Real-World Utilization Patterns, Safety, and Efficacy of Tafamidis in Patients With Hereditary Transthyretin Amyloidosis in Japan.

ABSTRACT:

Purpose

Patients with hereditary transthyretin (ATTRv) amyloidosis experience progressive degeneration of the somatic and peripheral nervous system that can impair ambulation, autonomy, and quality of life (QOL). Tafamidis meglumine (tafamidis) is the first pharmacotherapy approved to slow the progression of peripheral neurological impairment in ATTRv amyloidosis and was well tolerated and efficacious in clinical trials; however, longer-term safety in Japanese patients with ATTRv amyloidosis has not been fully elucidated. Consequently, the present study was conducted to understand the safety and efficacy of long-term use (up to 156 weeks) of tafamidis meglumine under postmarketing conditions in Japan.

Methods

This single-arm observational study (conducted from November 2013

SUBMITTER: Konishi H 

PROVIDER: S-EPMC12148832 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature