Defining Heart Failure Endpoint in ATTR Amyloidosis Clinical Trials: Time for an Update?

This viewpoint highlights the critical need for a standardized definition of heart failure (HF) endpoints in clinical trials specific to transthyretin cardiac amyloidosis (ATTR-CA). Current endpoint definitions vary significantly across standard HF and amyloidosis trials. The objective of this viewpoint is to support clinical trialists by providing practical considerations on how to better define HF events when designing amyloidosis clinical trials, as well as during the adjudication process. We suggest defining HF events based on three major components: acute hospitalizations or inpatient visits for HF, urgent outpatient HF visits, and recurrent outpatient visits. By refining these definitions, we aim to create a framework that accurately reflects the complexities of HF in amyloidosis and promotes consistency in endpoint definitions or HF event adjudication.