Evidence map›Paper›PMID 39611272›Full record

ArticleCirculation. Genomic and precision medicine2024

Patient Perceptions of Emerging Gene Therapies for Arrhythmogenic Right Ventricular Cardiomyopathy.

Emma M Schopp, Leonore Okwara, Crystal Tichnell, Amy Turriff, Brittney Murray, Andreas S Barth, Hugh Calkins, Leila Jamal, Cynthia A James

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Circulation. Genomic and precision medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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

9 authors.

Emma M SchoppCenter for Precision Health Research, National Human Genome Research Institute, Bethesda, MD (E.M.S., A.T.).ORCID 0000-0003-0419-049X
Leonore OkwaraDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).ORCID 0000-0001-6948-833X
Crystal TichnellDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).
Amy TurriffCenter for Precision Health Research, National Human Genome Research Institute, Bethesda, MD (E.M.S., A.T.).ORCID 0000-0002-8065-7445
Brittney MurrayDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).
Andreas S BarthDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).ORCID 0000-0001-5275-8049
Hugh CalkinsDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).ORCID 0000-0002-9262-9433
Leila JamalGenetics Branch, Center for Cancer Research, National Cancer Institute (L.J.).ORCID 0000-0003-1132-2411
Cynthia A JamesDivision of Cardiology, Johns Hopkins University, Baltimore, MD (L.O., C.T., B.M., A.S.B., H.C., C.A.J.).ORCID 0000-0001-8040-4600

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundNo disease-specific therapy currently exists for arrhythmogenic right ventricular cardiomyopathy (ARVC), a progressive cardiogenetic condition conferring elevated risk for ventricular arrhythmias, heart failure, and sudden cardiac death. Emerging gene therapies have the potential to fill this gap. However, little is known about how adults with ARVC, or any other inherited cardiomyopathy or arrhythmia syndrome, appraise the risks and benefits of gene therapy research and which considerations may influence their decisions about clinical trial participation.

methodsTwenty adults with clinically diagnosed and gene-positive ARVC participated in semi-structured interviews that explored perceptions of gene therapy and hypothetical decision-making for gene therapy clinical trial participation. Interview transcripts were qualitatively coded and analyzed.

resultsParticipants expressed enthusiasm for gene therapy with varied levels of personal interest in trial participation. Although clinical severity appeared to be associated with an elevated interest in early trial participation, participants anticipated weighing both personal and trial-specific factors including life stage, trial stage, risks, benefits, participation burden, study leadership, and anticipated cost of future gene therapy. Adaptation to living with ARVC and involvement in the ARVC patient community were also relevant to decision-making about trial participation. Potential ethical concerns included unquestioning trust in clinical teams collaborating on industry-led trials and vulnerability of those recently diagnosed or with high perceived severity of ARVC symptoms.

conclusionsSeveral characteristics of the individual and trial warrant consideration during the informed consent process. Insights from this study may affect trial planning and communication with participants who have inherited cardiac conditions.

Indexed as

Arrhythmogenic Right Ventricular DysplasiaGenetic TherapyAdultDecision MakingFemaleHumansMaleMiddle Agedcardiomyopathiesclinical trialsdecision makinggenetic therapypatients

Identifiers

PMID39611272
PMCPMC11651351

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.