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

Area of Science:

• Cardiology
• Clinical Trials
• Amyloidosis Research

Background:

• Standard heart failure (HF) and transthyretin cardiac amyloidosis (ATTR-CA) clinical trials currently lack standardized HF endpoint definitions.
• This variability complicates trial design, data interpretation, and comparison across studies.

Purpose of the Study:

• To propose a standardized framework for defining HF endpoints in ATTR-CA clinical trials.
• To provide practical guidance for clinical trialists in designing and adjudicating HF events in amyloidosis studies.

Main Methods:

• This viewpoint analyzes current endpoint definitions in HF and ATTR-CA trials.
• It proposes a refined definition for HF events based on three key components: acute hospitalizations, urgent outpatient visits, and recurrent outpatient visits.

Main Results:

• Current HF endpoint definitions are inconsistent across trials for transthyretin cardiac amyloidosis (ATTR-CA).
• A proposed framework includes acute hospitalizations, urgent outpatient visits, and recurrent outpatient visits for HF event definition.

Conclusions:

• Standardized HF endpoint definitions are essential for consistent and accurate clinical trial outcomes in ATTR-CA.
• Implementing refined definitions will improve the reliability of HF event adjudication and data interpretation in amyloidosis research.