Why carry out this study?

Tafamidis has been shown to be effective in delaying disease progression among patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN), but very little is known on whether it impacts mortality.

This report represents the first evaluation of the effect of tafamidis on mortality in Val30Met and non-Val30Met patients with ATTR-PN that uses data from clinical trials where patients received up to 8.5 years of treatment.

What was learned from the study?

Over the course of 8–9 years, there were very few deaths among patients with ATTR-PN treated with tafamidis.

This finding is potentially significant given the severely shortened life span of patients with ATTR-PN and suggests that tafamidis may confer a survival benefit.

Notwithstanding study limitations, the current analysis offers an important evaluation of patients treated with tafamidis in clinical trials.