METHODS: The article provides a comprehensive review about the successes, hopes, and challenges related to changing indications for LTx in children based on the literature review and our own experience. Retrospective review of the indications for LTx at a tertiary referral pediatric hospital was also presented.
CONCLUSIONS: In the context of the new therapies that have emerged, the need for LTx has decreased in patients with chronic hepatitis B and C infection and tyrosinemia type 1. In primary hyperoxaluria type 1, new RNAi-based therapy has eliminated the requirement for LTx (both isolated or combined). There is a hope that introduction of ileal bile acid transporter (IBAT) blockers reduces the need for LTx in patients with Alagille syndrome or progressive familial intrahepatic cholestasis. The number of children qualified for LTx with urea cycle disorders (UCDs) as a prophylaxis of neurodevelopmental impairment is increasing.
方法:这篇文章提供了关于成功的全面回顾,希望,根据文献综述和我们自己的经验,与改变儿童LTx适应症有关的挑战。还对三级转诊儿科医院的LTx适应症进行了回顾性审查。
结论:在已经出现的新疗法的背景下,慢性乙型和丙型肝炎感染和酪氨酸血症1型的患者对LTx的需求下降.在原发性高草酸尿症1型中,新的基于RNAi的疗法消除了对LTx(分离的或组合的)的需求。希望引入回肠胆汁酸转运蛋白(IBAT)阻滞剂可以减少Alagille综合征或进行性家族性肝内胆汁淤积患者对LTx的需求。有资格接受具有尿素循环障碍(UCD)的LTx作为预防神经发育障碍的儿童数量正在增加。