{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,9]],"date-time":"2026-04-09T14:00:35Z","timestamp":1775743235761,"version":"3.50.1"},"reference-count":62,"publisher":"BMJ","issue":"1","license":[{"start":{"date-parts":[[2025,1,22]],"date-time":"2025-01-22T00:00:00Z","timestamp":1737504000000},"content-version":"unspecified","delay-in-days":21,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["RMD Open"],"accepted":{"date-parts":[[2024,12,27]]},"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>To identify the best evidence on the efficacy of treatment interventions for inclusion body myositis (IBM) and to describe their safety.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusions<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1136\/rmdopen-2024-005176","type":"journal-article","created":{"date-parts":[[2025,1,22]],"date-time":"2025-01-22T22:35:46Z","timestamp":1737585346000},"page":"e005176","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":4,"title":["Efficacy and safety of pharmacological treatments in inclusion body myositis: a systematic review"],"prefix":"10.1136","volume":"11","clinical-trial-number":[{"clinical-trial-number":"nct04789070","registry":"10.18810\/clinical-trials-gov"},{"clinical-trial-number":"nct05721573","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0557-2377","authenticated-orcid":false,"given":"Eduardo Jos\u00e9 Ferreira","family":"Santos","sequence":"first","affiliation":[{"name":"Polytechnic Institute of Viseu, Viseu, Portugal"},{"name":"Health Sciences Research Unit: Nursing (UICISA: E), Nursing School of Coimbra (ESEnfC), Coimbra, Portugal"},{"name":"Department of Neuromuscular Diseases, University College London, London, UK"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9394-1103","authenticated-orcid":false,"given":"Bayram","family":"Farisogullari","sequence":"additional","affiliation":[{"name":"Department of Neuromuscular Diseases, University College London, London, UK"},{"name":"Division of Rheumatology, Department of Internal Medicine, Hacettepe University, Ankara, Turkey"}]},{"given":"Nicholas","family":"Yapp","sequence":"additional","affiliation":[{"name":"University of Oxford, Oxford, UK"}]},{"given":"Hermaleigh","family":"Townsley","sequence":"additional","affiliation":[{"name":"Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK"}]},{"given":"Pedro","family":"Sousa","sequence":"additional","affiliation":[{"name":"Health Sciences Research Unit: Nursing (UICISA: E), Nursing School of Coimbra (ESEnfC), Coimbra, Portugal"},{"name":"Center for Innovative Care and Health Technology (ciTechCare), Polytechnic of Leiria, Leiria, Portugal"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8411-7972","authenticated-orcid":false,"given":"Pedro M","family":"Machado","sequence":"additional","affiliation":[{"name":"Department of Neuromuscular Diseases, University College London, London, UK"},{"name":"National Institute for Health Research University College London Hospitals Biomedical Research Centre, University College London Hospitals NHS Fountation Trust, London, UK"}]}],"member":"239","published-online":{"date-parts":[[2025,1,22]]},"reference":[{"key":"2025090312510749000_11.1.e005176.1","article-title":"Treatment for inclusion body myositis","volume":"7","author":"Rose","year":"2015","journal-title":"Cochrane Database Syst Rev"},{"key":"2025090312510749000_11.1.e005176.2","doi-asserted-by":"crossref","unstructured":"Sangha G , Yao B , Lunn D , et al . Longitudinal observational study investigating outcome measures for clinical trials in inclusion body myositis. J Neurol Neurosurg Psychiatry 2021:jnnp-2020-325141. doi:10.1136\/jnnp-2020-325141","DOI":"10.1136\/jnnp-2020-325141"},{"key":"2025090312510749000_11.1.e005176.3","doi-asserted-by":"crossref","DOI":"10.1007\/s11926-014-0477-9","article-title":"Ongoing developments in sporadic inclusion body myositis","volume":"16","author":"Machado","year":"2014","journal-title":"Curr Rheumatol Rep"},{"key":"2025090312510749000_11.1.e005176.4","doi-asserted-by":"crossref","first-page":"e115","DOI":"10.1016\/S2665-9913(23)00322-3","article-title":"Idiopathic inflammatory myopathies: current insights and future frontiers","volume":"6","author":"Connolly","year":"2024","journal-title":"Lancet Rheumatol"},{"key":"2025090312510749000_11.1.e005176.5","doi-asserted-by":"crossref","first-page":"e40","DOI":"10.1016\/S2665-9913(20)30280-0","article-title":"Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial","volume":"3","author":"Benveniste","year":"2021","journal-title":"Lancet Rheumatol"},{"key":"2025090312510749000_11.1.e005176.6","doi-asserted-by":"crossref","first-page":"900","DOI":"10.1016\/S1474-4422(23)00275-2","article-title":"Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial","volume":"22","author":"Machado","year":"2023","journal-title":"Lancet Neurol"},{"key":"2025090312510749000_11.1.e005176.7","doi-asserted-by":"crossref","first-page":"3176","DOI":"10.1093\/brain\/awr213","article-title":"Long-term observational study of sporadic inclusion body myositis","volume":"134","author":"Benveniste","year":"2011","journal-title":"Brain (Bacau)"},{"key":"2025090312510749000_11.1.e005176.8","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1016\/S1474-4422(19)30200-5","article-title":"Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial","volume":"18","author":"Hanna","year":"2019","journal-title":"Lancet Neurol"},{"key":"2025090312510749000_11.1.e005176.9","doi-asserted-by":"publisher","DOI":"10.1038\/s41584-019-0186-x"},{"key":"2025090312510749000_11.1.e005176.10","doi-asserted-by":"crossref","first-page":"127","DOI":"10.3233\/JND-160198","article-title":"A Systematic Review and Meta-Analysis of Prevalence Studies of Sporadic Inclusion Body Myositis","volume":"4","author":"Callan","year":"2017","journal-title":"J Neuromuscul Dis"},{"key":"2025090312510749000_11.1.e005176.11","first-page":"e2653","article-title":"Epidemiology and Natural History of Inclusion Body Myositis: A 40-Year Population-Based Study","volume":"96","author":"Shelly","year":"2021","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.12","doi-asserted-by":"crossref","first-page":"1679","DOI":"10.1080\/03007995.2018.1486294","article-title":"Health care costs and comorbidities for patients with inclusion body myositis","volume":"34","author":"Keshishian","year":"2018","journal-title":"Curr Med Res Opin"},{"key":"2025090312510749000_11.1.e005176.13","doi-asserted-by":"crossref","first-page":"101761","DOI":"10.1016\/j.berh.2022.101761","article-title":"Inclusion body myositis: The interplay between ageing, muscle degeneration and autoimmunity","volume":"36","author":"McLeish","year":"2022","journal-title":"Best Pract Res Clin Rheumatol"},{"key":"2025090312510749000_11.1.e005176.14","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-018-0658-8"},{"key":"2025090312510749000_11.1.e005176.15","doi-asserted-by":"crossref","first-page":"1090","DOI":"10.1002\/art.40045","article-title":"Immune-Array Analysis in Sporadic Inclusion Body Myositis Reveals HLA-DRB1 Amino Acid Heterogeneity Across the Myositis Spectrum","volume":"69","author":"Rothwell","year":"2017","journal-title":"Arthritis Rheumatol"},{"key":"2025090312510749000_11.1.e005176.16","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23840"},{"key":"2025090312510749000_11.1.e005176.17","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23822"},{"key":"2025090312510749000_11.1.e005176.18","doi-asserted-by":"crossref","first-page":"384","DOI":"10.55563\/clinexprheumatol\/r625rm","article-title":"Diagnostic and prognostic value of anti-cN1A antibodies in inclusion body myositis","volume":"40","author":"Salam","year":"2022","journal-title":"Clin Exp Rheumatol"},{"key":"2025090312510749000_11.1.e005176.19","doi-asserted-by":"crossref","first-page":"2590","DOI":"10.1093\/brain\/awz207","article-title":"Highly differentiated cytotoxic T cells in inclusion body myositis","volume":"142","author":"Greenberg","year":"2019","journal-title":"Brain (Bacau)"},{"key":"2025090312510749000_11.1.e005176.20","doi-asserted-by":"crossref","unstructured":"Higgins JPT , Thomas J , Chandler J , et al . Cochrane Handbook for Systematic Reviews of Interventions Version 6.0 (Updated July 2019). In: Cochrane, Ed. Cochrane, 2019.","DOI":"10.1002\/9781119536604"},{"key":"2025090312510749000_11.1.e005176.21","doi-asserted-by":"crossref","DOI":"10.1136\/bmj.n71","article-title":"The PRISMA 2020 statement: an updated guideline for reporting systematic reviews","volume":"372","author":"Page","year":"2021","journal-title":"BMJ"},{"key":"2025090312510749000_11.1.e005176.22","article-title":"Interventions for dysphagia in long-term, progressive muscle disease","volume":"2","author":"Jones","year":"2016","journal-title":"Cochrane Database Syst Rev"},{"key":"2025090312510749000_11.1.e005176.23","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410380504"},{"key":"2025090312510749000_11.1.e005176.24","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.03.014"},{"key":"2025090312510749000_11.1.e005176.25","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.003"},{"key":"2025090312510749000_11.1.e005176.26","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.08.007"},{"key":"2025090312510749000_11.1.e005176.27","unstructured":"European Union . Directive 2001\/83\/EC of the European parliament and of the council of 6 November 2001 on the community code relating to medicinal products for human use. Official journal of the European communities no l-311\/67 of 28 November 2001. 2001."},{"key":"2025090312510749000_11.1.e005176.28","unstructured":"OCEBM Levels of Evidence Working Group . The oxford levels of evidence. Oxford centre for evidence-based medicine (updated in 2012). 2009. Available: https:\/\/www.cebm.ox.ac.uk\/resources\/levels-of-evidence\/ocebm-levels-of-evidence"},{"key":"2025090312510749000_11.1.e005176.29","doi-asserted-by":"crossref","DOI":"10.1136\/bmj.d5928","article-title":"The Cochrane Collaboration\u2019s tool for assessing risk of bias in randomised trials","volume":"343","author":"Higgins","year":"2011","journal-title":"BMJ"},{"key":"2025090312510749000_11.1.e005176.30","doi-asserted-by":"crossref","DOI":"10.12707\/RV21077","article-title":"How to measure and explore heterogeneity in a meta-analysis: Key methodological strategies","volume":"6","author":"Santos","year":"2022","journal-title":"Rev Enferm Ref"},{"key":"2025090312510749000_11.1.e005176.31","doi-asserted-by":"publisher","DOI":"10.1097\/XEB.0000000000000065"},{"key":"2025090312510749000_11.1.e005176.32","unstructured":"Tufanaru C , Munn Z , Aromataris E , et al . Chapter 3: systematic reviews of effectiveness. In: Aromataris E , Munn Z , eds. Joanna Briggs Institute Reviewer\u2019s Manual. The Joanna Briggs Institute, 2017."},{"key":"2025090312510749000_11.1.e005176.33","doi-asserted-by":"publisher","DOI":"10.1002\/ana.10121"},{"key":"2025090312510749000_11.1.e005176.34","doi-asserted-by":"crossref","first-page":"2239","DOI":"10.1212\/WNL.0000000000001070","article-title":"Treatment of sporadic inclusion body myositis with bimagrumab","volume":"83","author":"Amato","year":"2014","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.35","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1212\/WNL.56.3.323","article-title":"A controlled study of intravenous immunoglobulin combined with prednisone in the treatment of IBM","volume":"56","author":"Dalakas","year":"2001","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.36","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1212\/01.WNL.0000134675.98525.79","article-title":"Randomized pilot trial of high-dose \u03b2INF-1a in patients with inclusion body myositis","volume":"63","year":"2004","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.37","first-page":"1566","article-title":"Randomized pilot trial of \u03b2INF1a (Avonex) in patients with inclusion body myositis","volume":"57","year":"2001","journal-title":"Neurol (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.38","doi-asserted-by":"crossref","first-page":"1081","DOI":"10.1212\/WNL.58.7.1081","article-title":"A pilot randomized trial of oxandrolone in inclusion body myositis","volume":"58","author":"Rutkove","year":"2002","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.39","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aad4583","article-title":"Targeting protein homeostasis in sporadic inclusion body myositis","volume":"8","author":"Ahmed","year":"2016","journal-title":"Sci Transl Med"},{"key":"2025090312510749000_11.1.e005176.40","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1212\/WNL.48.3.712","article-title":"Treatment of inclusion-body myositis with IVIg: a double-blind, placebo-controlled study","volume":"48","author":"Dalakas","year":"1997","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.41","doi-asserted-by":"publisher","DOI":"10.1007\/s004150050005"},{"key":"2025090312510749000_11.1.e005176.42","doi-asserted-by":"crossref","first-page":"260","DOI":"10.1212\/01.WNL.0000071852.27182.C7","article-title":"Anti-T-lymphocyte globulin treatment in inclusion body myositis: a randomized pilot study","volume":"61","author":"Lindberg","year":"2003","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.43","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1097\/00005792-199307000-00002","article-title":"The treatment of inclusion body myositis: a retrospective review and a randomized, prospective trial of immunosuppressive therapy","volume":"72","author":"Leff","year":"1993","journal-title":"Medicine (Baltimore)"},{"key":"2025090312510749000_11.1.e005176.44","first-page":"370","article-title":"Current status of clinical outcome measures in inclusion body myositis: a systematised review","volume":"41","author":"Roy","year":"2023","journal-title":"Clin Exp Rheumatol"},{"key":"2025090312510749000_11.1.e005176.45","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1097\/WCO.0000000000001095","article-title":"Inclusion body myositis: evolving concepts","volume":"35","author":"Perez-Rosendahl","year":"2022","journal-title":"Curr Opin Neurol"},{"key":"2025090312510749000_11.1.e005176.46","doi-asserted-by":"publisher","DOI":"10.55563\/clinexprheumatol\/0q2voe"},{"key":"2025090312510749000_11.1.e005176.47","doi-asserted-by":"crossref","unstructured":"Salam S , Symonds T , Doll H , et al . Measurement properties of the Inclusion Body Myositis Functional Rating Scale. J Neurol Neurosurg Psychiatry 2024:jnnp-2024-333617. doi:10.1136\/jnnp-2024-333617","DOI":"10.1136\/jnnp-2024-333617"},{"key":"2025090312510749000_11.1.e005176.48","doi-asserted-by":"crossref","DOI":"10.1212\/CPJ.0000000000200168","article-title":"Study to Assess Content Validity and Interrater and Intrarater Reliability of the Inclusion Body Myositis Functional Rating Scale","volume":"13","author":"Symonds","year":"2023","journal-title":"Neurol Clin Pract"},{"key":"2025090312510749000_11.1.e005176.49","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1016\/j.nmd.2024.03.001","article-title":"272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16\u201318 June 2023, Hoofddorp, The Netherlands","volume":"37","author":"Lilleker","year":"2024","journal-title":"Neuromuscul Disord"},{"key":"2025090312510749000_11.1.e005176.50","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1097\/BOR.0000000000001060","article-title":"Inclusion body myositis: an update","volume":"37","author":"Anderson","year":"2025","journal-title":"Curr Opin Rheumatol"},{"key":"2025090312510749000_11.1.e005176.51","doi-asserted-by":"crossref","first-page":"1108","DOI":"10.1002\/jcsm.13451","article-title":"Effect of sirolimus on muscle in inclusion body myositis observed with magnetic resonance imaging and spectroscopy","volume":"15","author":"Reyngoudt","year":"2024","journal-title":"J Cachexia Sarcopenia Muscle"},{"key":"2025090312510749000_11.1.e005176.52","first-page":"e865","article-title":"Longitudinal Changes in MRI Muscle Morphometry and Composition in People With Inclusion Body Myositis","volume":"99","author":"Laurent","year":"2022","journal-title":"Neurology (ECronicon)"},{"key":"2025090312510749000_11.1.e005176.53","doi-asserted-by":"crossref","first-page":"997","DOI":"10.3233\/JND-240053","article-title":"Quantitative muscle MRI in sporadic inclusion body myositis (sIBM): A prospective cohort study","volume":"11","author":"Schlaffke","year":"2024","journal-title":"J Neuromuscul Dis"},{"key":"2025090312510749000_11.1.e005176.54","doi-asserted-by":"crossref","unstructured":"Allameen NA , Salam S , Reddy V , et al . Inclusion body myositis and immunosenescence: current evidence and future perspectives. Rheumatol (Oxford) 2024:keae614. doi:10.1093\/rheumatology\/keae614","DOI":"10.1093\/rheumatology\/keae614"},{"key":"2025090312510749000_11.1.e005176.55","doi-asserted-by":"publisher","DOI":"10.1034\/j.1600-065X.2003.00051.x"},{"key":"2025090312510749000_11.1.e005176.56","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2002-07-2103"},{"key":"2025090312510749000_11.1.e005176.57","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1007\/s11357-009-9100-9","article-title":"KLRG1--more than a marker for T cell senescence","volume":"31","author":"Henson","year":"2009","journal-title":"Age (Dordr)"},{"key":"2025090312510749000_11.1.e005176.58","doi-asserted-by":"crossref","first-page":"1008","DOI":"10.1136\/annrheumdis-2022-eular.2141","article-title":"POS1342\u2005DEPLETION OF KLRG1+ T CELLS IN A FIRST-IN-HUMAN CLINICAL TRIAL OF ABC008 IN INCLUSION BODY MYOSITIS (IBM)","volume":"81","author":"Goel","year":"2022","journal-title":"Ann Rheum Dis"},{"key":"2025090312510749000_11.1.e005176.59","first-page":"340","article-title":"Two emerging phenotypes of atypical inclusion body myositis: illustrative cases","volume":"41","author":"Salam","year":"2023","journal-title":"Clin Exp Rheumatol"},{"key":"2025090312510749000_11.1.e005176.60","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-022-11459-3"},{"key":"2025090312510749000_11.1.e005176.61","doi-asserted-by":"publisher","DOI":"10.1097\/WCO.0000000000000129"},{"key":"2025090312510749000_11.1.e005176.62","first-page":"445","article-title":"Phenotypic spectrum of inclusion body myositis","volume":"42","author":"Roy","year":"2024","journal-title":"Clin Exp Rheumatol"}],"container-title":["RMD Open"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/syndication.highwire.org\/content\/doi\/10.1136\/rmdopen-2024-005176","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,3]],"date-time":"2025-09-03T19:51:19Z","timestamp":1756929079000},"score":1,"resource":{"primary":{"URL":"https:\/\/rmdopen.bmj.com\/lookup\/doi\/10.1136\/rmdopen-2024-005176"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1]]},"references-count":62,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2025,1,22]]},"published-print":{"date-parts":[[2025,1]]}},"alternative-id":["10.1136\/rmdopen-2024-005176"],"URL":"https:\/\/doi.org\/10.1136\/rmdopen-2024-005176","relation":{},"ISSN":["2056-5933"],"issn-type":[{"value":"2056-5933","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1]]}}}