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Efficacy and safety of pharmacological treatments in inclusion body myositis: a systematic review

2025-01-22Journal article Open access
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datacite.subject.fosCiências Médicas::Ciências da Saúde
datacite.subject.sdg03:Saúde de Qualidade
datacite.subject.sdg17:Parcerias para a Implementação dos Objetivos
dc.contributor.authorSantos, Eduardo José Ferreira
dc.contributor.authorFarisogullari, Bayram
dc.contributor.authorYapp, Nicholas
dc.contributor.authorTownsley, Hermaleigh
dc.contributor.authorSousa, Pedro
dc.contributor.authorMachado, Pedro
dc.contributor.editorCombe, Bernard
dc.date.accessioned2025-05-26T15:01:55Z
dc.date.available2025-05-26T15:01:55Z
dc.date.issued2025-01-22
dc.descriptionAcknowledgements: This paper will contribute towards a Post Doctoral Program for EJFS. This paper was presented as an abstract at EULAR 2024 (Santos E, et al. POS1314: Efficacy of pharmacological treatment in inclusion body myositis: a systematic review. Annals of the Rheumatic Diseases 2024;83:633)
dc.descriptionSupplemental material: This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.
dc.description.abstractObjective: To identify the best evidence on the efficacy of treatment interventions for inclusion body myositis (IBM) and to describe their safety. Methods: Systematic review of randomised controlled trials (RCTs) of pharmacological treatments of adults with IBM, conducted according to the Cochrane Handbook, updating a previous Cochrane review. The search strategy was run on Cochrane Neuromuscular Disease Group Specialized Register, CENTRAL, MEDLINE and EMBASE, ClinicalTrials.gov and the WHO International Clinical Trials Registry Platform. Assessment of risk of bias, data extraction and synthesis were performed independently by two reviewers. Data pooled in statistical meta-analyses, if possible. Results: From a total of 487 records, 48 were selected for full-text review, 14 fulfilled the inclusion criteria, but only 2 RCTs were included in meta-analyses due to clinical heterogeneity (different drug interventions or dosages). Treatments included various immunosuppressive and immunomodulatory agents, alongside interventions modulating muscle growth and protein homoeostasis. Efficacy was assessed across multiple outcomes, namely muscle strength, physical function, mobility and muscle trophicity. Trials of methotrexate (MTX), intravenous immunoglobulin, interferon beta-1a and MTX, MTX and anti-T-lymphocyte immunoglobulin, oxandrolone, MTX and azathioprine, bimagrumab, arimoclomol, and sirolimus provided low-quality to high-quality evidence of having no effect on the progression of IBM. Conclusions: Drug interventions for IBM were not effective for most of the outcomes of interest. We observed inconsistency of outcome measures across trials. More RCTs are needed, of adequate size and duration, and using a standardised set of outcome measures.eng
dc.identifier.citationSantos EJF, Farisogullari B, Yapp N, et al. Efficacy and safety of pharmacological treatments in inclusion body myositis: a systematic review. RMD Open 2025;11:e005176. doi:10.1136/ rmdopen-2024-005176
dc.identifier.doi10.1136/rmdopen-2024-005176
dc.identifier.issn2056-5933
dc.identifier.urihttp://hdl.handle.net/10400.8/12989
dc.language.isoeng
dc.peerreviewedyes
dc.publisherBMJ
dc.relation.hasversionhttps://rmdopen.bmj.com/content/11/1/e005176
dc.relation.ispartofRMD Open
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/
dc.subjectAntirheumatic Agents
dc.subjectAutoimmunity
dc.subjectBiological Therapy
dc.subjectOutcome Assessment
dc.subjectHealth Care
dc.subjectTreatment
dc.titleEfficacy and safety of pharmacological treatments in inclusion body myositis: a systematic revieweng
dc.typejournal article
dspace.entity.typePublication
oaire.citation.endPage9
oaire.citation.issue1
oaire.citation.startPage1
oaire.citation.titleRMD Open: Rheumatic and Musculoskeletal Diseases
oaire.citation.volume11
oaire.versionhttp://purl.org/coar/version/c_970fb48d4fbd8a85
person.familyNameSousa
person.familyNameMachado
person.givenNamePedro
person.givenNamePedro
person.identifier.orcid0000-0002-6313-532X
person.identifier.orcid0000-0002-8411-7972
relation.isAuthorOfPublication64c18128-48dc-421e-95f8-ad42f9b0d227
relation.isAuthorOfPublicationad353c9c-66fb-4baa-ab7c-b797c69d2167
relation.isAuthorOfPublication.latestForDiscovery64c18128-48dc-421e-95f8-ad42f9b0d227

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