临床小儿外科杂志2025，Vol.24Issue(3)：220-225,6.DOI:10.3760/cma.j.cn101785-202501006-004
亲体肝移植治疗儿童遗传代谢病的疗效分析
Analysis of the efficacy of living donor liver transplantation in the treatment of pediatric inborn errors of metabolism
摘要
Abstract
Objective To analyze the clinical outcomes of living donor liver transplantation in the treat-ment of pediatric inborn errors of metabolism(IEM).Methods A retrospective analysis was conducted on the clinical data of 18 pediatric patients with IEM who underwent living donor liver transplantation at the First Affil-iated Hospital of Xinjiang Medical University from January 2021 to July 2024(IEM group).The treatment out-comes and postoperative complications were analyzed and compared with 21 pediatric patients without IEM who underwent living donor liver transplantation at the same period(non-IEM group).Preoperative clinical data and cumulative survival rates were compared between the two groups,as well as changes in weight Z-scores and height Z-scores in the IEM group before surgery and at six months postoperatively.Results Among the 18 pe-diatric patients with IEM,the primary diseases included Wilson's disease(n=8),tyrosinemia(n=2),congen-ital hepatic fibrosis(n=4),maple syrup urine disease(n=1),Caroli disease(n=1),glycogen storage dis-ease type Ⅰ(n=1),and Alagille syndrome(n=1).The median age at transplantation in the IEM and non-IEM groups was 114.00(96.00,132.00)months and 6.57(5.68,77.00)months,respectively,and the height Z-scores were(-0.77±1.62)and(-1.91±2.30),respectively.These differences were statistically significant(P＜0.05).Postoperatively,seven patients in the IEM group experienced varying degrees of compli-cations,all of whom recovered after symptomatic treatment.One patient died in the perioperative period due to disseminated intravascular coagulation.The median follow-up period was 19.75 months.The cumulative postop-erative survival rates in the IEM and non-IEM groups were 87.5％and 84.6％,respectively,with no statistical-ly significant difference(P＞0.05).In the IEM group,the height and weight Z-scores before surgery and six months postoperatively were(-0.77±1.62)vs.(-1.22±2.26)and(-0.27±1.29)vs.(0.03±0.92),respectively,with statistically significant differences(P＜0.05).Conclusions Living donor liver transplanta-tion is an effective treatment for certain inborn errors of metabolism,significantly improving growth and develop-ment in affected children while enhancing survival rates and quality of life.关键词
遗传代谢性疾病/亲体肝移植/外科手术/儿童Key words
Living Donor Liver Transplantation/Inborn Errors of Metabolism/Surgical Procedures,Operative/Child引用本文复制引用
刘辉,叶力阿曼·加依拉吾,陈治达,王浩宇,哈力穆拉提·胡西塔尔,李万富,高伟,董冲,孙超,阿尔新·哈布丁,古力米热木·买买提江,阿依古再丽·麦麦江..亲体肝移植治疗儿童遗传代谢病的疗效分析[J].临床小儿外科杂志,2025,24(3):220-225,6.基金项目
2023天山英才人才培养计划(TSYC202301A072) （TSYC202301A072）
新疆维吾尔自治区重点研发任务专项项目资助(2023B03018-2) （2023B03018-2）
新疆医科大学第一附属医院2023年青年科研启航专项基金(2023YFY-QKQN-10) 2023 Tianshan Talent Training Program(TSYC202301A072) （2023YFY-QKQN-10）
Special Project of Key Research and Development Tasks of Xinjiang Uygur Autonomous Region(2023B03018-2) （2023B03018-2）
2023 Youth Re-search Initiative Fund of the First Affiliated Hospital of Xinjiang Medical University(2023YFY-QKQN-10) （2023YFY-QKQN-10）
