TY - JOUR

T1 - Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy

T2 - SEQUOIA-HCM Baseline Characteristics and Study Design

AU - Coats, Caroline J

AU - Maron, Martin S

AU - Abraham, Theodore P

AU - Olivotto, Iacopo

AU - Lee, Matthew M Y

AU - Arad, Michael

AU - Cardim, Nuno

AU - Ma, Chang-Sheng

AU - Choudhury, Lubna

AU - Düngen, Hans-Dirk

AU - Garcia-Pavia, Pablo

AU - Hagège, Albert A

AU - Lewis, Gregory D

AU - Michels, Michelle

AU - Oreziak, Artur

AU - Owens, Anjali T

AU - Tfelt-Hansen, Jacob

AU - Veselka, Josef

AU - Watkins, Hugh C

AU - Heitner, Stephen B

AU - Jacoby, Daniel L

AU - Kupfer, Stuart

AU - Malik, Fady I

AU - Meng, Lisa

AU - Wohltman, Amy

AU - Masri, Ahmad

AU - SEQUOIA-HCM Investigators

N1 - Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.

PY - 2024

Y1 - 2024

N2 - Patients with obstructive hypertrophic cardiomyopathy (oHCM) have increased risk of arrhythmia, stroke, heart failure, and sudden death. Contemporary management of oHCM has decreased annual hospitalization and mortality rates, yet patients have worsening health-related quality of life due to impaired exercise capacity and persistent residual symptoms. Here we consider the design of clinical trials evaluating potential oHCM therapies in the context of SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM). This large, phase 3 trial is now fully enrolled (N = 282). Baseline characteristics reflect an ethnically diverse population with characteristics typical of patients encountered clinically with substantial functional and symptom burden. The study will assess the effect of aficamten vs placebo, in addition to standard-of-care medications, on functional capacity and symptoms over 24 weeks. Future clinical trials could model the approach in SEQUOIA-HCM to evaluate the effect of potential therapies on the burden of oHCM. (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM [SEQUOIA-HCM]; NCT05186818).

AB - Patients with obstructive hypertrophic cardiomyopathy (oHCM) have increased risk of arrhythmia, stroke, heart failure, and sudden death. Contemporary management of oHCM has decreased annual hospitalization and mortality rates, yet patients have worsening health-related quality of life due to impaired exercise capacity and persistent residual symptoms. Here we consider the design of clinical trials evaluating potential oHCM therapies in the context of SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM). This large, phase 3 trial is now fully enrolled (N = 282). Baseline characteristics reflect an ethnically diverse population with characteristics typical of patients encountered clinically with substantial functional and symptom burden. The study will assess the effect of aficamten vs placebo, in addition to standard-of-care medications, on functional capacity and symptoms over 24 weeks. Future clinical trials could model the approach in SEQUOIA-HCM to evaluate the effect of potential therapies on the burden of oHCM. (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM [SEQUOIA-HCM]; NCT05186818).

U2 - 10.1016/j.jchf.2023.10.004

DO - 10.1016/j.jchf.2023.10.004

M3 - Review

C2 - 38032573

VL - 12

SP - 199

EP - 215

JO - J A C C: Heart Failure

JF - J A C C: Heart Failure

SN - 2213-1779

IS - 1

ER -