Current treatment decisions in cardiac transthyretin amyloidosis: a multicentre analysis.

Abstract

Background: The efficacy of transthyretin stabilisation in cardiac transthyretin amyloidosis (ATTR-CM) has been demonstrated in a clinical trial setting, but little is known about treatment decision-making in the real world. Particularly, initiating or discontinuing specific therapy is challenging in early and advanced disease. We evaluated current decision pathways for tafamidis in ATTR-CM.

Methods: This multicentre retrospective study included consecutive patients from 15 tertiary centres in Germany in whom ATTR-CM was newly diagnosed between January and June 2024, as well as patients, in whom tafamidis treatment was discontinued during this period.

Results: Out of 516 patients with newly established ATTR-CM included in the present analysis, tafamidis was initiated in 414 (80%). The 99 patients without recommendation for tafamidis were older (p = 0.002), had a higher amyloidosis disease stage (NAC score), worse NYHA class (both p < 0.001), and higher NT-proBNP levels (p = 0.002) compared to those with tafamidis initiation. During the same observation period, tafamidis therapy was discontinued in 28 ATTR-CM patients. Treatment decisions were mainly taken by an interdisciplinary board (73% of centres). The most frequent reasons for not starting or stopping tafamidis were 'frailty' (47%/61%) and 'life expectancy or comorbidity' (38%/43%), respectively.

Conclusions: In this multicentre analysis, treatment with tafamidis was initiated in about 80% of patients with newly diagnosed ATTR-CM. In most centres, treatment decisions were made by an interdisciplinary board, and the reasons for treatment decisions were similar across centres. Due to the lack of consensus criteria, our data may help to standardise decision pathways for ATTR-CM.