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Publications

Journal Article Randomized Controlled Trial Ppublish 1 citation

PubMed: 42544039 DOI: 10.1093/ejhf/xuag244

Long-term impact of aficamten on patient-reported outcome measures in obstructive hypertrophic cardiomyopathy: results from FOREST-HCM.

Eur J Heart Fail 28(4):819-830

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

Affiliations

  • Division of Cardiology, Department of Medicine, Columbia University Irving Medical Center, 173 Fort Washington Avenue, New York, NY 10032, USA.
  • Division of Cardiology, Department of Medicine, Oregon Health and Science University, Portland, OR, USA.
  • Division of Cardiology, Department of Medicine, University of California San Francisco, San Francisco, CA, USA.
  • Inherited Cardiovascular Diseases Unit, Complejo Hospitalario Universitario de A Coruña, INIBIC, CIBERCV-ISCIII, A Coruña, Spain.
  • Division of Cardiovascular Medicine, Liverpool John Moores University, Liverpool, UK.
  • Inherited Cardiovascular Disease Unit, Barts Heart Centre and University College London, London, UK.
  • Division of Cardiology, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Division of Cardiovascular Medicine, Lahey Hospital and Medical Center, Burlington, MA, USA.
  • National Institute of Cardiology, Warsaw, Poland.
  • Division of Cardiology, Department of Medicine, University of Michigan Medical Center, Ann Arbor, MI, USA.
  • Division of Cardiology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
  • University of Missouri-Kansas City's Healthcare Institute for Innovations in Quality and Saint Luke's Mid America Heart Institute, Kansas City, MO, USA.
  • Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Boston, MA, USA.
  • Cytokinetics, Incorporated, South San Francisco, CA, USA.

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 enrolment in FOREST-HCM. The Kansas City Cardiomyopathy Questionnaire (KCCQ), Seattle Angina Questionnaire 7-item, EuroQol Five-Dimensional Questionnaire index, and visual analogue scale 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 31 August 2024, and were included. Significant improvements in all PROs were observed by week 12 and sustained through week 48. At 48 weeks, mean Kansas City Cardiomyopathy Questionnaire-Overall Summary Score improved by 18.8 (16.9-20.6) points, (P < .001), with the largest gains in the KCCQ Quality of Life domain (25.8 points [23.2-28.5; P < .001]). Seattle Angina Questionnaire 7-item -Summary Scores increased by 17.2 (15.1-19.4; P < .001), and EuroQol Five-Dimensional Questionnaire index and visual analogue scale scores improved by 0.11 and 12.7 points, respectively (P < .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.

CLINICAL TRIALS REGISTRATION

NCT04848506.

MeSH

Heart Failure, Cardiomyopathy, Hypertrophic, Patient Reported Outcome Measures, Forests, Cardiomyopathies