Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design
Publikation: Bidrag til tidsskrift › Review › Forskning › fagfællebedømt
Standard
Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy : SEQUOIA-HCM Baseline Characteristics and Study Design. / SEQUOIA-HCM Investigators.
I: JACC. Heart failure, Bind 12, Nr. 1, 2024, s. 199-215.Publikation: Bidrag til tidsskrift › Review › Forskning › fagfællebedømt
Harvard
APA
Vancouver
Author
Bibtex
}
RIS
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 -
ID: 378130783