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儿童肝移植治疗Alagille综合征的单中心经验

檀玉乐 朱志军 孙丽莹 魏林 曲伟 曾志贵 张海明 刘颖 王君 李宏宇

檀玉乐, 朱志军, 孙丽莹, 等. 儿童肝移植治疗Alagille综合征的单中心经验[J]. 器官移植, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010
引用本文: 檀玉乐, 朱志军, 孙丽莹, 等. 儿童肝移植治疗Alagille综合征的单中心经验[J]. 器官移植, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010
Tan Yule, Zhu Zhijun, Sun Liying, et al. Pediatric liver transplantation for Alagille syndrome: a single-center experience[J]. ORGAN TRANSPLANTATION, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010
Citation: Tan Yule, Zhu Zhijun, Sun Liying, et al. Pediatric liver transplantation for Alagille syndrome: a single-center experience[J]. ORGAN TRANSPLANTATION, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010

儿童肝移植治疗Alagille综合征的单中心经验

doi: 10.3969/j.issn.1674-7445.2022.01.010
基金项目: 

首都卫生发展科研专项 首发2020-1-2024

详细信息
    作者简介:

    檀玉乐,男,1980年生,博士,主治医师,研究方向为肝移植,Email:tanyule007@163.com

    通讯作者:

    朱志军,男,1968年生,博士,主任医师,研究方向为肝移植,Email:zhu-zhijun@outlook.com

  • 中图分类号: R617, R596

Pediatric liver transplantation for Alagille syndrome: a single-center experience

More Information
  • 摘要:   目的  探讨儿童肝移植治疗Alagille综合征(ALGS)的疗效。  方法  收集12例ALGS患儿的临床资料进行回顾性分析,分析ALGS患儿的临床特征和病肝组织病理学特点、肝移植的特点及其术后并发症和随访情况。  结果  12例患儿均存在JAG1基因突变和典型面部特征,黄疸是最常见的首发症状,在出生后7(3,40)d出现。患儿行肝移植时身高和体质量Z评分分别为-2.14(-3.11,-1.83)分和-2.32(-3.12,-1.12)分,5例患儿严重发育迟缓,4例患儿严重营养不良。12例患儿中8例伴有心血管异常。病理学检查结果显示,4例患儿病肝的小叶结构基本保持,8例存在大小不等结节性肝硬化,其中1例病肝检测到单个早期中分化肝细胞癌。3例患儿因误诊为胆道闭锁行葛西手术治疗。8例患儿行活体肝移植,3例患儿行尸体供肝肝移植(包括2例劈离式肝移植和1例尸体全肝移植),1例患儿行多米诺肝移植(供肝来自枫糖尿病患者)。在随访30.0(24.5,41.7)个月时,患儿和移植物存活率均为100%。在随访过程中,患儿身高和体质量Z评分分别为-1.24(-2.11,0.60)分和-0.83(-1.65,-0.43)分,表明患儿术后生长和发育情况明显改善。  结论  肝移植是治疗肝硬化失代偿期和严重瘙痒伴生活质量差的ALGS患儿的有效方法。伴随心血管异常的ALGS患儿,术前应进行详细评估,必要时联合儿科心脏病专家进行治疗。

     

  • 表  1  12例ALGS患儿的临床资料

    Table  1.   Clinical data of 12 children with ALGS

    例序 性别 月龄(月) 病肝组织病理学特点 心脏或血管异常 眼睛异常 椎体异常 其他 肝移植类型 GRWR(%) 术后并发症 随访时间(月)
    1 17 胆汁淤积、肝硬化、小胆管缺乏 - 蝴蝶椎 高甘油三酯血症 活体肝移植(左外叶) 3.8 肠穿孔 29
    2 16 胆汁淤积、肝硬化、小胆管增生 三尖瓣轻度反流 - 高甘油三酯血症 活体肝移植(左外叶) 2.5 28
    3 108 小胆管缺乏、肝硬化 活体肝移植(左外叶) 1.6 腹腔出血 26
    4 14 胆汁淤积、肝硬化、中分化肝细胞癌 动脉导管未闭、卵圆孔未闭、肺动脉狭窄 - 高甘油三酯血症 活体肝移植(左外叶) 3.2 24
    5 36 胆汁淤积 卵圆孔未闭、肺动脉狭窄 - 高甘油三酯血症 活体肝移植(左外叶) 1.6 19
    6 12 胆汁淤积、小胆管缺乏 肺动脉狭窄 尸体供肝肝移植 4.2 移植肝动脉闭塞 48
    7 11 肝硬化、小胆管缺乏 室间隔缺损(膜周部)、动脉导管未闭、卵圆孔未闭 - 蝴蝶椎 黄色瘤、高甘油三酯血症 活体肝移植(左外叶) 2.9 43
    8 72 小胆管缺乏 室间隔缺损、动脉导管未闭 - 高甘油三酯血症、黄色瘤 活体肝移植(左外叶) 2.1 38
    9 24 小胆管缺乏、胆汁淤积 主动脉瓣二叶瓣畸形、主动脉瓣狭窄 - 高甘油三酯血症 活体肝移植(左外叶) 3.0 31
    10 10 小胆管缺乏、胆汁淤积、肝硬化 动脉导管未闭、肺动脉狭窄 - 多米诺供肝肝移植(枫糖尿病) 2.7 12
    11 132 胆汁淤积、肝硬化、小胆管增生 - 蝴蝶椎 劈离式肝移植(左半肝) 1.5 42
    12 16 胆汁淤积、肝硬化、小胆管增生 蝴蝶椎 高甘油三酯血症 劈离式肝移植(左外叶) 3.1 41
    注:①GRWR为移植物与受者质量比。
    ②-为无数据。
    下载: 导出CSV
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  • 收稿日期:  2021-08-22
  • 网络出版日期:  2022-01-12
  • 刊出日期:  2022-01-15

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