Impact of disease-modifying drugs in patients with transthyretin amyloidosis after liver transplantation: a systematic review

IF 3.6 2区医学 Q2 IMMUNOLOGY

Transplantation Reviews Pub Date : 2025-08-22 DOI:10.1016/j.trre.2025.100960

Christiane Santo, Cristhian Romero, Bruno Vaz Kerges Bueno, Andre Dabarian, Fabio Fernandes

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引用次数: 0

Abstract

Background

Orthotopic liver transplant (OLT) was the first approved treatment for hereditary transthyretin amyloidosis (ATTRv). However, some patients continue to deteriorate due to ongoing wild-type TTR deposition and residual synthesis from extrahepatic sources. In recent years, disease-modifying therapies including TTR stabilizers (e.g., Tafamidis) and gene-silencing agents (e.g., Patisiran) have emerged, but their role in post-OLT patients remains unclear due to their exclusion from most clinical trials.

Methods

A systematic search was conducted in PubMed, Cochrane, and Embase (up to June 2025) using terms related to transthyretin amyloidosis, liver transplantation, and disease-modifying therapies. The objective was to evaluate clinical benefits and safety of these agents in symptomatic ATTRv patients after OLT.

Results

Disease-modifying therapies showed potential benefits in post-OLT ATTR patients. A total of 39 patients treated with tafamidis, inotersen, or patisiran were analyzed. Neurological improvements, including autonomic symptoms, NIS score, and quality of life, were based on 3 case reports and 32 patients from observational studies. Cardiovascular results were from 4 case reports, and biomarker findings from 3 case reports.

Conclusions

Disease-modifying therapies may offer clinical benefits in post-OLT ATTRv patients. However, robust prospective studies and randomized trials are needed to confirm efficacy and ensure safety in this population.

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改善疾病药物对肝移植后转甲状腺蛋白淀粉样变患者的影响：系统综述

背景：原位肝移植（OLT）是首个被批准治疗遗传性甲状腺转蛋白淀粉样变性（ATTRv）的方法。然而，由于持续的野生型TTR沉积和肝外来源的残留合成，一些患者继续恶化。近年来，包括TTR稳定剂（如Tafamidis）和基因沉默剂（如Patisiran）在内的疾病改善疗法已经出现，但由于它们被排除在大多数临床试验之外，它们在olt后患者中的作用尚不清楚。方法系统检索PubMed、Cochrane和Embase（截至2025年6月），检索与转甲状腺蛋白淀粉样变性、肝移植和疾病改善疗法相关的术语。目的是评估这些药物在OLT后有症状的ATTRv患者中的临床益处和安全性。结果疾病修饰疗法在olt后ATTR患者中显示出潜在的益处。总共分析了39例接受他法米底斯、intertersen或patisiran治疗的患者。神经系统的改善，包括自主神经症状、NIS评分和生活质量，是基于观察性研究的3例病例报告和32例患者。心血管结果来自4例报告，生物标志物结果来自3例报告。结论疾病改良治疗可为olt后ATTRv患者提供临床益处。然而，需要强有力的前瞻性研究和随机试验来确认其在该人群中的有效性和安全性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Transplantation Reviews IMMUNOLOGY-TRANSPLANTATION

CiteScore

7.50

自引率

2.50%

发文量

审稿时长

29 days

期刊介绍： Transplantation Reviews contains state-of-the-art review articles on both clinical and experimental transplantation. The journal features invited articles by authorities in immunology, transplantation medicine and surgery.