Evidence map›Paper›PMID 39462184›Full record

ReviewESC heart failure2025

Considerations for drug trials in hypertrophic cardiomyopathy.

John P Farrant, Matthias Schmitt, Anna B Reid, Clifford J Garratt, William G Newman, Aneil Malhotra, Rhys Beynon, Masliza Mahmod, Betty Raman, Robert M Cooper and 8 more

Abstract readReview
In one paragraph

Review in ESC heart failure, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
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

18 authors.

John P FarrantDivision of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, University of Manchester, Oxford Road, Manchester, M13 9PL, UK.ORCID https://orcid.org/0000-0002-9241-2410
Matthias SchmittDivision of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, University of Manchester, Oxford Road, Manchester, M13 9PL, UK.ORCID https://orcid.org/0000-0001-8514-0261
Anna B ReidDivision of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, University of Manchester, Oxford Road, Manchester, M13 9PL, UK.
Clifford J GarrattDivision of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, University of Manchester, Oxford Road, Manchester, M13 9PL, UK.
William G NewmanManchester Centre for Genomic Medicine Manchester University NHS Foundation Trust, Oxford Road, Manchester, M13 9WL, UK.ORCID https://orcid.org/0000-0002-6382-4678
Aneil MalhotraManchester University NHS Foundation Trust, Southmoor Road, Wythenshawe, Manchester, M23 9LT, UK.ORCID https://orcid.org/0000-0002-8670-3764
Rhys BeynonManchester University NHS Foundation Trust, Southmoor Road, Wythenshawe, Manchester, M23 9LT, UK.
Masliza MahmodDivision of Cardiovascular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, OX3 9DU, UK.ORCID https://orcid.org/0000-0002-0601-3510
Betty RamanDivision of Cardiovascular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, OX3 9DU, UK.ORCID https://orcid.org/0000-0002-1239-9608
Robert M CooperLiverpool Heart and Chest Hospital, Thomas Dr, Liverpool, L14 3PE, UK.ORCID https://orcid.org/0000-0001-7482-828X
Dana DawsonSchool of Medicine, University of Aberdeen, Aberdeen, AB25 2ZD, UK.ORCID https://orcid.org/0000-0003-2815-4469
Thomas GreenCardiology Department, Northumbria Healthcare NHS Trust, Northumberland, UK.
Sanjay K PrasadRoyal Brompton and Harefield NHS Foundation Trust, Sydney St, London, SW3 6NP, UK.
Anvesha SinghDepartment of Cardiovascular Sciences, University of Leicester and the NIHR Leicester Biomedical Research Centre, Glenfield Hospital, Groby Road, Leicester, LE3 9QP, UK.ORCID https://orcid.org/0000-0003-1112-3973
Susanna DoddDepartment of Health Data Sciences, Institute of Population Health, Faculty of Health and Life Sciences, University of Liverpool, Block F, Waterhouse Boulevard, 1-5 Brownlow Street, Liverpool, L69 3GL, UK.ORCID https://orcid.org/0000-0003-2851-3337
Hugh WatkinsDivision of Cardiovascular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, OX3 9DU, UK.ORCID https://orcid.org/0000-0002-5287-9016
Stefan NeubauerDivision of Cardiovascular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, OX3 9DU, UK.ORCID https://orcid.org/0000-0001-9017-5645
Christopher A MillerDivision of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, Manchester Academic Health Science Centre, University of Manchester, Oxford Road, Manchester, M13 9PL, UK.

Funding

BHF Centre of Research Excellence, OxfordBritish Heart Foundation AA/18/4/34221British Heart Foundation BBC/F/21/220106CureHeartNIHR Manchester Biomedical Research Centre NIHR203308NIHR Oxford Biomedical Research CentreProgramme Grants for Applied Research NIHR300867Programme Grants for Applied Research NIHR301338Wellcome Career Development Award fellowshipWellcome Trust
6 · The paper itself

Abstract

Hypertrophic cardiomyopathy (HCM) is a heterogeneous condition with potentially serious manifestations. Management has traditionally comprised therapies to palliate symptoms and implantable cardioverter-defibrillators to prevent sudden cardiac death. The need for disease-modifying therapies has been recognized for decades. More recently, an increasing number of novel and repurposed therapies hypothesized to target HCM disease pathways have been evaluated, culminating in the recent regulatory approval of mavacamten, a novel oral myosin inhibitor. HCM poses several unique challenges for clinical trials, which are important to recognize when designing trials and interpreting findings. This manuscript discusses the key considerations in the context of recent and ongoing randomized trials, including the roles of genotype, phenotype and symptom status in patient selection, the evidence base for clinical and mechanistic outcome measurements, trial duration and sample size.

Indexed as

Cardiomyopathy, HypertrophicClinical Trials as TopicDeath, Sudden, CardiacRandomized Controlled Trials as TopicHumansClinical trialsDisease‐modifying therapyHypertrophic cardiomyopathyPatient selectionTrial endpoints

Identifiers

PMID39462184
PMCPMC11911595

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.