针对遗传代谢疾病的儿科肝移植 - - 当前挑战
Roshni Vara1,2, Nedim Hadzic2
1Department of Metabolic Medicine, Evelina Children's Hospital, London, UK.
概括
肝移植 (LT) 已经显著发展,为儿科患者提供超过90%的存活率. 它对于治疗遗传代谢疾病 (IMD) 越来越重要,提供治愈或纠正的选择.
科学领域:
- 肝病学和移植外科手术
- 儿科胃肠病学和肝病学
- 医学遗传学和新陈代谢
背景情况:
- 儿科肝移植 (LT) 早期面临挑战,但从20世纪70年代开始,外科手术技术和免疫抑制显著改善.
- 目前,选择性儿科LT的10年生存率超过90%,这表明该领域取得了重大进展.
- 遗传代谢性疾病 (IMD) 现在是儿科LT的主要征兆,占病例的25-30%.
研究的目的:
- 审查肝移植在治疗儿童遗传代谢疾病中的演变作用.
- 突出当前的概念,挑战和争议,围绕着IMDs的LT.
- 检查LT在各种IMD中的治愈和纠正潜力.
主要方法:
- 关于儿科LT技术和免疫抑制疗法的历史数据和进展的审查.
- 分析自20世纪70年代末以来,在IMD中越来越多地使用LT.
- 检查特定的IMDs当LT是指示的,包括1型铁血病和尿素循环障碍.
主要成果:
- 儿科LT结局有了显著的改善,长期移植和患者存活率高.
- 对于肝脏受限缺陷的IMD来说,LT已被确立为治愈性治疗,对于肝脏外参与的IMD来说,它是部分纠正性的.
- 对IMD的LT的范围已经扩大,并强调了早期的干预.
结论:
- 肝移植是患有遗传代谢疾病的儿科患者的关键治疗选择.
- 持续的进步正在提高对IMDs的LT的有效性和安全性.
- 随着症状的不断扩大和对早期干预的重视,需要对儿科中 IMD 的 LT 的挑战和争议进行持续的研究.
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