Long-term impact of aficamten on patient-reported outcome measures in obstructive hypertrophic cardiomyopathy: results from FOREST-HCM
Year: 2026
Type: article
Abstract: AIMS: Treatment with aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM) led to significant improvements in patient-reported outcomes (PROs) in the pivotal SEQUOIA-HCM trial over 24 weeks, but its longer-term impact has not been described. The aim of this study was to describe the long-term effects of aficamten across multiple PROs in FOREST-HCM, an open-label extension study. METHODS AND RESULTS: Participants with oHCM completing an aficamten trial were offered enrollment in FOREST-HCM. The Kansas City Cardiomyopathy Questionnaire (KCCQ), Seattle Angina Questionnaire 7-item (S), EuroQol Five-Dimensional Questionnaire (EQ-5D-5L) index, and visual analogue scale (VAS) were administered at baseline and at weeks 12, 24, 36, and 48. Associations among PROs and clinical markers of oHCM severity were assessed with Spearman correlation coefficients.A total of 172 participants (mean age 60.4 [SD ±13.1] years; 45.3% female) completed 48 weeks of follow up as of August 31, 2024, and were included. Significant improvements in all PROs were observed by week 12 and sustained through week 48. At 48 weeks, mean KCCQ-OSS improved by 18.8 (16.9-20.6) points, (P < 0.001, with the largest gains in the KCCQ Quality of Life domain (25.8 points [23.2-28.5; P < 0.001]). S-Summary Scores increased by 17.2 (15.1-19.4; p < 0.001), and EQ-5D-5L index and VAS scores improved by 0.11 and 12.7 points, respectively (p < 0.001). Improvements in PROs were significantly correlated with important clinical measures of oHCM. CONCLUSIONS: Aficamten resulted in sustained improvements across multiple health status dimensions, which also correlated with clinical markers of oHCM severity.
DOI: 10.1093/ejhf/xuag244
Authors: Shepard D Weiner, Lusha W Liang, Ahmad Masri, Theodore P Abraham, Roberto Barriales-Villa, Robert M Cooper, Perry M Elliott, Alejandro de Feria, Martin S Maron, Artur Oreziak, Sara Saberi, Scott D Solomon, John A Spertus, Albree Tower-Rader, Michael Butzner, Justin Godown, Stephen B Heitner, Daniel L Jacoby, Stuart Kupfer, Xueli Liu, Fady I Malik, Chiara Melloni, Tyrell J Simkins, Jenny Wei, Michael E Nassif, Anjali T Owens
Institutions: Columbia University Irving Medical Center, Oregon Health & Science University, University of California, San Francisco, Centro de Investigación en Red en Enfermedades Cardiovasculares, Complexo Hospitalario Universitario A Coruña, Instituto de Investigación Biomédica de A Coruña, Liverpool John Moores University, University College London, University of Pennsylvania, Lahey Hospital and Medical Center, Lahey Medical Center, Institute of Cardiology, University of Michigan, Michigan Medicine, Brigham and Women's Hospital, Harvard University, Saint Luke's Hospital, University of Missouri–Kansas City, Massachusetts General Hospital, Cytokinetics (United States)
Language: English
Subfield: Cardiology and Cardiovascular Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $5,300
Funder: Cytokinetics
OpenAlex ID: W7172303452