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

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.