Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design

Caroline J. Coats; Martin S. Maron; SEQUOIA‐HCM Investigators; Theodore P. Abraham; Iacopo Olivotto; Matthew M.Y. Lee; Michael Arad; Nuno Cardim; Chang Sheng Ma; Lubna Choudhury; Hans Dirk Düngen; Pablo Garcia-Pavia; Albert A. Hagège; Gregory D. Lewis; Michelle Michels; Artur Oreziak; Anjali T. Owens; Jacob Tfelt-Hansen; Josef Veselka; Hugh C. Watkins; Stephen B. Heitner; Daniel L. Jacoby; Stuart Kupfer; Fady I. Malik; Lisa Meng; Amy Wohltman; Ahmad Masri

doi:10.1016/j.jchf.2023.10.004

Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design

Caroline J. Coats^*, Martin S. Maron, SEQUOIA‐HCM Investigators, Theodore P. Abraham, Iacopo Olivotto, Matthew M.Y. Lee, Michael Arad, Nuno Cardim, Chang Sheng Ma, Lubna Choudhury, Hans Dirk Düngen, Pablo Garcia-Pavia, Albert A. Hagège, Gregory D. Lewis, Michelle Michels, Artur Oreziak, Anjali T. Owens, Jacob Tfelt-Hansen, Josef Veselka, Hugh C. WatkinsStephen B. Heitner, Daniel L. Jacoby, Stuart Kupfer, Fady I. Malik, Lisa Meng, Amy Wohltman, Ahmad Masri

^*Corresponding author for this work

Cardiology

Research output: Contribution to journal › Review article › Academic › peer-review

20 Citations (Scopus)

9 Downloads (Pure)

Abstract

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).

Original language	English
Pages (from-to)	199-215
Number of pages	17
Journal	JACC: Heart Failure
Volume	12
Issue number	1
DOIs	https://doi.org/10.1016/j.jchf.2023.10.004
Publication status	Published - Jan 2024

Bibliographical note

Access to Document

10.1016/j.jchf.2023.10.004Licence: CC BY

1-s2.0-S2213177923006893-mainFinal published version, 1.3 MBLicence: CC BY

Cite this

Coats, C. J., Maron, M. S., SEQUOIA‐HCM Investigators, Abraham, T. P., Olivotto, I., Lee, M. M. Y., Arad, M., Cardim, N., Ma, C. S., Choudhury, L., Düngen, H. D., Garcia-Pavia, P., Hagège, A. A., Lewis, G. D., Michels, M., Oreziak, A., Owens, A. T., Tfelt-Hansen, J., Veselka, J., ... Masri, A. (2024). Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design. JACC: Heart Failure, 12(1), 199-215. https://doi.org/10.1016/j.jchf.2023.10.004

@article{cc9decf7aef14e50b8c54b5a6290946a,

title = "Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design",

abstract = "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).",

author = "Coats, {Caroline J.} and Maron, {Martin S.} and {SEQUOIA‐HCM Investigators} and Abraham, {Theodore P.} and Iacopo Olivotto and Lee, {Matthew M.Y.} and Michael Arad and Nuno Cardim and Ma, {Chang Sheng} and Lubna Choudhury and D{\"u}ngen, {Hans Dirk} and Pablo Garcia-Pavia and Hag{\`e}ge, {Albert A.} and Lewis, {Gregory D.} and Michelle Michels and Artur Oreziak and Owens, {Anjali T.} and Jacob Tfelt-Hansen and Josef Veselka and Watkins, {Hugh C.} and Heitner, {Stephen B.} and Jacoby, {Daniel L.} and Stuart Kupfer and Malik, {Fady I.} and Lisa Meng and Amy Wohltman and Ahmad Masri",

note = "Publisher Copyright: {\textcopyright} 2024 The Authors",

year = "2024",

month = jan,

doi = "10.1016/j.jchf.2023.10.004",

language = "English",

volume = "12",

pages = "199--215",

journal = "JACC: Heart Failure",

issn = "2213-1779",

publisher = "Elsevier",

number = "1",

}

Coats, CJ, Maron, MS, SEQUOIA‐HCM Investigators, Abraham, TP, Olivotto, I, Lee, MMY, Arad, M, Cardim, N, Ma, CS, Choudhury, L, Düngen, HD, Garcia-Pavia, P, Hagège, AA, Lewis, GD, Michels, M, Oreziak, A, Owens, AT, Tfelt-Hansen, J, Veselka, J, Watkins, HC, Heitner, SB, Jacoby, DL, Kupfer, S, Malik, FI, Meng, L, Wohltman, A & Masri, A 2024, 'Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design', JACC: Heart Failure, vol. 12, no. 1, pp. 199-215. https://doi.org/10.1016/j.jchf.2023.10.004

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 - SEQUOIA‐HCM Investigators

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

PY - 2024/1

Y1 - 2024/1

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).

UR - http://www.scopus.com/inward/record.url?scp=85180435450&partnerID=8YFLogxK

U2 - 10.1016/j.jchf.2023.10.004

DO - 10.1016/j.jchf.2023.10.004

M3 - Review article

C2 - 38032573

AN - SCOPUS:85180435450

SN - 2213-1779

VL - 12

SP - 199

EP - 215

JO - JACC: Heart Failure

JF - JACC: Heart Failure

IS - 1

ER -

Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design

Abstract

Bibliographical note

Access to Document

Other files and links

Fingerprint

Cite this