Kortbeek Simone, Anderson Sarah G, Alonso Estella M, Rand Elizabeth B, Bucuvalas John, Mazariegos George V, Campbell Kathleen M, Lobritto Steven J, Feldman Amy G, Mysore Krupa R, Anand Ravinder, Selzner Nazia, Ng Vicky L
Department of Pediatrics, Section of Pediatric Gastroenterology, Hepatology and Nutrition, Alberta Children's Hospital, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.
The Emmes Corporation, Rockville, MD.
J Pediatr. 2024 Jan;264:113744. doi: 10.1016/j.jpeds.2023.113744. Epub 2023 Sep 17.
To compare long-term outcomes of pediatric liver transplant (LT) recipients off immunosuppression (IS) with matched controls on IS using data from the Society of Pediatric Liver Transplant (SPLIT) registry.
This was a retrospective case-control study. SPLIT participants <18 years of age, ≥4 years after isolated LT, and off IS for ≥1 year (cases) were age- and sex-matched 1:2 to patients with the same primary diagnosis and post-LT follow-up duration (controls). Primary outcomes included retransplantation, allograft rejection, IS comorbidities, and prevalence of SPLIT-derived composite ideal outcome (c-IO) achieved at the end of the follow-up period. Differences were compared using multiple linear regression for continuous outcomes and logistic regression for dichotomous data.
The study cohort was composed of 33 cases (42.4% male, 60.6% biliary atresia, median age at LT of 0.7 [P25, P75, 0.5, 1.6] years, median IS withdrawal time of 9 [P25, P75, 6, 12] years after LT) and 66 age- and sex-matched controls. No cases required retransplantation. Cases and controls had similar growth parameters, laboratory values, calculated glomerular filtration rates, rates of post-transplant lymphoproliferative disease, graft rejection, and attainment of c-IO.
No differences in allograft rejection rates, IS complications, or c-IO prevalence were seen between SPLIT patients off IS and age- and sex-matched controls remaining on IS. Discontinuation of IS most commonly occurred in the context of rigorously designed IS withdrawal trials. The available sample size was small, affecting generalizability to the broader pediatric LT population.
利用小儿肝移植协会（SPLIT）登记处的数据，比较停用免疫抑制（IS）的小儿肝移植（LT）受者与使用IS的匹配对照组的长期结局。
这是一项回顾性病例对照研究。SPLIT中年龄<18岁、孤立肝移植术后≥4年且停用IS≥1年的参与者（病例组）按年龄和性别1:2与具有相同原发性诊断和肝移植术后随访时间的患者（对照组）匹配。主要结局包括再次移植、移植物排斥反应、IS合并症以及随访期末实现的SPLIT衍生综合理想结局（c-IO）的患病率。对于连续结局，使用多元线性回归比较差异；对于二分数据，使用逻辑回归比较差异。
研究队列由33例病例（男性占42.4%，胆道闭锁占60.6%，肝移植时中位年龄为0.7[P25，P75，0.5，1.6]岁，肝移植术后IS停用中位时间为9[P25，P75，6，12]年）和66例年龄及性别匹配的对照组组成。没有病例需要再次移植。病例组和对照组在生长参数、实验室值、计算的肾小球滤过率、移植后淋巴细胞增生性疾病发生率、移植物排斥反应以及c-IO的实现情况方面相似。
在SPLIT中，停用IS的患者与继续使用IS的年龄及性别匹配的对照组相比，移植物排斥率、IS并发症或c-IO患病率没有差异。IS的停用最常发生在精心设计的IS撤药试验背景下。可用样本量较小，影响了对更广泛小儿肝移植人群的普遍性。
