Evidence map›Paper›PMID 39217450›Full record

ArticleEuropean heart journal2024

Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy.

Pablo Garcia-Pavia, Artur Oręziak, Ahmad Masri, Roberto Barriales-Villa, Theodore P Abraham, Anjali T Owens, Morten K Jensen, Wojciech Wojakowski, Tim Seidler, Albert Hagege and 9 more

2 registry-linked trialsAbstract readMulticenter Study
In one paragraph

Article in European heart journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 52 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
52citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03470545 phase3completednot on this map

A Randomized, Double Blind, Placebo Controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy

TypeinterventionalSponsorMyoKardia, Inc.Ran2018 to 2020Enrolled251ConditionsObstructive Hypertrophic CardiomyopathyArmsmavacamten, Placebo
NCT03723655 phase2 / phase3completednot on this map

A Long-Term Safety Extension Study of Mavacamten (MYK-461) in Adults With Hypertrophic Cardiomyopathy Who Have Completed the MAVERICK-HCM (MYK-461-006) or EXPLORER-HCM (MYK-461-005) Trials (MAVA-LTE)

TypeinterventionalSponsorBristol-Myers SquibbRan2018 to 2026Enrolled314ConditionsHypertrophic Cardiomyopathy, Obstructive Hypertrophic Cardiomyopathy, Non-obstructive Hypertrophic CardiomyopathyArmsmavacamten
3 · Its place in the literature

Who cites it

52 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
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  15. [Update on hypertrophic cardiomyopathy].Innere Medizin (Heidelberg, Germany) · 2026
    Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors.

Pablo Garcia-PaviaDepartment of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, Manuel de Falla 2, 28222, Madrid, Spain.ORCID 0000-0002-5470-2257
Artur OręziakDepartment of Arrhythmia, National Institute of Cardiology, Warsaw, Poland.ORCID 0000-0003-3318-3643
Ahmad MasriDivision of Cardiovascular Medicine, School of Medicine, Oregon Health & Science University, Portland, OR, USA.ORCID 0000-0002-6390-6526
Roberto Barriales-VillaInherited Cardiovascular Diseases Unit, Complexo Hospitalario Universitario A Coruña, INIBIC, CIBERCV (ISCIII), A Coruña, Spain.ORCID 0000-0002-6721-3487
Theodore P AbrahamSchool of Medicine, University of California San Francisco, San Francisco, CA, USA.
Anjali T OwensCenter for Inherited Cardiac Disease, Division of Cardiovascular Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA.ORCID 0000-0002-9669-8495
Morten K JensenSchool of Medicine, Aarhus University Hospital, Aarhus, Denmark.ORCID 0000-0003-1144-4603
Wojciech WojakowskiChief Division of Cardiology and Structural Heart Diseases, Medical University of Silesia, Katowice, Poland.ORCID 0000-0002-3681-5207
Tim SeidlerDepartment of Cardiology and Pulmonology, University of Göttingen, Göttingen, Germany.ORCID 0000-0002-6158-5087
Albert HagegeAP-HP, Hôpital Européen Georges Pompidou, Paris, France.ORCID 0000-0003-4685-6077
Neal K LakdawalaCardiovascular Medicine, Brigham and Women's Hospital, Boston, MA, USA.
Andrew WangCardiology, Duke University Hospital, Durham, NC, USA.ORCID 0000-0001-8729-0933
Matthew T WheelerCardiovascular Medicine, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0001-8721-3022
Lubna ChoudhuryCardiovascular Medicine, Northwestern University, Feinberg School of Medicine, Chicago, IL, USA.ORCID 0000-0003-4039-8490
Ganesh BalaratnamBristol Myers Squibb, Princeton, NJ, USA.
Ashish ShahBristol Myers Squibb, Princeton, NJ, USA.
Shawna FoxBristol Myers Squibb, Princeton, NJ, USA.
Sheila M HegdeCardiovascular Medicine, Brigham and Women's Hospital, Boston, MA, USA.ORCID 0000-0001-8157-8899
Iacopo OlivottoCardiology Unit, Meyer Children's Hospital, IRCCS, Florence, Italy.ORCID 0000-0003-1751-9266

Funding

Bristol Myers Squibb
6 · The paper itself

Abstract

BACKGROUND AND

aimsLong-term safety and efficacy of mavacamten in patients with obstructive hypertrophic cardiomyopathy (HCM) are unknown. MAVA-LTE (NCT03723655) is an ongoing, 5-year, open-label extension study designed to evaluate the long-term effects of mavacamten.

methodsParticipants from EXPLORER-HCM (NCT03470545) could enrol in MAVA-LTE upon study completion.

resultsAt the latest data cut-off, 211 (91.3%) of the 231 patients originally enrolled in MAVA-LTE still received mavacamten. Median (range) time on study was 166.1 (6.0-228.1) weeks; 185 (80.1%) and 99 (42.9%) patients had completed the Week 156 and 180 visits, respectively. Sustained reductions from baseline to Week 180 occurred in left ventricular outflow tract gradients [mean (standard deviation): resting, -40.3 (32.7) mmHg; Valsalva, -55.3 (33.7) mmHg], N-terminal pro B-type natriuretic peptide [median (interquartile range): -562 (-1162.5, -209) ng/L], and EQ-5D-5L score [mean (standard deviation): 0.09 (0.17)]. Mean left ventricular ejection fraction (LVEF) decreased from 73.9% (baseline) to 66.6% (Week 24) and 63.9% (Week 180). At Week 180, 74 (77.9%) of the 95 patients improved by at least one New York Heart Association class from baseline. Over 739 patient-years exposure, 20 patients (8.7%; exposure-adjusted incidence: 2.77/100 patient-years) experienced 22 transient reductions in LVEF to <50% resulting in temporary treatment interruption (all recovered LVEF of ≥50%). Five (2.2%) patients died (all considered unrelated to mavacamten).

conclusionsLong-term mavacamten treatment resulted in sustained improvements in cardiac function and symptoms in patients with obstructive HCM, with no new safety concerns identified. Transient, reversible reductions in LVEF were observed in a small proportion of patients during long-term follow-up.

Indexed as

Cardiomyopathy, HypertrophicAdultAgedBenzylaminesFemaleHumansMaleMiddle AgedNatriuretic Peptide, BrainQuality of LifeStroke VolumeTreatment OutcomeUracilBenzylaminesMYK-461Natriuretic Peptide, BrainUracilEfficacyLong-term outcomesMavacamtenMAVA-LTE (EXPLORER cohort)Obstructive hypertrophic cardiomyopathySafety

Identifiers

PMID39217450
PMCPMC11646600

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.