肝移植治疗酸血症:生存和代谢结果
Xiaochen Bo1, Yiqing Zhang, Jiahao Ge
1Department of Liver Surgery, Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Hepatology communications
|January 16, 2026
概括
肝移植 (LT) 显著改善了患有酸血症 (PA),一种严重的代谢障碍的儿童的结果. 活体供体肝移植有效减少代谢浪费,提高儿科PA患者的生活质量.
科学领域:
- 代谢障碍 代谢障碍 代谢障碍
- 儿科外科手术 儿科外科手术
- 器官移植 器官移植 器官移植
背景情况:
- propionic acidemia (PA) 是一种严重的遗传代谢障碍,导致多器官损伤.
- 目前对PA的管理策略的有效性有限.
- 肝移植 (LT) 是一种潜在的治疗方法,但大规模的证据很少.
研究的目的:
- 评估接受肝移植的PA儿科患者的临床结果.
- 评估活体供体肝移植在治疗PA方面的有效性.
- 分析诊断到移植间隔对患者结果的影响.
主要方法:
- 从2017年9月到2023年10月接受LT的39名儿科PA患者的回顾性分析.
- 包括人口,外科,生化和临床数据.
- 基于诊断到移植间隔的比较分析 (<20个月 vs. ≥20个月).
主要成果:
- 97.4%的患者接受了活体供体肝移植.
- 在LT后观察到关键代谢标志物 (propionylcarnitine/acetylcarnitine比率,尿中的甲基酸盐,3-基酸) 的显著减少.
- 临床症状显著改善,包括运动延迟,代谢酸症,超血症和食困难 (所有p<0.001).
- 患者和移植的5年生存率达到97.4%.
结论:
- 活体肝移植,特别是活体供体肝移植,是儿科PA的有效治疗方法.
- 肝移植减少了代谢废物,改善了代谢稳定性,提高了生活质量.
- 对于由于PA而导致代谢不稳定的儿科患者来说,LT是一种可行的治疗选择.
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