Recent Advances Have Improved Prognosis in ATTR-CM Patients, with Ahmad Masri, MD, MS

New research is shedding light on the evolving risk profile and baseline characteristics among patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in the US.

Presented at the American Heart Association (AHA) Annual Scientific Sessions 2024, , results of the study provide evidence of a changing risk profile and outcomes, which investigators suggest is indicative of the effects disease-modifying treatments, such as tafamidis, acoramidis, and vutrisiran, and diagnostic advances have had on earlier diagnosis of patients with ATTR-CM.^1,2,3

To better understand temporal trends in baseline risks of patients enrolled in trials for ATTR-CM, investigators designed their systematic review to leverage data from a search of the Embase, MEDLINE, and CENTRAL databases as well as conference websites on November 23, 2023, for peer-reviewed articles and abstracts. For the purpose of analysis investigator included randomized and single-arm clinical trials examining treatments for ATTR-CM. The primary interest of investigators was comparing baseline characteristics and outcomes in patients treated with placebo across the studies.¹

The review returned evidence suggesting several baseline characteristics were comparable across studies, including sex, age, race/ethnicity, genotype, and troponin I levels. However, notable differences were observed for characteristics such as improved NYHA class, with fewer patients classified as NYHA Class III, as well as improvements in NT-proBNP and eGFR levels in more recent studies.¹

Additionally, assessments of outcomes in the placebo groups suggested all-cause mortality (ACM) rates at 12 months decreased from 9% in ATTR-ACT (2013–2015 enrollment) to 6.9% in ATTRibute-CM (2019–2020 enrollment) and 5.6% in APOLLO-B (2019–2021 enrollment). At 30 months, ACM rates fell from 42.9% in ATTR-ACT to 25.7% in ATTRibute-CM.¹

For more on this study and how it might influence trials in the future, check out our interview with principal investigator Ahmad Masri, MD, MS, director of the Hypertrophic Cardiomyopathy Center at Oregon Health and Science University, from the floor at AHA 2024.

Relevant disclosures for Masri include Cytokinetics, Bristol Myers Squibb, BridgeBio, Pfizer, Ionis, Lexicon, Attralus, Alnylam, Haya, Alexion, BioMarin, and AstraZeneca.

References:

1. Masri A. Evolving Baseline Risk in Patients With Transthyretin Amyloid Cardiomyopathy: A Systematic Literature Review of Clinical Trials. Presented at the American Heart Association (AHA) Scientific Sessions 2024. Chicago, Illinois. November 16-18, 2024.
2. Campbell P. FDA accepts NDA submission for Acoramidis in attr-cm. HCP Live. February 6, 2024. Accessed November 21, 2024. https://www.hcplive.com/view/fda-accepts-nda-submission-for-acoramidis-in-attr-cm.
3. Iapoce C. Alnylam submits sNDA for vutrisiran for treatment of attr-cm. HCP Live. October 11, 2024. Accessed November 21, 2024. https://www.hcplive.com/view/alnylam-submits-snda-for-vutrisiran-for-treatment-of-attr-cm.