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Biliary strictures post pediatric liver transplantation—incidence and risk factors in a single tertiary referral transplant center 小儿肝移植术后胆道狭窄--一家三级转诊移植中心的发病率和风险因素
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-13 DOI: 10.1111/petr.14727
Odelia Vingrovich, Shiri Cooper, Michael Gurevich, Aenov Cohen, Yael Mozer‐Glassberg, Michal Rosenfeld Bar‐Lev, Raanan Shamir, Orith Waisbourd‐Zinman
BackgroundBiliary strictures are a significant cause of morbidity and graft loss in pediatric liver transplant recipients. Risk factors for the development of biliary strictures are not fully established. We aimed to evaluate the incidence of biliary strictures and treatment modalities outcomes and to identify potential risk factors for occurrence.MethodsPediatric patients who underwent liver transplantation in the single tertiary pediatric liver transplant center in Israel were evaluated. We compared demographics, presentation, laboratory results, imaging, treatment, and outcomes between patients with and without biliary stricture. Multivariate regression analyses were used to identify risk factors for biliary strictures.ResultsAmong 121 pediatric liver transplant patients, 65 (53.7%) were males; the median age at the time of liver transplantation was 43 (3–215) months. Fifteen patients (12.4%) had biliary strictures following transplantation. One (7%) patient with biliary stricture was treated via endoscopic retrograde cholangiopancreatography, and 12 patients (80%) underwent interventions via a percutaneous transhepatic approach. Nine of the 12 patients were treated successfully, requiring one or multiple procedures, while the remaining had surgery or laser therapy. Risk factors for the development of biliary strictures were biliary leak, acute cellular rejection, and the presence of two biliary anastomoses.ConclusionsIn our cohort, the presence of two biliary anastomoses and post‐transplant complications including acute cellular rejection and early biliary leaks were associated with biliary strictures in pediatric liver transplantation recipients. Percutaneous transhepatic interventions result in good outcomes in most patients.
背景胆道狭窄是小儿肝移植受者发病率和移植物损失的重要原因。胆道狭窄发生的风险因素尚未完全确定。我们旨在评估胆道狭窄的发生率和治疗效果,并确定发生胆道狭窄的潜在风险因素。我们比较了有胆道狭窄和无胆道狭窄患者的人口统计学特征、发病情况、实验室结果、影像学检查、治疗和结果。结果在121例小儿肝移植患者中,65例(53.7%)为男性;肝移植时的中位年龄为43(3-215)个月。15名患者(12.4%)在移植后出现胆道狭窄。1名胆道狭窄患者(7%)通过内镜逆行胰胆管造影术进行了治疗,12名患者(80%)通过经皮经肝途径进行了介入治疗。12 名患者中有 9 人治疗成功,只需进行一次或多次手术,其余患者则接受了手术或激光治疗。结论在我们的队列中,存在两个胆道吻合口以及移植后并发症(包括急性细胞排斥反应和早期胆漏)与小儿肝移植受者胆道狭窄有关。经皮经肝介入治疗可为大多数患者带来良好的治疗效果。
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引用次数: 0
Small‐for‐size liver graft syndrome. Not everything is what it seems to be: Even salt looks like sugar 小尺寸肝脏移植综合征。并非所有东西都是表里如一的:盐看起来也像糖
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-13 DOI: 10.1111/petr.14748
Jean de Ville de Goyet

CONFLICT OF INTEREST STATEMENT

No conflict of interest.

利益冲突声明 无利益冲突。
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引用次数: 0
Pediatric donor heart utilization variability among organ procurement organizations 器官获取组织之间小儿捐献心脏利用率的差异
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-13 DOI: 10.1111/petr.14747
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.
背景器官获取组织(OPO)负责器官捐献者的医疗管理。鉴于OPO之间小儿捐献心脏利用率的差异,我们研究了可能解释这种差异的因素,包括捐献者医疗管理、器官质量和候选者因素的差异。方法查询器官获取和移植网络数据库,了解2010年至2019年期间小儿(<18岁)心脏捐献者和接受小儿捐献心脏的候选者。根据小儿心脏供体利用率对OPO进行分层,并根据供体管理策略和结果对最高和最低的五分位数进行比较。结果在基线供体特征、供体与排序中心之间的距离、管理策略或器官质量方面,最高和最低五分位数的OPO之间没有显著的临床差异。机器学习建模表明,OPO供体管理和心脏功能都不是器官是否被接受的主要驱动因素。结论OPO临床实践的差异似乎并不能解释OPO之间儿科捐献者心脏利用率的差异。挂牌中心接受器官的做法和之前被拒绝的捐献者数量似乎是心脏利用率的重要驱动因素,考虑到OPO和挂牌中心之间的区域关联,至少可以部分解释OPO心脏利用率的差异。
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引用次数: 0
A new method to predict venous complications in pediatric liver transplantation: Evaluation of splenic parameters by ultrasonography 预测小儿肝移植静脉并发症的新方法:通过超声波评估脾脏参数
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14737
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.
背景小儿肝移植术后静脉并发症严重影响患者和移植物的存活率。目前,诊断指标尚未统一。静脉并发症可引起门静脉高压,从而导致脾脏肿大和脾静脉扩张。因此,脾脏的变化可能与静脉并发症密切相关。本研究的目的是探讨超声脾脏参数与静脉并发症的关系,并研究这些脾脏参数是否可用于静脉并发症的诊断。方法我们回顾性地纳入了接受肝移植的儿科患者,并收集了肝移植前、肝移植后1-3天、1-3周、1-3个月和4-12个月的超声脾脏参数。我们观察了肝移植后 1 年内是否有门静脉或肝静脉并发症。结果在我们的研究中,109 例肝移植后的小儿患者中有 11 例出现门静脉并发症,9 例出现肝静脉并发症。脾脏横径、脾脏纵径、脾脏门静脉直径、脾脏指数、脾脏横径比、脾脏纵径比、脾脏指数比是静脉并发症的独立危险因素。脾横径(AUROC:0.73)、脾指数(AUROC:0.70)、脾横径比(AUROC:0.71)和脾指数比(AUROC:0.72)预测静脉并发症的准确性高于其他参数。
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引用次数: 0
Postoperative outcomes of acute‐on‐chronic liver failure in infants and children with biliary atresia 胆道闭锁婴幼儿急性-慢性肝功能衰竭的术后结果
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14736
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.
导言急性肝衰竭(ACLF)与胆道闭锁(BA)患者死亡率和发病率的增加有关。我们对 2011 年至 2021 年期间在本院接受 LT 的 18 岁胆道闭锁患者进行了回顾性分析。结果 在接受LT的107例患者中(65%为女性;中位年龄14 [9-31]个月),13例(12%)在LT前入院时出现ACLF。2例(15%)为1级,4例(30%)为2级,7例(55%)为≥3级前交叉韧带损伤。与无 ACLF 组相比,ACLF 组患者在入院时(5 [4-8] 个月 vs. 9 [6-24] 个月;p < .001)和 LT 时(8 [8-11] 个月 vs. 16 [10-40] 个月,p < .001)更年轻。术中,ACLF 患者失血量更高(40 [20-53] 对 10 [6-19] mL/kg;p < .001),输血需求更高(33 [21-69] 对 18 [7-25] mL/kg;p = .004)。术后,他们需要更多的血管加压支持(31% vs. 10.6%;p = .04),住院总时间也更长(106 [45-151] 天 vs. 13 [7-30] 天;p = .023)。结论尽管围手术期并发症较高,但ACLF患者在LT术后的生存状况良好,与无ACLF患者相当。这些令人鼓舞的数据重申了对这些重症患儿进行LT器官分配时的优先顺序。
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引用次数: 0
Let's get physical: Aerobic capacity, muscle strength, and muscle endurance after pediatric heart transplantation 让我们运动起来小儿心脏移植后的有氧运动能力、肌肉力量和肌肉耐力
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14731
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.
背景小儿心脏(HTx)和肾脏移植(KTx)受者的体能可能低于健康儿童。本研究旨在量化移植受者的体能水平,调查其与临床因素和生活质量的关系,并确定快速、简单的壁坐测试是否可以作为纵向评估整体体能的替代指标。方法对移植儿童和匹配的健康对照组的有氧能力(6 分钟步行测试,6MWT)、归一化肌力、肌耐力、体力活动问卷(PAQ)和生活质量(PedsQL™)进行前瞻性评估。HTx 的 6MWT 预测百分比(87.2 [69.9-118.6] %)短于对照组(99.9 [80.4-120] %),但与 KTx(90.3 [78.6-115] %)相似。与对照组相比,HTx 三角肌(6.15 [4.35-11.3] kg/m2)和 KTx 股四头肌(9.27 [8.65-19.1] kg/m2)的肌肉力量较低。同样,与对照组相比,HTx 俯卧撑(28.6 [0-250] %预测值)、KTx 俯卧撑(8.35 [0-150] %预测值)、HTx 卷发(115 [0-450] %预测值)和 KTx 坐墙时间(18.5 [10.0-54.0] 秒)的肌肉耐力也较低。与只有 9% 的 HTx 相反,所有 KTx 都在接受类固醇治疗。坐墙测试与其他体能参数(归一化股四头肌力量 R = .31、#push-ups R = .39、#curl-ups R = .43)和 PedsQL™ (R = .36)有明显相关性。方便的壁坐测试与体能和报告的生活质量相关,因此可作为纵向评估的有用简便常规方法。
{"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":"276 1","pages":""},"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}
引用次数: 0
Antibody‐mediated rejection in pediatric kidney transplant recipients: A report from the Pediatric Nephrology Research Consortium 小儿肾移植受者抗体介导的排斥反应:小儿肾脏病研究联合会的报告
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14734
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 &lt;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 &lt;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":"85 1","pages":""},"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}
引用次数: 0
Haploidentical hematopoietic stem cell transplantation with busulfan, cyclophosphamide, and fludarabine conditioning for X‐linked adrenal cerebral leukodystrophy 使用丁砜、环磷酰胺和氟达拉滨治疗 X 连锁肾上腺脑白质营养不良症的同种异体造血干细胞移植手术
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14735
Yao Chen, Lan‐ping Xu, Xiao‐hui Zhang, Huan Chen, Kai‐yan Liu, Jiong Qing, Yan‐ling Yang, Xiao‐jun Huang
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.
方法 对2014年12月至2022年4月期间接受治疗的29例ALD患者的移植数据进行了回顾性分析。评估了神经功能评分(NFS)。治疗方案为丁硫醇9.6毫克/千克、环磷酰胺200毫克/千克和氟达拉滨90毫克/平方米(BFC)。结果 29例中,14例(NFS=0)无症状,15例(NFS≥1)有症状。接受 SCT 时的中位年龄为 8 岁(范围:4-16 岁);中位随访时间为 1058 天(范围:398-3092 天);28 例为父亲捐献者,1 例为祖父捐献者。所有患者的造血重建均获得成功,所有患者在移植时均实现了完全的供体嵌合。主要死因仍是原发性疾病进展(4 例)。无症状患者无重大功能障碍的存活率为 100%,而有症状组为 66.67%(P = .018)。
{"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":"51 1","pages":""},"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}
引用次数: 0
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 预防性使用棘白菌素对接受含硫柳胺调理方案进行干细胞移植的患者出现神经系统并发症的影响:单中心回顾性研究
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-11 DOI: 10.1111/petr.14728
Shogo Horikawa, Kenji Kishimoto, Suguru Uemura, Sayaka Hyodo, Aiko Kozaki, Atsuro Saito, Toshiaki Ishida, Takeshi Mori, Daiichiro Hasegawa, Yoshiyuki Kosaka
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.
背景虽然神经毒性是与丁苯磺胺相关的主要不良事件,但有关丁苯磺胺治疗方案中药物相互作用与神经症状之间关系的信息却很少。本研究评估了干细胞移植患者在接受含丁苯磺胺的调理方案时,预防性使用棘白类药物与神经系统并发症之间的关联。方法我们回顾性地纳入了2007年至2022年期间在本院接受静脉注射丁苯磺胺作为调理方案的连续患者。预防性使用棘白菌素是指使用棘白菌素类抗真菌药物预防干细胞移植受者感染真菌疾病。主要研究结果是开始使用硫酸氢钠后7天内神经系统并发症的发生率,并在棘白菌素组(接受预防性棘白菌素治疗的患者)和非棘白菌素组(接受棘白菌素以外的预防性抗真菌药物治疗的患者和未接受抗真菌预防治疗的患者)之间进行比较。结果在纳入本研究的 59 名患者中,棘白菌素组(26 人)和非棘白菌素组(33 人)的神经系统并发症发生率分别为 30.8% 和 63.6%。在对接受预防性棘白菌素类药物的倾向评分进行调整后,我们发现预防性使用棘白菌素类药物与神经系统并发症的发生呈负相关(调整后的几率比为 0.294,95% 置信区间为 0.090 至 0.959)。我们观察到,使用棘白菌素类药物组的神经系统并发症发生率低于未使用棘白菌素类药物组。
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引用次数: 0
Semilunar valve growth and function 10 years after infant heart transplantation: Predicting long‐term outcomes of partial heart transplants 婴儿心脏移植10年后半月瓣的生长和功能:预测部分心脏移植的长期结果
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2024-04-03 DOI: 10.1111/petr.14746
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.
导言部分心脏移植是一种新型的儿科移植手术,用匹配的捐赠者心脏中含有必要瓣膜的部分替代有缺陷的心脏瓣膜。部分心脏移植的短期疗效非常好,但长期疗效尚不清楚。为了预测部分心脏移植的长期预后,我们对婴儿期作为心脏移植一部分移植的半月形心脏瓣膜的长期生长和功能进行了评估。排除了心脏移植后和 10 年后超声心动图无法进行复查的患儿。由两位作者对超声心动图进行复查,分析半月瓣瓣环直径、Z-评分、瓣膜峰值梯度和瓣膜反流。统计差异采用双尾配对样本 t 检验,多重比较采用 Bonferroni 校正。移植后,主动脉瓣环平均为 1.3 厘米(0.7-1.8 厘米不等),10 年后平均增至 1.7 厘米(1.4-2.3 厘米不等)(p < .001)。10 年后,主动脉瓣峰值梯度增加了 5.1 mmHg(范围为 2.1-15.5 mmHg),没有一个瓣膜出现轻微的反流。移植后肺动脉瓣环平均为 1.5 厘米(范围为 1.1-2.5 厘米),10 年后增长到平均 2.1 厘米(范围为 1.0-2.9 厘米)(p < .001)。10年后,肺动脉瓣峰值梯度平均为4.3 mmHg(范围为1.1-13.8 mmHg),7%的瓣膜有中度反流。 讨论在婴儿期作为心脏移植手术一部分移植的半月形心脏瓣膜在10年后显示出显著的统计学增长和良好的功能。这预示着部分心脏移植的长期疗效极佳。
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Pediatric Transplantation
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