Association Between age or Duration of Diagnosis in Obstructive Hypertrophic Cardiomyopathy and Response to Mavacamten Treatment: Exploratory Analysis of the EXPLORER-HCM Trial

ANDREW WANG, NEAL K. LAKDAWALA, THEODORE P. ABRAHAM, ESTER KIM NILLES, DANIEL M. WOJDYLA, ANJALI TIKU OWENS, RICHARD G. BACH, S. A.R.A. SABERI, A. M.Y. SEHNERT, SHARON CRESCI

Research output: Contribution to journalArticlepeer-review

Abstract

Background and Aims: In patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM), it is unclear if response to cardiac myosin inhibition varies with older age or a longer duration of diagnosis. This study evaluated the response of these subgroups to mavacamten therapy for all primary, secondary and exploratory endpoints in the EXPLORER-HCM trial (ClinicalTrials.gov: NCT03470545). Methods: Patients were stratified by age (≤ 60 vs > 60 years) and duration of HCM diagnosis (≤ 5 vs > 5 years). To estimate treatment differences and evaluate age and diagnosis duration by treatment interaction, analysis of covariance was used to model changes in continuous endpoints, and a generalized linear model was used for binary endpoints. Results: Older patients were more commonly female (53% vs 29%), had lower prevalences of pathogenic/likely pathogenic HCM gene variants (17% vs 36%), lower mean peak oxygen consumption (pVO2) (17.6 vs 21.1 mL/kg/min), and higher mean NT-proBNP levels (817 vs 592 ng/L) but similar NYHA classes and quality-of-life scores. Patients with longer vs shorter diagnosis duration had similar mean ages (59.0 ± 11.6 vs 57.9 ± 12.3 years) but more family histories of HCM (38% vs 16%) and higher mean NT-pro BNP levels (938 ± 118 vs 494 ± 145 ng/mL). No differences were observed in improvement in peak oxygen consumption, NYHA class or patient-reported outcomes among older patients and those with longer durations of diagnosis. Conclusions: In EXPLORER-HCM, mavacamten treatment had a similar benefit for all primary, secondary and exploratory endpoints in patients with symptomatic, obstructive HCM, regardless of age or duration of diagnosis.

Original languageEnglish
JournalJournal of cardiac failure
DOIs
StateAccepted/In press - 2025

Fingerprint

Dive into the research topics of 'Association Between age or Duration of Diagnosis in Obstructive Hypertrophic Cardiomyopathy and Response to Mavacamten Treatment: Exploratory Analysis of the EXPLORER-HCM Trial'. Together they form a unique fingerprint.

Cite this