jats:italic>p</jats:italic> < .001) and at LT (8 [8–11] vs. 16 [10–40] months, <jats:italic>p</jats:italic> < .001) compared to no‐ACLF group. Intraoperatively, ACLF patients had higher blood loss (40 [20–53] vs. 10 [6–19] mL/kg; <jats:italic>p</jats:italic> < .001) and blood transfusion requirements (33 [21–69] vs. 18 [7–25] mL/kg; <jats:italic>p</jats:italic> = .004). Postoperatively, they needed higher vasopressor support (31% vs. 10.6%; <jats:italic>p</jats:italic> = .04) and had higher total hospital length of stay (106 [45–151] vs. 13 [7–30] days; <jats:italic>p</jats:italic> = .023). Rate of return to the operating room, hospital readmission rates, and 1‐year post‐LT survival rates were comparable between the groups.ConclusionDespite higher perioperative complications, survival outcomes for ACLF in BA after LT are favorable and comparable to those without ACLF. These encouraging data reiterate prioritization during organ allocation of these critically ill children for LT.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580297","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Amanda R. Krysler, Chantal J. Allan, Ingrid Larsen, Sunita Mathur, Catherine Morgan, Steven C. Greenway, Tom Blydt‐Hansen, Michael Khoury, Lori J. West, Simon Urschel
BackgroundPediatric heart (HTx) and kidney transplant (KTx) recipients may have lower physical fitness than healthy children. This study sought to quantify fitness levels in transplant recipients, investigate associations to clinical factors and quality of life, and identify whether a quick, simple wall‐sit test is feasible as a surrogate for overall fitness for longitudinal assessment.MethodsAerobic capacity (6‐min walk test, 6MWT), normalized muscle strength, muscle endurance, physical activity questionnaire (PAQ), and quality of life (PedsQL™) were prospectively assessed in transplanted children and matched healthy controls.ResultsTwenty‐two HTx were compared to 20 controls and 6 KTx. 6MWT %predicted was shorter in HTx (87.2 [69.9–118.6] %) than controls (99.9 [80.4–120] %), but similar to KTx (90.3 [78.6–115] %). Muscle strength was lower in HTx deltoids (6.15 [4.35–11.3] kg/m2) and KTx quadriceps (9.27 [8.65–19.1] kg/m2) versus controls. Similarly, muscle endurance was lower in HTx push‐ups (28.6 [0–250] %predicted), KTx push‐ups (8.35 [0–150] %predicted), HTx curl‐ups (115 [0–450] %predicted), and KTx wall‐sit time (18.5 [10.0–54.0] s) than controls. In contrast to HTx with only 9%, all KTx were receiving steroid therapy. The wall‐sit test significantly correlated with other fitness parameters (normalized quadriceps strength R = .31, #push‐ups R = .39, and #curl‐ups R = .43) and PedsQL™ (R = .36).ConclusionsCompared to controls, pediatric HTx and KTx have similarly lower aerobic capacity, but different deficits in muscle strength, likely related to steroid therapy in KTx. The convenient wall‐sit test correlates with fitness and reported quality of life, and thus could be a useful easy routine for longitudinal assessment.
{"title":"Let's get physical: Aerobic capacity, muscle strength, and muscle endurance after pediatric heart transplantation","authors":"Amanda R. Krysler, Chantal J. Allan, Ingrid Larsen, Sunita Mathur, Catherine Morgan, Steven C. Greenway, Tom Blydt‐Hansen, Michael Khoury, Lori J. West, Simon Urschel","doi":"10.1111/petr.14731","DOIUrl":"https://doi.org/10.1111/petr.14731","url":null,"abstract":"BackgroundPediatric heart (HTx) and kidney transplant (KTx) recipients may have lower physical fitness than healthy children. This study sought to quantify fitness levels in transplant recipients, investigate associations to clinical factors and quality of life, and identify whether a quick, simple wall‐sit test is feasible as a surrogate for overall fitness for longitudinal assessment.MethodsAerobic capacity (6‐min walk test, 6MWT), normalized muscle strength, muscle endurance, physical activity questionnaire (PAQ), and quality of life (PedsQL™) were prospectively assessed in transplanted children and matched healthy controls.ResultsTwenty‐two HTx were compared to 20 controls and 6 KTx. 6MWT %predicted was shorter in HTx (87.2 [69.9–118.6] %) than controls (99.9 [80.4–120] %), but similar to KTx (90.3 [78.6–115] %). Muscle strength was lower in HTx deltoids (6.15 [4.35–11.3] kg/m<jats:sup>2</jats:sup>) and KTx quadriceps (9.27 [8.65–19.1] kg/m<jats:sup>2</jats:sup>) versus controls. Similarly, muscle endurance was lower in HTx push‐ups (28.6 [0–250] %predicted), KTx push‐ups (8.35 [0–150] %predicted), HTx curl‐ups (115 [0–450] %predicted), and KTx wall‐sit time (18.5 [10.0–54.0] s) than controls. In contrast to HTx with only 9%, all KTx were receiving steroid therapy. The wall‐sit test significantly correlated with other fitness parameters (normalized quadriceps strength <jats:italic>R</jats:italic> = .31, #push‐ups <jats:italic>R</jats:italic> = .39, and #curl‐ups <jats:italic>R</jats:italic> = .43) and PedsQL™ (<jats:italic>R</jats:italic> = .36).ConclusionsCompared to controls, pediatric HTx and KTx have similarly lower aerobic capacity, but different deficits in muscle strength, likely related to steroid therapy in KTx. The convenient wall‐sit test correlates with fitness and reported quality of life, and thus could be a useful easy routine for longitudinal assessment.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Isa F. Ashoor, Rachel M. Engen, Dechu Puliyanda, Nicole Hayde, Caitlin G. Peterson, Rima S. Zahr, Sonia Solomon, Mahmoud Kallash, Rouba Garro, Amrish Jain, Lyndsay A. Harshman, Scott T. McEwen, Asifhusen Mansuri, Mathew J. Gregoski, Katherine E. Twombley
BackgroundAntibody‐mediated rejection (AMR) is a major cause of kidney allograft loss. There is a paucity of large‐scale pediatric‐specific data regarding AMR treatment outcomes.MethodsData were obtained from 14 centers within the Pediatric Nephrology Research Consortium. Kidney transplant recipients aged 1–18 years at transplant with biopsy‐proven AMR between 2009 and 2019 and at least 12 months of follow‐up were included. The primary outcome was graft failure or an eGFR <20 mL/min/1.73 m2 at 12 months following AMR treatment. AMR treatment choice, histopathology, and DSA class were also examined.ResultsWe reviewed 123 AMR episodes. Median age at diagnosis was 15 years at a median 22 months post‐transplant. The primary outcome developed in 27.6%. eGFR <30 m/min/1.73 m2 at AMR diagnosis was associated with a 5.6‐fold higher risk of reaching the composite outcome. There were no significant differences in outcome by treatment modality. Histopathology scores and DSA class at time of AMR diagnosis were not significantly associated with the primary outcome.ConclusionsIn this large cohort of pediatric kidney transplant recipients with AMR, nearly one‐third of patients experienced graft failure or significant graft dysfunction within 12 months of diagnosis. Poor graft function at time of diagnosis was associated with higher odds of graft failure.
背景抗体介导的排斥反应（AMR）是造成肾脏异体移植损失的主要原因。有关 AMR 治疗结果的大规模儿科特定数据很少。2009年至2019年期间，年龄在1-18岁的肾移植受者在移植时经活检证实患有AMR，并接受了至少12个月的随访。主要结果是移植失败或AMR治疗后12个月的eGFR<20 mL/min/1.73 m2。我们还对AMR治疗选择、组织病理学和DSA分级进行了研究。中位诊断年龄为 15 岁，中位移植时间为 22 个月。确诊 AMR 时，eGFR <30 m/min/1.73 m2 与达到综合结果的风险高出 5.6 倍有关。不同治疗方式的结果无明显差异。在这一庞大的AMR小儿肾移植受者队列中，近三分之一的患者在确诊后12个月内出现移植失败或严重的移植功能障碍。诊断时移植物功能不佳与移植物失败的几率较高有关。
{"title":"Antibody‐mediated rejection in pediatric kidney transplant recipients: A report from the Pediatric Nephrology Research Consortium","authors":"Isa F. Ashoor, Rachel M. Engen, Dechu Puliyanda, Nicole Hayde, Caitlin G. Peterson, Rima S. Zahr, Sonia Solomon, Mahmoud Kallash, Rouba Garro, Amrish Jain, Lyndsay A. Harshman, Scott T. McEwen, Asifhusen Mansuri, Mathew J. Gregoski, Katherine E. Twombley","doi":"10.1111/petr.14734","DOIUrl":"https://doi.org/10.1111/petr.14734","url":null,"abstract":"BackgroundAntibody‐mediated rejection (AMR) is a major cause of kidney allograft loss. There is a paucity of large‐scale pediatric‐specific data regarding AMR treatment outcomes.MethodsData were obtained from 14 centers within the Pediatric Nephrology Research Consortium. Kidney transplant recipients aged 1–18 years at transplant with biopsy‐proven AMR between 2009 and 2019 and at least 12 months of follow‐up were included. The primary outcome was graft failure or an eGFR <20 mL/min/1.73 m<jats:sup>2</jats:sup> at 12 months following AMR treatment. AMR treatment choice, histopathology, and DSA class were also examined.ResultsWe reviewed 123 AMR episodes. Median age at diagnosis was 15 years at a median 22 months post‐transplant. The primary outcome developed in 27.6%. eGFR <30 m/min/1.73 m<jats:sup>2</jats:sup> at AMR diagnosis was associated with a 5.6‐fold higher risk of reaching the composite outcome. There were no significant differences in outcome by treatment modality. Histopathology scores and DSA class at time of AMR diagnosis were not significantly associated with the primary outcome.ConclusionsIn this large cohort of pediatric kidney transplant recipients with AMR, nearly one‐third of patients experienced graft failure or significant graft dysfunction within 12 months of diagnosis. Poor graft function at time of diagnosis was associated with higher odds of graft failure.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
ObjectiveWe investigated the safety and efficacy of haploidentical stem cell transplantation (SCT) in pediatric patients with X‐linked adrenoleukodystrophy (ALD).MethodsA retrospective analysis of transplantation data from 29 cases of ALD, treated between December 2014 and April 2022, was conducted. Neurologic function scores (NFS) were assessed. The conditioning regimen was busulfan 9.6 mg/kg, cyclophosphamide 200 mg/kg, and fludarabine 90 mg/m2 (BFC). Graft‐versus‐host disease prophylaxis consisted of anti‐human thymocyte globulin, cyclosporine A, mycophenolate mofetil, and short course of methotrexate.ResultsAmong the 29 cases, 14 cases (NFS = 0) were asymptomatic, and 15 (NFS ≥ 1) were symptomatic. The median age at SCT was 8 years (range: 4–16 years); the median follow‐up time was 1058 days (range: 398–3092 days); 28 cases were father donors and 1 case was a grandfather donor. Hematopoietic reconstitution was successful in all patients, and all of them achieved complete donor chimerism at the time of engraftment. The leading cause of death was still primary disease progression (n = 4). Survival free of major functional disabilities was 100% in asymptomatic patients versus 66.67% in the symptomatic group (p = .018).ConclusionBFC regimen used in haploidentical SCT was administered safely without major transplant‐related complications even in symptomatic patients, and neurological symptoms were stabilized after SCT.
{"title":"Haploidentical hematopoietic stem cell transplantation with busulfan, cyclophosphamide, and fludarabine conditioning for X‐linked adrenal cerebral leukodystrophy","authors":"Yao Chen, Lan‐ping Xu, Xiao‐hui Zhang, Huan Chen, Kai‐yan Liu, Jiong Qing, Yan‐ling Yang, Xiao‐jun Huang","doi":"10.1111/petr.14735","DOIUrl":"https://doi.org/10.1111/petr.14735","url":null,"abstract":"ObjectiveWe investigated the safety and efficacy of haploidentical stem cell transplantation (SCT) in pediatric patients with X‐linked adrenoleukodystrophy (ALD).MethodsA retrospective analysis of transplantation data from 29 cases of ALD, treated between December 2014 and April 2022, was conducted. Neurologic function scores (NFS) were assessed. The conditioning regimen was busulfan 9.6 mg/kg, cyclophosphamide 200 mg/kg, and fludarabine 90 mg/m<jats:sup>2</jats:sup> (BFC). Graft‐versus‐host disease prophylaxis consisted of anti‐human thymocyte globulin, cyclosporine A, mycophenolate mofetil, and short course of methotrexate.ResultsAmong the 29 cases, 14 cases (NFS = 0) were asymptomatic, and 15 (NFS ≥ 1) were symptomatic. The median age at SCT was 8 years (range: 4–16 years); the median follow‐up time was 1058 days (range: 398–3092 days); 28 cases were father donors and 1 case was a grandfather donor. Hematopoietic reconstitution was successful in all patients, and all of them achieved complete donor chimerism at the time of engraftment. The leading cause of death was still primary disease progression (<jats:italic>n</jats:italic> = 4). Survival free of major functional disabilities was 100% in asymptomatic patients versus 66.67% in the symptomatic group (<jats:italic>p</jats:italic> = .018).ConclusionBFC regimen used in haploidentical SCT was administered safely without major transplant‐related complications even in symptomatic patients, and neurological symptoms were stabilized after SCT.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580300","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
BackgroundAlthough neurotoxicity is a major adverse event associated with busulfan, little information is available regarding the association between drug interactions and neurological symptoms during busulfan‐based regimens. This study evaluated the association between prophylactic echinocandins and neurological complications in patients receiving busulfan‐containing conditioning regimens for stem cell transplantation.MethodsWe retrospectively included consecutive patients who administered intravenous busulfan as a conditioning regimen at our facility between 2007 and 2022. Prophylactic echinocandin use was defined as the use of an echinocandin antifungal drug to prevent invasive fungal disease in SCT recipients. The primary outcome was the incidence of neurological complications within 7 days of busulfan initiation and was compared between the echinocandin group (patients received prophylactic echinocandin) and nonechinocandin group (patients received prophylactic antifungal drugs other than echinocandin and those without antifungal prophylaxis).ResultsAmong the 59 patients included in this study, the incidence of neurological complications in the echinocandin (n = 26) and nonechinocandin groups (n = 33) was 30.8% and 63.6%, respectively. We observed a negative association between prophylactic echinocandin use and the development of neurological complications after adjusting for the propensity score for receiving prophylactic echinocandins (adjusted odds ratio 0.294, 95% confidence interval 0.090 to 0.959). We observed a lower incidence of neurological complications in the echinocandin group than in the nonechinocandin group.ConclusionOur results suggested that the choice of antifungal prophylaxis is associated with busulfan neurotoxicity.
{"title":"Impact of prophylactic echinocandin on the development of neurological complications in patients receiving busulfan‐containing conditioning regimens for stem cell transplantation: A single‐center retrospective study","authors":"Shogo Horikawa, Kenji Kishimoto, Suguru Uemura, Sayaka Hyodo, Aiko Kozaki, Atsuro Saito, Toshiaki Ishida, Takeshi Mori, Daiichiro Hasegawa, Yoshiyuki Kosaka","doi":"10.1111/petr.14728","DOIUrl":"https://doi.org/10.1111/petr.14728","url":null,"abstract":"BackgroundAlthough neurotoxicity is a major adverse event associated with busulfan, little information is available regarding the association between drug interactions and neurological symptoms during busulfan‐based regimens. This study evaluated the association between prophylactic echinocandins and neurological complications in patients receiving busulfan‐containing conditioning regimens for stem cell transplantation.MethodsWe retrospectively included consecutive patients who administered intravenous busulfan as a conditioning regimen at our facility between 2007 and 2022. Prophylactic echinocandin use was defined as the use of an echinocandin antifungal drug to prevent invasive fungal disease in SCT recipients. The primary outcome was the incidence of neurological complications within 7 days of busulfan initiation and was compared between the echinocandin group (patients received prophylactic echinocandin) and nonechinocandin group (patients received prophylactic antifungal drugs other than echinocandin and those without antifungal prophylaxis).ResultsAmong the 59 patients included in this study, the incidence of neurological complications in the echinocandin (<jats:italic>n</jats:italic> = 26) and nonechinocandin groups (<jats:italic>n</jats:italic> = 33) was 30.8% and 63.6%, respectively. We observed a negative association between prophylactic echinocandin use and the development of neurological complications after adjusting for the propensity score for receiving prophylactic echinocandins (adjusted odds ratio 0.294, 95% confidence interval 0.090 to 0.959). We observed a lower incidence of neurological complications in the echinocandin group than in the nonechinocandin group.ConclusionOur results suggested that the choice of antifungal prophylaxis is associated with busulfan neurotoxicity.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Taufiek K. Rajab, Mohamed Abdelrahman, Elridge J. Schwartzenburg, Berk Aykut, Joseph W. Turek, Deani H. McVadon
IntroductionPartial heart transplants are a new type of pediatric transplant that replace defective heart valves with the parts of matched donor hearts containing the necessary valves. Short‐term outcomes of partial heart transplants are excellent, but long‐term outcomes are unknown. In order to predict the long‐term outcomes of partial heart transplants, we evaluated long‐term growth and function of semilunar heart valves transplanted in infancy as part of a heart transplant.MethodsAll children who underwent infant heart transplantation at a single center from 1997 to 2014 were included in this study. Children in whom echocardiograms after heart transplantation and after 10 years were not available for review were excluded. The echocardiograms were reviewed by two authors to analyze semilunar valve annulus diameters, Z‐scores, peak valve gradients, and valve regurgitation. Statistical difference was determined using two‐tailed, paired sample t‐tests with Bonferroni correction for multiple comparisons.ResultsData from 15 patients were analyzed. The aortic valve annulus averaged 1.3 cm (range 0.7–1.8 cm) immediately after transplantation and grew to an average of 1.7 cm (range 1.4–2.3 cm) after 10 years (p < .001). After 10 years, the aortic valve peak gradient avereraged 5.1 mmHg (range 2.1–15.5 mmHg) and none of the valves had more than trivial regurgitation. The pulmonary valve annulus averaged 1.5 cm (range 1.1–2.5 cm) immediately after transplantation and grew to an average of 2.1 cm (range 1.0–2.9 cm) after 10 years (p < .001). After 10 years, the pulmonary valve peak gradient averaged 4.3 mmHg (range 1.1–13.8 mmHg), and 7% of valves had moderate regurgitation.DiscussionSemilunar heart valves transplanted in infancy as part of a heart transplant demonstrate statistically significant growth and excellent function after 10 years. This predicts excellent long‐term outcomes of partial heart transplants.
{"title":"Semilunar valve growth and function 10 years after infant heart transplantation: Predicting long‐term outcomes of partial heart transplants","authors":"Taufiek K. Rajab, Mohamed Abdelrahman, Elridge J. Schwartzenburg, Berk Aykut, Joseph W. Turek, Deani H. McVadon","doi":"10.1111/petr.14746","DOIUrl":"https://doi.org/10.1111/petr.14746","url":null,"abstract":"IntroductionPartial heart transplants are a new type of pediatric transplant that replace defective heart valves with the parts of matched donor hearts containing the necessary valves. Short‐term outcomes of partial heart transplants are excellent, but long‐term outcomes are unknown. In order to predict the long‐term outcomes of partial heart transplants, we evaluated long‐term growth and function of semilunar heart valves transplanted in infancy as part of a heart transplant.MethodsAll children who underwent infant heart transplantation at a single center from 1997 to 2014 were included in this study. Children in whom echocardiograms after heart transplantation and after 10 years were not available for review were excluded. The echocardiograms were reviewed by two authors to analyze semilunar valve annulus diameters, <jats:italic>Z</jats:italic>‐scores, peak valve gradients, and valve regurgitation. Statistical difference was determined using two‐tailed, paired sample <jats:italic>t</jats:italic>‐tests with Bonferroni correction for multiple comparisons.ResultsData from 15 patients were analyzed. The aortic valve annulus averaged 1.3 cm (range 0.7–1.8 cm) immediately after transplantation and grew to an average of 1.7 cm (range 1.4–2.3 cm) after 10 years (<jats:italic>p</jats:italic> < .001). After 10 years, the aortic valve peak gradient avereraged 5.1 mmHg (range 2.1–15.5 mmHg) and none of the valves had more than trivial regurgitation. The pulmonary valve annulus averaged 1.5 cm (range 1.1–2.5 cm) immediately after transplantation and grew to an average of 2.1 cm (range 1.0–2.9 cm) after 10 years (<jats:italic>p</jats:italic> < .001). After 10 years, the pulmonary valve peak gradient averaged 4.3 mmHg (range 1.1–13.8 mmHg), and 7% of valves had moderate regurgitation.DiscussionSemilunar heart valves transplanted in infancy as part of a heart transplant demonstrate statistically significant growth and excellent function after 10 years. This predicts excellent long‐term outcomes of partial heart transplants.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580298","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Adolescents and young adults with and without chronic illnesses partake in risk‐taking behavior. Clinicians in transplant clinics should be aware of the prevalence of risk‐taking behavior in their adolescent and young adult solid organ transplant patients in order to provide complete care. Creating an environment where teens and young adults feel comfortable discussing risky behavior is important and includes creating a privacy policy and increasing comfort of the healthcare provider in asking sensitive questions. This review is intended to help the providers in the transplant clinic screen for and counsel about risk‐taking behaviors with their adolescent and young adult patients, specifically around sexual and reproductive health.
{"title":"Sexual and reproductive health screening and counseling in adolescent and young adult transplant recipients","authors":"Paula Cody","doi":"10.1111/petr.14741","DOIUrl":"https://doi.org/10.1111/petr.14741","url":null,"abstract":"Adolescents and young adults with and without chronic illnesses partake in risk‐taking behavior. Clinicians in transplant clinics should be aware of the prevalence of risk‐taking behavior in their adolescent and young adult solid organ transplant patients in order to provide complete care. Creating an environment where teens and young adults feel comfortable discussing risky behavior is important and includes creating a privacy policy and increasing comfort of the healthcare provider in asking sensitive questions. This review is intended to help the providers in the transplant clinic screen for and counsel about risk‐taking behaviors with their adolescent and young adult patients, specifically around sexual and reproductive health.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Isa F. Ashoor, Samhar Al‐Akash, Sarah Kizilbash, Asha Moudgil, Dechu Puliyanda, Saritha Ranabothu, Yi Shi, Vikas Dharnidharka
BackgroundThere are scant data on the effect of rituximab on EBV DNA levels and prevention of post‐transplant lymphoproliferative disorder (PTLD) in pediatric kidney transplant recipients with EBV DNAemia.MethodsKidney transplant recipients with EBV DNAemia treated with rituximab to prevent PTLD between 7/1999 and 7/2019 at five pediatric centers were included. Those with confirmed PTLD at the onset of rituximab were excluded. Primary outcomes included percentage change in EBV DNAemia and occurrence of PTLD post rituximab.ResultsTwenty‐six pediatric kidney transplant recipients were included. Median age at transplant was 4 years (IQR 2.1–10.3). EBV DNA load monitoring by qPCR was performed at 1–3 month intervals. EBV DNAemia onset occurred at a median of 73 days post‐transplant (IQR 52–307), followed by DNAemia peak at a median of 268 days (IQR 112–536). Rituximab was administered at a median of 9 days post peak (IQR 0–118). Rituximab regimens varied; median dose 375 mg/m2 (IQR 375–439) weekly for 1–4 doses per course. Following rituximab, EBV DNA load decreased to <10% of baseline at 120 days in 20/26 patients; however, only 30% achieved complete resolution at last follow‐up (median 2094 days post‐transplant [IQR 1538–3463]). Two (7%) developed PTLD at 915 and 1713 days post rituximab. All recipients had functioning grafts. One death occurred in a child with PTLD following remission due to unrelated reasons.ConclusionsIn the largest pediatric kidney transplant recipient case series with EBV DNAemia given rituximab to prevent PTLD, rituximab achieved a short‐term reduction in DNA load; however, recurrent DNAemia is common.
{"title":"Effect of pre‐emptive rituximab on EBV DNA levels and prevention of post‐transplant lymphoproliferative disorder in pediatric kidney transplant recipients: A case series from the pediatric nephrology research consortium","authors":"Isa F. Ashoor, Samhar Al‐Akash, Sarah Kizilbash, Asha Moudgil, Dechu Puliyanda, Saritha Ranabothu, Yi Shi, Vikas Dharnidharka","doi":"10.1111/petr.14743","DOIUrl":"https://doi.org/10.1111/petr.14743","url":null,"abstract":"BackgroundThere are scant data on the effect of rituximab on EBV DNA levels and prevention of post‐transplant lymphoproliferative disorder (PTLD) in pediatric kidney transplant recipients with EBV DNAemia.MethodsKidney transplant recipients with EBV DNAemia treated with rituximab to prevent PTLD between 7/1999 and 7/2019 at five pediatric centers were included. Those with confirmed PTLD at the onset of rituximab were excluded. Primary outcomes included percentage change in EBV DNAemia and occurrence of PTLD post rituximab.ResultsTwenty‐six pediatric kidney transplant recipients were included. Median age at transplant was 4 years (IQR 2.1–10.3). EBV DNA load monitoring by qPCR was performed at 1–3 month intervals. EBV DNAemia onset occurred at a median of 73 days post‐transplant (IQR 52–307), followed by DNAemia peak at a median of 268 days (IQR 112–536). Rituximab was administered at a median of 9 days post peak (IQR 0–118). Rituximab regimens varied; median dose 375 mg/m<jats:sup>2</jats:sup> (IQR 375–439) weekly for 1–4 doses per course. Following rituximab, EBV DNA load decreased to <10% of baseline at 120 days in 20/26 patients; however, only 30% achieved complete resolution at last follow‐up (median 2094 days post‐transplant [IQR 1538–3463]). Two (7%) developed PTLD at 915 and 1713 days post rituximab. All recipients had functioning grafts. One death occurred in a child with PTLD following remission due to unrelated reasons.ConclusionsIn the largest pediatric kidney transplant recipient case series with EBV DNAemia given rituximab to prevent PTLD, rituximab achieved a short‐term reduction in DNA load; however, recurrent DNAemia is common.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140580013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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