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Review
. 2025 Aug 1;8(8):CD015854.
doi: 10.1002/14651858.CD015854.

Targeted immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies

Joost Raaphorst  1 , Nicola J Gullick  2   3   4 , Farhad Shokraneh  5   6   7   8   9 , Ruth Brassington  10 , Minoesch Min  11 , Saadia S Ali  12 , Patrick A Gordon  12   13
Affiliations
Review

Targeted immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies

Joost Raaphorst et al. Cochrane Database Syst Rev. .

Abstract

Background: Idiopathic inflammatory myopathies (IIM) are autoimmune-mediated inflammatory disorders of skeletal muscles with non-muscle involvement in some people. Treatment of IIM represents an area of unmet need; a previous Cochrane review (2012) found little or no evidence to guide treatment. Since then, potentially promising treatments targeting B and T cells and complement inhibitors have been investigated.

Objectives: To assess the effects (benefits and harms) of targeted immunosuppressant and immunomodulatory treatments for the idiopathic inflammatory myopathies: dermatomyositis (DM, including juvenile dermatomyositis (JDM) and amyopathic dermatomyositis), immune-mediated necrotising myopathy (IMNM), anti-synthetase syndrome (ASS), overlap-myositis (OM), polymyositis (PM) and cancer-related myositis.

Search methods: We searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, Embase and clinical trial registers until February 2023. We intended to check references and citations, and contact experts to identify additional studies, but lacked the resources.

Selection criteria: We included randomised controlled trials (RCTs) or quasi-RCTs of targeted immunosuppressive and immunomodulatory therapies in adults and children with IIM. Primary outcomes were improvement of function or disability and improvement of muscle strength. Secondary outcomes were achievement of definitions of improvement, cumulative corticosteroid dose, change in skin disease activity, serious adverse event and withdrawals for lack of benefit or adverse events. Our preferred follow-up was six months, although we accepted three months.

Data collection and analysis: We followed standard Cochrane methodology. To assess risk of bias we used the domain-based Cochrane tool (RoB 1). We used fixed-effect models, and, when needed, random-effects models for meta-analysis. We created summary of findings tables for any comparison for which data were available, but chose in advance to prioritise comparisons of rituximab, abatacept or complement inhibitors with placebo, no treatment or standard care. We assessed the certainty of evidence using GRADE.

Main results: We included 16 studies (830 participants). All studies were at risk of bias (10/16 high risk in at least one domain; four studies with unclear risk in ≥ 2 domains judged as high risk). Selective reporting was the most frequent reason for high risk of bias (37%). None of the treatments assessed showed moderate or high-certainty evidence of response compared to placebo for any of the primary or secondary outcomes. Improvement of function or disability For rituximab, function or disability improvement was not reported separately. Abatacept may have little or no effect on disability measured as change on the Health Assessment Questionnaire Disability Index (HAQ-DI) (range 0 to 3, lower scores better) (mean difference (MD) -0.14, 95% confidence interval (CI) -0.29 to 0.02; 2 RCTs, 147 participants; low certainty). For complement inhibitors, the evidence for change on the HAQ-DI is very uncertain (MD -0.15, 95% CI -0.61 to 0.32; 1 RCT, 26 participants; very low certainty). Improvement of muscle strength Muscle strength was not reported separately in the rituximab study. Abatacept may have little or no effect on muscle strength (Manual Muscle Test-8 (MMT8): range 0 to 150 bilateral, 0 to 80 unilateral; higher scores better) (MD 3.6, 95% CI -0.15 to 7.35; 2 RCTs, number of participants unclear; low certainty). For complement inhibitors, there was no clear difference in muscle strength (proximal muscle strength score, range 0 to 140, 140 equates to full strength) (MD 3.89, 95% CI -6.17 to 13.95; 1 RCT, 25 participants; very low certainty). Achievement of definitions of improvement International Myositis Assessment and Clinical Studies Group definitions of improvement (IMACS DOI) In the rituximab study, the evidence for the effect on the IMACS DOI is very uncertain and could favour rituximab or placebo (risk ratio (RR) 0.72, 95% CI 0.39 to 1.34; 1 RCT, 200 participants; very low certainty). Response was measured at eight weeks (shorter than our preferred time point) and was considered indirect. Abatacept may improve achievement of IMACS DOI after three to six months (RR 1.42, 95% CI 1.02 to 1.98; 2 RCTs, 167 participants; low certainty). Myositis Response Criteria Total Improvement Score (TIS) Achievement of moderate or major TIS was reported for abatacept but could favour abatacept or placebo (RR 1.12, 95% CI 0.81 to 1.57; 1 RCT, 120 participants). TIS minimal improvement was reported for complement inhibitors and could favour treatment or placebo (RR 1.09, 95% CI 0.51 to 2.31; 1 RCT, 25 participants; very low certainty). Cumulative corticosteroid dose Cumulative steroid dose was not measured or reported separately in the selected studies. Change in skin disease activity Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) was not measured in the selected studies. Serious adverse event We were unable to extract serious adverse event data for the randomised period of the rituximab study. For abatacept, the evidence was very uncertain because of study limitations and serious imprecision (RR 0.97, 95% CI 0.25 to 3.75; 2 RCTs, 167 participants; very low certainty). For complement inhibitors, the evidence was very uncertain (RR 0.18, 95% CI 0.01 to 3.11; 2 RCTs, 40 participants; very low certainty). Withdrawals for either lack of benefit or adverse events For rituximab, the evidence was very uncertain (RR 1.47, 95% CI 0.25 to 8.59; 1 RCT, 190 participants; very low certainty). The evidence was very uncertain for abatacept (RR 0.63, 95% CI 0.19 to 2.14; 2 RCTs, 167 participants; very low certainty). For complement inhibitors, one study reported no withdrawals (27 participants) and the other did not provide data (very low certainty).

Authors' conclusions: The evidence for the use of rituximab, abatacept and complement inhibitors in IIM is largely uncertain. Abatacept may improve achievement of IMACS DOI at three or six months, although the evidence is of low certainty. More research is needed to investigate the benefits and harms of targeted immunosuppressive and immunomodulatory therapies in IIM. Ideally, studies should be sufficiently powered to ensure detection of effects in subgroups (e.g. IMNM, DM, ASS). Because of the rarity of IIM, international collaborative efforts should be encouraged to embark on multicentre trials.

Trial registration: ClinicalTrials.gov NCT04988087.

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Conflict of interest statement

JR: is PI of an ongoing investigator‐initiated study on IVIg in IIM (Time is Muscle) at Amsterdam University Medical Centre (Amsterdam UMC, formerly AMC) funded by Prinses Beatrix Spierfonds (Dutch non‐profit fund). Initially, the study‐related costs for home care nurses were co‐funded by Sanquin Foundation (Dutch blood bank), by means of Sanquin Plasma Products (SPP), both non‐profit organisations. From May 2022, due to changes at Sanquin, a for‐profit entity, Prothya Biosolutions, took over this element of the funding. This funding is to AMC Medical Research, rather than departmental, and is used solely for the purpose of the study. JR and his department have not received financial compensation from SPP or Prothya Biosolutions. JR works as a neurologist involved in the care of patients with neuromuscular disorders, including myositis at the Amsterdam UMC, the Netherlands. He has been sub‐investigator of the ProDERM study (not included in this review), and is sub‐investigator of two ongoing studies: Alkivia (Argenx; NCT05523167) and Valor (Priovant; NCT05437263).

NG: declares clinical trial contracts to her institution from Izana Bioscience, AstraZeneca, Eli Lilly, Janssen and Novartis, none for studies relevant to this review. She has received honoraria for lectures and educational events from Abbvie, Celgene, Janssen, Eli Lilly, Novartis and UCB. She has received support for conference attendance and travel from Celgene, Janssen, Eli Lilly, Novartis and UCB and payment for Advisory Boards relating to the treatment of psoriatic arthritis and axial spondyloarthropathy from Abbvie, Janssen, Novartis and UCB. Writing assistance for papers relating to clinical trials in psoriatic arthritis has been supported by Abbvie, Janssen and Novartis. NG is a consultant physician involved in the care of patients with inflammatory myositis at the University Hospital Coventry & Warwickshire. NG was an investigator for the ARTEMIS study (of abatacept in myositis), included in this review, funded in the UK by a grant from Bristol‐Myers Squibb (BMS) and the Myositis Support Group. BMS provided the study drug; the study was initiated, conducted and performed independently of BMS. NG did not make study eligibility decisions about, extract data from, carry out the risk of bias assessment for or perform GRADE assessments for the ARTEMIS study.

FS: no known conflicts of interest. He is the acting Information Specialist for Cochrane Neuromuscular, Cochrane Schizophrenia, Cochrane Gut and Cochrane Heart, Director at Systematic Review Consultants Ltd, Evidence Synthesis Manager at the University of Oxford, and Senior Research Associate at the University of Bristol. He holds an honorary assistant professorship from the University of Nottingham and an honorary lectureship from University College London.

RB: has no known conflicts of interest. She was Managing Editor of Cochrane Neuromuscular until October 2023. She withdrew from an editorial role for this work upon joining the author team.

MM: no known conflicts of interest. MM worked as a physician‐researcher at Amsterdam UMC, the Netherlands, besides her work as a physician assistant in the Emergency Department.

SA: SA is a sub‐investigator for two ongoing studies, Argenx (NCT05523167) and MYOJAK. She does not have control or access to the institutional grants for these trials. She was funded to attend the EULAR e‐congress in 2023 by BMS. SA's institution has received a grant on her behalf from the charity Myositis UK. SA is a clinical fellow involved in the care of patients with inflammatory myositis at King's College Hospital.

PG: PG's institution has received funding for eight studies relevant to this review. One was a study funded by various non‐commercial grant‐giving bodies (Chung 2007), one study was funded in the UK by Bristol Myers Squibb and the Myositis Support Group (ARTEMIS, NCT01315938) and one excluded study by the Arthritis Research Campaign (SELAM, ISRCTN40085050). One study was a commercial trial funded by Corbus Pharmaceuticals. Inc (DETERMINE, NCT03813160), and four are ongoing, funded by Eli Lilly (MYOJAK, NCT04208464), Alexion Pharmaceuticals (NCT04999020), Argenx (NCT05523167) and Galapagos NV (Galarisso, NCT05695950). PG's institution has received a grant from Corbus Pharmaceuticals. PG had or has control of or access to the institutional income for the ARTEMIS, DETERMINE, NCT04999020, NCT05523167, NCT05695950 and MYOJAK trials and the Corbus Pharmaceuticals grant. He is the chief investigator in the UK and primary investigator at his institute for the commercial POETYK SLE study (NCT05620407), which is not relevant to this review but is funded by Bristol Myers Squibb. He received personal payments for an Eli Lilly International Systemic Lupus Erythematosus Advisory Board in December 2020, a speaker's honorarium from UCB in 2021, and performed consultancy work with Galapagos from 2023. He is a member of the steering committee for the Galapagos‐funded study NCT05695950. He was funded to attend the EULAR e‐congress in 2020 by AbbVie and the EULAR congress in 2023 by Celltrion. He was due to be an investigator for the study NCT02612857, a commercial trial funded by Idera Pharmaceuticals Inc., and was an author on an abstract presenting the protocol but had to withdraw from the study in 2017 due to personal illness. PG did not make study eligibility decisions about, extract data from, carry out the risk of bias assessment for or perform GRADE assessments for any of these studies for this review. He is due to be an investigator in a Phase 1/2, Open‐Label Study to Evaluate the Safety and Efficacy of Autologous CD19‐specific Chimeric Antigen Receptor T cells (CABA‐201) in Subjects with Active Idiopathic Inflammatory Myopathy (NCT06154252).

References

References to studies included in this review

ARTEMIS {published data only (unpublished sought but not used)}
    1. EUCTR2009-015957-20-CZ. Abatacept treatment in polymyositis and dermatomyositis - ARTEMIS. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2009-015957-20-CZ 2010.
    1. EUCTR2009-015957-20-SE. Abatacept treatment in polymyositis and dermatomyositis - ARTEMIS. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2009-015957-20-SE 2010.
    1. Lundberg IE, Tjarnlund A, Tang Q, Wick C, Dastmalchi M, Mann HF, et al. Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomized, treatment delayed-start trial. Arthritis & Rheumatology 2015;67(S9):Abstract No. 2361.
    1. NCT01315938. Abatacept treatment in polymyositis and dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT01315938 2011.
    1. Tang Q, Ramskold D, Krystufkova O, Mann HF, Wick C, Dastmalchi M, et al. Effect of CTLA4-Ig (abatacept) treatment on T cells and B cells in peripheral blood of patients with polymyositis and dermatomyositis. Scandinavian Journal of Immunology 2019;89(1):e12732. - PubMed
DETERMINE {published data only (unpublished sought but not used)}
    1. Dan J, Sprow G, Concha J, Kodali N, Diaz D, Vazquez T, et al. Sensitivity of three skin-specific efficacy outcomes to detect patient and physician-reported improvement in overall skin disease in dermatomyositis. Arthritis & Rheumatology 2022;74:397-9.
    1. EUCTR2018-003273-10-DE. A study of safety and efficacy of lenabasum in dermatomyositis patients. https://clinicaltrialsregister.eu/ctr-search/trial/2018-003273-10/DE 2018.
    1. EUCTR2018-003273-10-GB. A study of safety and efficacy of lenabasum in dermatomyositis patients. https://clinicaltrialsregister.eu/ctr-search/trial/2018-003273-10/GB 2018.
    1. EUCTR2018-003273-10-HU. A study of safety and efficacy of lenabasum in dermatomyositis patients. https://clinicaltrialsregister.eu/ctr-search/trial/2018-003273-10/HU 2019.
    1. JapicCTI-194904. JBT101-DM-002 A phase 3 safety and efficacy study of lenabasum in dermatomyositis subjects. https://rctportal.niph.go.jp/en/detail?trial_id=JapicCTI-194904 2019.
EUCTR2012‐005772‐34 {published data only}
    1. EUCTR2012-005772-34-DE. A placebo-controlled, proof-of concept study of the efficacy of gevokizumab subcutaneously over 24 weeks in the treatment of patients with polymyositis, dermatomyositis or necrotizing autoimmune myopathy disease. trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2012-005772-34-DE 2013.
    1. EUCTR2012-005772-34-SE. A placebo-controlled, proof-of concept study of the efficacy of gevokizumab subcutaneously over 24 weeks in the treatment of patients with polymyositis, dermatomyositis or necrotizing autoimmune myopathy disease. www.clinicaltrialsregister.eu/ctr-search/trial/2012-005772-34/SE 2013.
NCT00033891 {published data only (unpublished sought but not used)}
    1. Coyle K, Pokrovnichka A, French K, Joe G, Shrader J, Swan L. A randomized, double-blind, placebo-controlled trial of infliximab in patients with polymyositis and dermatomyositis. Arthritis and Rheumatism 2008;58:Abstract No: 2058.
    1. NCT00033891. Infliximab (Remicade) to treat dermatomyositis and polymyositis. https://clinicaltrials.gov/ct2/show/NCT00033891 2002.
    1. Schiffenbauer A, Garg M, Castro C, Pokrovnichka A, Joe G, Shrader J, et al. A randomized, double-blind, placebo-controlled trial of infliximab in refractory polymyositis and dermatomyositis. Seminars in Arthritis and Rheumatism 2018;47(6):858-64. - PMC - PubMed
NCT00112385 {published data only}
    1. Muscle Study Group. A randomized, pilot trial of etanercept in dermatomyositis. Annals of Neurology 2011;70(3):427-36. - PMC - PubMed
    1. NCT00112385. A pilot study of etanercept in dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT00112385 2006.
NCT00533091 {published data only (unpublished sought but not used)}
    1. Guo X, Higgs BW, Rebelatto M, Zhu W, Greth W, Yao Y, et al. Suppression of soluble T cell-associated proteins by an anti-interferon-alpha monoclonal antibody in adult patients with dermatomyositis or polymyositis. Rheumatology 2014;53(4):686-95. - PMC - PubMed
    1. Guo X, Higgs BW, Zhu W, Yao Y, White W. Proteomics study of a phase 1b trial with an anti-IFN-> monoclonal antibody indicates association of soluble interleukin 2 receptor with type I interferon activity in patients with dermatomyositis or polymyositis. Arthritis and Rheumatism 2012;64:S93-4.
    1. Higgs BW, Zhu W, Morehouse C, White W, Brohawn P, Le C, et al. Sifalimumab, an anti-IFN-alpha monoclonal antibody shows target suppression of a type I IFN signature in blood and muscle of dermatomyositis and polymyositis patients. Arthritis and Rheumatism 2012;64:S325.
    1. Higgs BW, Zhu W, Morehouse C, White WI, Brohawn P, Guo X, et al. A phase 1b clinical trial evaluating sifalimumab, an anti-IFN-alpha monoclonal antibody, shows target neutralisation of a type I IFN signature in blood of dermatomyositis and polymyositis patients. Annals of the Rheumatic Diseases 2014;73(1):256-62. - PMC - PubMed
    1. NCT00533091. A study to evaluate safety of multi-dose MEDI-545 in adult patients with dermatomyositis or polymyositis. https://clinicaltrials.gov/ct2/show/NCT00533091 2008.
NCT01148810 {published data only (unpublished sought but not used)}
    1. Danko K, Vencovsky J, Lundberg IE, Amato AA, Oddis CV, Molnar M, et al. The selective sphingosine-1-phosphate receptor 1/5 modulator siponimod (BAF312) shows beneficial effects in patients with active, treatment refractory polymyositis and dermatomyositis: a phase IIA proof-of-concept, double-blind, randomized trial. Arthritis & Rheumatology 2014;66:S403.
    1. EUCTR2008-006311-21-GB. A multi-centre, double-blind, placebo controlled, proof of concept study to evaluate the efficacy and tolerability of BAF312 in patients with polymyositis and dermatomyositis. clinicaltrialsregister.eu/ctr-search/trial/2008-006311-21/GB 2009.
    1. EUCTR2008-006311-21-HU. A multi-centre, double-blind, placebo controlled, proof of concept study to evaluate the efficacy and tolerability of BAF312 in patients with polymyositis and dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2008-006311-21-HU 2009.
    1. NCT01148810. Efficacy and tolerability of BAF312 in patients with polymyositis and dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT01148810 2010.
NCT01801917 {published data only}
    1. EUCTR2012-002859-42-BE. Study of efficacy and tolerability for BAF312 compared to placebo in patients with polymyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2012-002859-42-BE 2014.
    1. EUCTR2012-002859-42-HU. Study of efficacy and tolerability for BAF312 compared to placebo in patients with polymyositis. https://clinicaltrialsregister.eu/ctr-search/trial/2012-002859-42/HU 2012.
    1. NCT01801917. Efficacy and tolerability of BAF312 in patients with polymyositis. https://clinicaltrials.gov/ct2/show/NCT01801917 2013.
NCT02029274 {published data only (unpublished sought but not used)}
    1. EUCTR2013-001799-39-HU. Study of efficacy and tolerability for BAF312 compared to placebo in patients with active dermatomyositis. https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001799-39/HU 2013.
    1. EUCTR2013-001799-39-PL. Study of efficacy and tolerability for BAF312 compared to placebo in patients with active dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2013-001799-39-PL 2014.
    1. JapicCTI-142590. Safety and efficacy of BAF312 in dermatomyositis. https://rctportal.niph.go.jp/en/detail?trial_id=JapicCTI-142590 2014.
    1. NCT02029274. Safety and efficacy of BAF312 in dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT02029274 2013.
NCT02466243 {published data only (unpublished sought but not used)}
    1. Dan J, Patel J, Sprow G, Diaz D, Kodali N, Feng R, et al. Cutaneous dermatomyositis disease area and severity index activity score (CDASI-A) and associated patient-reported outcomes and biomarkers in a phase 2 clinical trial in dermatomyositis (DM). Arthritis & Rheumatology 2022;74:396-7.
    1. NCT02466243. Safety, tolerability, and efficacy of JBT-101 in subjects with dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT02466243 2015.
    1. Reddy N, Zeidi M, White B, Werth VP. Lenabasum, a cannabinoid type 2 receptor agonist, reduces T-cell population and downregulates type 1 and 2 interferon activities in lesional dermatomyositis skin. Arthritis & Rheumatology 2018;70:3357-8.
    1. Werth V, Concha J, Burroughs J, Okawa J, Feng R, Jobanputra A, et al. Long-term safety and efficacy of lenabasum during 3 years in an open-label extension (OLE) of a phase 2 study of lenabasum in refractory skin disease in dermatomyositis (DM). Annals of the Rheumatic Diseases 2021;80:383-4.
    1. Werth V, Oddis CV, Lundberg IE, Fiorentino D, Cornwall C, Dgetluck N, et al. Design of phase 3 study of lenabasum for the treatment of dermatomyositis. Annals of the Rheumatic Diseases 2019;78:1228.
NCT02612857 {published data only (unpublished sought but not used)}
    1. EUCTR2015-003277-15-GB. A phase 2 study of IMO-8400 in patients with dermatomyositis. https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003277-15/GB 2015.
    1. EUCTR2015-003277-15-HU. A phase 2 study of IMO-8400 in patients with dermatomyositis. https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003277-15/HU 2016.
    1. EUCTR2015-003277-15-SE. A phase 2 study of IMO-8400 in patients with dermatomyositis. https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003277-15/SE 2016.
    1. Gordon P, Cooper R, Chinoy H, Isenberg D, Lundberg IE, Werth V, et al. Design of a randomized, double-blind, placebo-controlled phase 2 clinical trial of the toll-like receptor antagonist IMO-8400 in patients with dermatomyositis. Annals of the Rheumatic Diseases 2016;75:1119.
    1. Gruis K, Brevard J, Granlund L, Horobin J, Nagaraju K, Mozaffar T, et al. Rationale for toll-like receptor antagonism as a potential novel therapeutic approach for dermatomyositis. Journal of Neuromuscular Diseases 2016;3:S57-8.
NCT02971683 {published data only (unpublished sought but not used)}
    1. Aggarwal R, Lundberg I, Song YW, Shaibani A, Werth V, Maldonado M. Randomized placebo-controlled trial to evaluate efficacy and safety of subcutaneous abatacept in adults with active idiopathic inflammatory myopathy: results of the 24-week double-blind and 28-week open-label periods. Arthritis & Rheumatology 2022;74:4443-6. - PMC - PubMed
    1. Aggarwal R, Lundberg IE, Song YW, Shaibani A, Werth VP, Maldonado MA. Randomized, double-blind, placebo-controlled trial to evaluate efficacy and safety of sc abatacept in adults with active idiopathic inflammatory myopathy. Annals of the Rheumatic Diseases 2022;81:711.
    1. EUCTR2016-002269-77-DE. A phase 3 trial to evaluate the effectiveness and safety of abatacept SC alongside standard treatment in comparison to standard treatment alone in improving disease activity in adults with active idiopathic inflammatory myopathy, a group of chronic autoimmune inflammatory diseases that affect skeletal muscle. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2016-002269-77-DE 2017.
    1. JapicCTI-173670. Trial to evaluate the efficacy and safety of abatacept in combination with standard therapy compared to standard therapy alone in improving disease activity in adults with active idiopathic inflammatory myopathy. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-JapicCTI-173670 2017.
    1. NCT02971683. Trial to evaluate the efficacy and safety of abatacept in combination with standard therapy compared to standard therapy alone in improving disease activity in adults with active idiopathic inflammatory myopathy. https://clinicaltrials.gov/ct2/show/NCT02971683 2017.
NCT04025632 {published data only (unpublished sought but not used)}
    1. EUCTR2019-001497-29-NL. A study to evaluate the safety, tolerability, and efficacy of zilucoplan in subjects with immune-mediated necrotizing myopathy. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2019-001497-29-NL 2020.
    1. NCT04025632. Safety and efficacy study of zilucoplan in subjects with immune-mediated necrotizing myopathy. https://clinicaltrials.gov/ct2/show/NCT04025632 2019.
RIM {published data only (unpublished sought but not used)}
    1. Aggarwal R, Bandos A, Reed AM, Ascherman DP, Barohn RJ, Feldman BM, et al. Predictors of clinical improvement in rituximab-treated refractory adult and juvenile dermatomyositis and adult polymyositis. Arthritis & Rheumatology 2014;66(3):740-9. - PMC - PubMed
    1. Aggarwal R, Levesque MC, Oddis CV, Reed AM, Ascherman DP, Barohn RJ, et al. Clinical and serologic predictors of response in rituximab-treated refractory adult and juvenile dermatomyositis (DM) and adult polymyositis (PM)-the RIM study. Arthritis and Rheumatism 2012;64:S682-3.
    1. Aggarwal R, Loganathan P, Koontz D, Qi Z, Reed AM, Oddis CV. Cutaneous improvement in refractory adult and juvenile dermatomyositis after treatment with rituximab. Rheumatology 2017;56(2):247-54. - PMC - PubMed
    1. Aggarwal R, Oddis CV, Bandos A, Koontz D, Zengbiao Q, Levesque MC, et al. Effect of B cell depletion therapy with rituximab on myositis associated autoantibody levels in idiopathic inflammatory myopathy. Arthritis and Rheumatism 2012;64:S325.
    1. Aggarwal R, Oddis CV, Goudeau D, Koontz D, Qi Z, Reed AM, et al. Autoantibody levels in myositis patients correlate with clinical response during B cell depletion with rituximab. Rheumatology 2016;55(6):991-9. - PMC - PubMed
Takada 2002 {published data only (unpublished sought but not used)}
    1. Takada K, Bookbinder S, Furie R, Oddis C, Mojcik C, Bombara M, et al. A pilot study of eculizumab in patients with dermatomyositis. Arthritis and Rheumatism 2002;46(Suppl):S489.
TIM NCT02043548 {published data only (unpublished sought but not used)}
    1. Aggarwal R, Rockette H, Venturupalli S, Marder G, Dimachkie M, Gazeley D, et al. Tocilizumab in myositis: results of a phase IIb double-blind randomized controlled trial. Arthritis & Rheumatology 2020;72:1922-3.
    1. NCT02043548. Tocilizumab in the treatment of refractory polymyositis and dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT02043548 2014.
    1. Oddis CV, Rockette HE, Zhu L, Koontz DC, Lacomis D, Venturupalli S, et al. Randomized trial of tocilizumab in the treatment of refractory adult polymyositis and dermatomyositis. ACR Open Rheumatology 2022;4(11):983-90. - PMC - PubMed

References to studies excluded from this review

Biesenbach 2016 {published data only}
    1. Biesenbach P, Eskandary F, Derfler K, Oberbauer R, Bohmig GA, Ay C, et al. Effect of combined treatment with immunoadsorption and membrane filtration on plasma coagulation - results of a randomized controlled crossover study. Journal of Clinical Apheresis 2016;31(1):29-37. - PubMed
Borelli 1973 {published data only}
    1. Borelli S, Gartmann H, Klaschka F. Clocortolone (Cl 68) versus fluocinolone, a double-blind, randomized lateral comparison. Der Hautarzt; Zeitschrift fur Dermatologie, Venerologie, und Verwandte Gebiete 1973;24(7):294-7. - PubMed
BTOUGH {published data only}
    1. Kimura N, Kawahara T, Uemura Y, Atsumi T, Sumida T, Mimura T, et al. Branched chain amino acids in the treatment of polymyositis and dermatomyositis: a phase II/III, multi-centre, randomized controlled trial. Rheumatology 2022;61(11):4445-54. - PubMed
    1. Kimura N, Kohsaka H. Branched chain amino acids in the treatment of polymyositis and dermatomyositis: results from the BTOUGH study. Annals of the Rheumatic Diseases 2019;78:846.
Bunch 1980 {published data only}
    1. Bunch TW, Worthington JW, Combs JJ, Ilstrup DM, Engel AG. Azathioprine with prednisone for polymyositis. A controlled, clinical trial. Annals of Internal Medicine 1980;92(3):365-9. - PubMed
Cherin 1995 {published data only}
    1. Cherin P, Auperin I, Bussel A, Pourrat J, Herson S. Plasma exchange in polymyositis and dermatomyositis: a multicenter study of 57 cases. Clinical and Experimental Rheumatology 1995;13(2):270-1. - PubMed
ChiCTR1800016629 {published data only}
    1. ChiCTR1800016629. An observation of tofacitinib in patients of MDA5+ clinically amyopathic dermatomyositis with interstitial lung disease. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR1800016629 2018.
ChiCTR1900025087 {published data only}
    1. ChiCTR1900025087. Cyclosporine A in the treatment of polymyositis /dermatomyositis associated interstitial pulmonary disease: a prospective, opened, multicenter, randomized, controlled clinical study. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR1900025087 2019.
ChiCTR1900028143 {published data only}
    1. ChiCTR1900028143. Effect of tacrolimus on dermatomyositis/polymyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR1900028143 2019.
ChiCTR2100042948 {published data only}
    1. ChiCTR2100042948. The safety and the efficacy study of allogenic adipose MSC-Exos in the treatment of anti-MDA5 positive dermatonyositis- associated interstitial lung disease. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2100042948 2021.
ChiCTR2100049656 {published data only}
    1. ChiCTR2100049656. Efficacy and safety evaluation of short interval cyclophosphamide pulse infusion in patients with refractory idiopathic inflammatory myopathy: a double-blind, multicenter randomized clinical trial. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2100049656 2021.
ChiCTR‐IPR‐16007958 {published data only}
    1. ChiCTR-IPR-16007958. An observation of pirfenidone in patients with rapidly progressive interstitial lung disease secondary to clinically amyopathic dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR-IPR-16007958 2016. - PMC - PubMed
ChiCTR‐TRC‐13003178 {published data only}
    1. ChiCTR-TRC-13003178. Study of Treatment Approaches in Myositis (STRAIM): a prospective, multicenter, randomized and open label clinical study. https://trialsearch.who.int/Trial2.aspx?TrialID=https://trialsearch.who.... 2013.
Dalakas 1993 {published data only}
    1. Anonymous. High dose intravenous immunoglobulin infusion in the treatment of dermatomyositis. Deutsche Medizinische Wochenschrift (1946) 1994;119(17):645.
    1. Basta M, Dalakas MC. High-dose intravenous immunoglobulin exerts its beneficial effect in patients with dermatomyositis by blocking endomysial deposition of activated complement fragments. Journal of Clinical Investigation 1994;94(5):1729-35. - PMC - PubMed
    1. Dalakas MC, Illa I, Dambrosia JM, Soueidan SA, Stein DP, Otero C, et al. A controlled trial of high-dose intravenous immune globulin infusions as treatment for dermatomyositis. New England Journal of Medicine 1993;329(27):1993-2000. - PubMed
    1. Dalakas MC. Intravenous immunoglobulin in patients with anti-GAD antibody-associated neurological diseases and patients with inflammatory myopathies: effects on clinicopathological features and immunoregulatory genes. Clinical Reviews in Allergy & Immunology 2005;29(3):255-69. - PubMed
Dalakas 2006 {published data only}
    1. Dalakas MC. The role of high-dose immune globulin intravenous in the treatment of dermatomyositis. International Immunopharmacology 2006;6(4):550-6. - PubMed
Dover 2021 {published data only}
    1. Dover S, Stephens S, Schneiderman JE, Pullenayegum E, Wells GD, Levy DM, et al. The effect of creatine supplementation on muscle function in childhood myositis: a randomized, double-blind, placebo-controlled feasibility study. Journal of Rheumatology 2021;48(3):434-41. - PubMed
EUCTR2018‐000391‐15‐IT {published data only}
    1. EUCTR2018-000391-15-IT. Multicentric, prospective open-label study to assess the efficacy and safety of tocilizumab in subjects with refractory myositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2018-000391-15-IT 2018.
EUCTR2020‐001762‐11 {published data only}
    1. EUCTR2020-001762-11-GB. A research study to collect long term safety information from subjects who have received study treatment from a qualifying lenabasum Corbus trial. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2020-001762-11-GB 2020.
    1. EUCTR2020-001762-11-SE. A research study to collect long term safety information from subjects who have received study treatment from a qualifying lenabasum Corbus trial. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2020-001762-11-SE 2020.
Huppertz 2000 {published data only}
    1. Huppertz HI, Frosch M, Martin C, Christen HJ, JDM-Studiengruppe. Treatment of juvenile dermatomyositis (JDM) with high dose oral steroids or with steroid pulses and low dose oral steroids. Annals of the Rheumatic Diseases 2000;59:728.
    1. Huppertz HI, Frosch M, Sonnichsen C, Christen HJ. Treatment of juvenile dermatomyositis (JDM) with high-dose oral steroids (HoS) or with steroid pulse therapy plus low-dose oral steroids (SPT). Zeitschrift fur Rheumatologie 2001;60(5):392.
ISRCTN48188950 {published data only}
    1. ISRCTN48188950. Dexa-Myositis Trial: treatment of polymyositis and dermatomyositis - dexamethasone versus prednisone. https://trialsearch.who.int/Trial2.aspx?TrialID=ISRCTN48188950 2005.
    1. NTR169. Dexa-myositis trial. https://trialsearch.who.int/Trial2.aspx?TrialID=NTR169 2005.
    1. Van De Vlekkert J, Dehaan J, De Visser M, Hoogendijk JE, Algra A, Van Der Tweel J, et al. Oral dexamethasone pulse therapy versus daily prednisone in subacute inflammatory myopathies: a randomized clinical trial. Neurology 2008;71(2):155.
    1. de Vlekkert J, Hoogendijk JE, Haan RJ, Algra A, Tweel I, Pol WL, et al. Oral dexamethasone pulse therapy versus daily prednisolone in sub-acute onset myositis, a randomised clinical trial. Neuromuscular Disorders 2010;20(6):382-9. - PubMed
ISRCTN87782942 {published data only}
    1. EUCTR2005-002463-88-AT. Efficacy and safety of a human normal immunoglobulin product for intravenous administration (IVIg) in the treatment of dermatomyositis (DM) and polymyositis (PM): prospective, randomised, double-blind, placebo-controlled study. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2005-002463-88-AT 2006.
    1. EUCTR2005-002463-88-DE. Efficacy and safety of a human normal immunoglobulin product for intravenous administration (IVIg) in the treatment of dermatomyositis (DM) and polymyositis (PM): prospective, randomised, double-blind, placebo-controlled study. https://clinicaltrialsregister.eu/ctr-search/trial/2005-002463-88/DE 2005.
    1. ISRCTN87782942. Efficacy and safety of L0133 in the treatment of dermatomyositis and polymyositis: prospective, randomised, double-blind, placebo-controlled study. https://trialsearch.who.int/Trial2.aspx?TrialID=ISRCTN87782942 2006.
Ito 2011 {published data only}
    1. Ito M, Ibi T, Sahashi K, Ichihara M, Ito M, Ohno K. Open-label trial and randomized, double-blind, placebo-controlled, crossover trial of hydrogen-enriched water for mitochondrial and inflammatory myopathies. Medical Gas Research 2011;1(1):24. - PMC - PubMed
JPRN‐UMIN000015469 {published data only}
    1. Fujisawa T, Hozumi H, Kamiya Y, Kaida Y, Akamatsu T, Kusagaya H, et al. Efficacy of combination therapy with prednisolone and calcineurin inhibitor in patients with myositis-associated interstitial lung disease. American Journal of Respiratory and Critical Care Medicine 2020;201(1):A4560.
    1. Fujisawa T, Hozumi H, Kamiya Y, Kaida Y, Akamatsu T, Kusagaya H, et al. Prednisolone and tacrolimus versus prednisolone and cyclosporin A to treat polymyositis/dermatomyositis-associated ILD: a randomized, open-label trial. Respirology (Carlton, Vic.) 2020;26(4):370-7. - PubMed
    1. JPRN-UMIN000015469. Comparison of efficacy between tacrolimus and cyclosporine for the treatment of polymyositis/dermatomyositis-associated interstitial lung disease. https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R0000... 2014.
JPRN‐UMIN000016233 {published data only}
    1. JPRN-UMIN000016233. A phase II/III, randomized, double-blind, placebo-controlled study and open active-treatment extension study to assess the efficacy and safety of the branch chain amino acids (BCAAs) in polymyositis and dDermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-UMIN000016233 2015.
JPRN‐UMIN000032022 {published data only}
    1. JPRN-UMIN000032022. Efficacy and safety of corticosteroid monotherapy versus combination therapy of corticosteroid and tacrolimus for patients with anti-aminoacyl-tRNA synthetase antibody-positive polymyositis/dermatomyositis-associated interstitial lung disease: a prospective randomized multicenter clinical trial. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-UMIN000032022 2018.
JPRN‐UMIN000032193 {published data only}
    1. JPRN-UMIN000032193. Efficacy and safety of combination therapy of corticosteroid and tacrolimus for patients with anti-melanoma differentiation antigen 5 antibody-positive dermatomyositis-associated interstitial lung disease: a prospective multicenter clinical trial. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-UMIN000032193 2018.
jRCTs031200005 {published data only}
    1. JRCTs031200005. Investigation on the safety and utility of apremilast for the treatment of refractory rash in patients with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=jRCTs031200005 2020.
Konishi 2022 {published data only}
    1. Konishi R, Tanaka R, Inoue S, Ichimura Y, Nomura T, Okiyama N. Evaluation of apremilast, an oral phosphodiesterase 4 inhibitor, for refractory cutaneous dermatomyositis: a phase 1b clinical trial. Journal of Dermatology 2022;49(1):118-23. - PubMed
Leitman 1991 {published data only}
    1. Leitman SF, Miller FW, Dowling RF. Randomized, double-blind trial of plasma exchange, leukapheresis and sham apheresis in patients with polymyositis/dermatomyositis (PM/DM). Transfusion 1991;31:43S, Abstract No: S153.
    1. Miller FW, Leitman SF, Cronin ME, Hicks JE, Leff RL, Wesley R, et al. Controlled trial of plasma exchange and leukapheresis in polymyositis and dermatomyositis. New England Journal of Medicine 1992;326(21):1380-4. - PubMed
Miller 2002 {published data only}
    1. Miller J, Walsh Y, Saminaden S, Lecky BR, Winer JB. Randomised double blind controlled trial of methotrexate and steroids compared with azathioprine and steroids in the treatment of idiopathic inflammatory myopathy. Journal of the Neurological Sciences 2002;199:S53.
    1. Miller JA, Walsh Y, Saminaden S, Lecky BR, Winer JB. Randomised double blind trial of methotrexate and steroids compared with azathioprine and steroids in the treatment of idiopathic inflammatory myopathy. Journal of Neurology Neurosurgery and Psychology 2002;72(2):222, Abstract No: 44.
NCT00001261 {published data only}
    1. NCT00001261. Intravenous immunoglobulin (IVIg) for the treatment of inflammatory myopathies. https://ClinicalTrials.gov/ct2/show/NCT00001261 1999.
NCT00035958 {published data only}
    1. NCT00035958. Understanding the pathogenesis and treatment of childhood onset dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT00035958 2002.
NCT00335985 {published data only}
    1. Miyasaka N, Hara M, Koike T, Saito E, Yamada M, Tanaka Y. Effects of intravenous immunoglobulin therapy in Japanese patients with polymyositis and dermatomyositis resistant to corticosteroids: a randomized double-blind placebo-controlled trial. Modern Rheumatology 2012;22(3):382-93. - PMC - PubMed
    1. NCT00335985. Efficacy and safety study of GB-0998 for treatment of steroid-resistant polymyositis and dermatomyositis (PM/DM). https://clinicaltrials.gov/ct2/show/NCT00335985 2006.
NCT02780674 {published data only}
    1. NCT02780674. A phase 1 study of MEDI7734 in type I interferon-mediated autoimmune diseases. https://clinicaltrials.gov/ct2/show/NCT02780674 2016.
NCT02821689 {published data only}
    1. NCT02821689. Pirfenidone in progressive interstitial lung disease associated with clinically amyopathic dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT02821689 2016. - PMC - PubMed
NCT03092180 {published data only}
    1. NCT03092180. Optimizing treatment on idiopathic inflammatory myopathies. https://clinicaltrials.gov/ct2/show/NCT03092180 2017.
NCT03686969 {published data only}
    1. EUCTR2017-002710-31-DE. Double-blind, randomized, placebo-controlled phase iii study evaluating efficacy and safety of subcutaneous human immunoglobulin (octanorm) in patients with dermatomyositis. https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-002710-31/DE 2018.
    1. ISRCTN11520709. Efficacy and safety of Octanorm in patients with dermatomyositis. https://www.isrctn.com/ISRCTN11520709 2018.
    1. NCT03686969. Study evaluating efficacy and safety of octanorm in patients with dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT03686969 2018.
NCT03770663 {published data only}
    1. NCT03770663. Cyclophosphamide and azathioprine vs tacrolimus in antisynthetase syndrome-related interstitial lung disease. https://clinicaltrials.gov/ct2/show/NCT03770663 2021.
NCT03817424 {published data only}
    1. NCT03817424. A study to evaluate VIB7734 in participants with systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE), Sjogren's syndrome, systemic sclerosis, polymyositis, and dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT03817424 2018.
NCT03857854 {published data only}
    1. NCT03857854. Efficacy and safety of pirfenidone in patient with dermatomyositis interstitial lung disease (DM-ILD). https://clinicaltrials.gov/ct2/show/NCT03857854 2018.
NCT04044690 {published data only}
    1. EUCTR2018-003171-35-FR. A study to evaluate the efficacy, safety, and pharmacokinetics of IgPro20 in adults with dermatomyositis (DM). https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2018-003171-35-FR 2019.
    1. NCT04044690. A study to evaluate the efficacy, safety, and pharmacokinetics of IgPro20 in adults with dermatomyositis (DM). https://clinicaltrials.gov/ct2/show/NCT04044690 2019.
NCT04286178 {published data only}
    1. NCT04286178. Exercise, creatine and coenzyme Q10 for childhood myositis. https://clinicaltrials.gov/ct2/show/NCT04286178 2021.
NCT04450654 {published data only}
    1. NCT04450654. Monotherapy IVIG Gamunex-C for HMG-CoA reductase auto-antibody positive necrotizing myopathy treatment (the MIGHT trial). https://clinicaltrials.gov/ct2/show/NCT04450654 2021.
NCT04628936 {published data only}
    1. NCT04628936. Open-label extension to the phase 2 crossover study (PRESIDIO) evaluating KZR-616 in patients with PM and DM. https://clinicaltrials.gov/ct2/show/NCT04628936 2020.
NCT04976140 {published data only}
    1. NCT04976140. Evaluate the safety, tolerability and efficacy of PN-101 in patients with refractory polymyositis or dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT04976140 2021.
NCT05375435 {published data only}
    1. NCT05375435. Efficacy and safety of triple therapy in patients with anti-MDA5 antibody-positive dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT05375435 2022.
NCT05505409 {published data only}
    1. NCT05505409. Efficacy and safety of pirfenidone in CTD-ILD. https://clinicaltrials.gov/ct2/show/NCT05505409 2022.
NTR3184 {published data only}
    1. Habers EA, Brussel M, Langbroek-Amersfoort AC, Royen-Kerkhof A, Takken T. Design of the muscles in motion study: a randomized controlled trial to evaluate the efficacy and feasibility of an individually tailored home-based exercise training program for children and adolescents with juvenile dermatomyositis. BMC Musculoskeletal Disorders 2012;13:108. - PMC - PubMed
    1. Habers GE, Bos GJ, Royen-Kerkhof A, Lelieveld OT, Armbrust W, Takken T, et al. Muscles in motion: a randomized controlled trial on the feasibility, safety and efficacy of an exercise training programme in children and adolescents with juvenile dermatomyositis. Rheumatology 2016;55(7):1251-62. - PubMed
NTR667 {published data only}
    1. NTR667. Phase III clinical trials in new onset juvenile dermatomyositis: steroids treatment versus steroids plus cyclosporine treatment versus steroids plus methotrexate treatment. https://trialsearch.who.int/Trial2.aspx?TrialID=NTR667 2006.
PRINTO {published data only}
    1. Dale A, Milosevic I, Goldacre B. The PRINTO juvenile dermatomyositis trial. Lancet 2016;387(10038):2600-1. - PubMed
    1. EUCTR2005-003956-37-NL. Five year single-blind, phase III effectiveness randomised actively controlled clinical trial in new onset juvenile dermatomyositis:prednisone versus prednisone plus cyclosporine A versus prednisone plus mehotrexate. https://clinicaltrialsregister.eu/ctr-search/trial/2005-003956-37/NL 2006.
    1. EUCTR2005-003956-37-SE. Five year single blind, phase III effectiveness randomised actively controlled clinical trial in new onset juvenile dermatomyositis: prednisone versus prednisone plus cyclosporine A versus prednisone plus methotrexate. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2005-003956-37-SE 2006.
    1. Giancane G, Lavarello C, Pistorio A, Oliveira SK, Zulian F, Cuttica R, et al. The PRINTO evidence-based proposal for glucocorticoids tapering/discontinuation in new onset juvenile dermatomyositis patients. Pediatric Rheumatology 2019;17(1):24. - PMC - PubMed
    1. Giancane G, Lavarello C, Pistorio A, Rider LG, Aggarwal R, Oliveira S, et al. Comparison of the PRINTO 2010 and PRINTO/International Myositis and Clinical Studies Group (IMACS) 2016 improvement criteria in the PRINTO trial in new onset juvenile dermatomyositis. Arthritis & Rheumatology 2016;68:1717-8.
ProDERM {published data only}
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. A randomized, double-blind, placebocontrolled phase III trial of IVIg 10% in patients with dermatomyositis. the ProDERM study: results on efficacy and safety. Annals of the Rheumatic Diseases 2021;80:4-5.
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. Effect of Octagam 10% IVIg versus placebo on clinical response in patients with dermatomyositis: the ProDERM randomized clinical trial. Muscle & Nerve 2020;62:S83.
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. Efficacy and safety of IVIG (Octagam 10%) in patients with active dermatomyositis. Results of a randomized, double-blind, placebo-controlled phase III trial (ProDERM study). Arthritis & Rheumatology 2020;72:1918-20.
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. Safety and tolerability of IVIG (Octagam 10%) in patients with active dermatomyositis. Results of a randomized, double-blind, placebo-controlled phase III trial. Arthritis & Rheumatology 2021;73:1409-12.
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. The ProDERM study: a randomized, double-blinded, placebo controlled trial of IVIG (Octagam 10%) in patients with dermatomyositis. Neuromuscular Disorders 2020;30:S133.
PROMETHEUS {published data only}
    1. EUCTR2007-004410-13-CZ. A prospective, randomised, assessor-blind, multicenter study of efficacy and safety of combined treatment of methotrexate + glucocorticoids versus glucocorticoids alone in patients with polymyositis and dermatomyositis. - PROMETHEUS. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2007-004410-13-CZ 2007.
    1. EUCTR2007-004410-13-SE. A prospective, randomised, assessor-blind, multicenter study of efficacy and safety of combined treatment of methotrexate + glucocorticoids versus glucocorticoids alone in patients with polymyositis and dermatomyositis. - PROMETHEUS. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2007-004410-13-SE 2008.
    1. Kubinova K, Vencovsky J. Semi-quantitative and quantitative evaluation of magnetic resonance imaging in patients with idiopathic inflammatory myopathies-a subanalysis of the PROMETHEUS study. Arthritis & Rheumatology 2019;71:2224-5.
    1. Kubinova K, Vencovsky J. Use of magnetic resonance imaging for assessment of patients with idiopathic inflammatory myopathies-a subanalysis of the PROMETHEUS study. Annals of the Rheumatic Diseases 2019;78:1018-9.
    1. Mann H, Jarosova K, Blumhardt S, Maurer B, Dastmalchi M, Danko K, et al. A prospective, randomized, open-label, assessor-blind, multicenter study of efficacy and safety of combined treatment of methotrexate + glucocorticoids versus glucocorticoids alone in patients with polymyositis and dermatomyositis (PROMETHEUS trial). Annals of the Rheumatic Diseases 2014;73:171.
Sallum 2014 {published data only}
    1. Sallum A, Solis M, Hayashi AP, Artioli G, Sapienza M, Otaduy M. Safety and efficacy of creatine supplementation in juvenile dermatomyositis: a randomized double-blind placebo-controlled cross-over trial. Pediatric Rheumatology 2014;12:P96.
    1. Solis MY, Hayashi AP, Artioli GG, Roschel H, Sapienza MT, Otaduy MC, et al. Efficacy and safety of creatine supplementation in juvenile dermatomyositis: a randomized, double-blind, placebo-controlled crossover trial. Muscle & Nerve 2016;53(1):58-66. - PubMed
SELAM {published data only}
    1. Choy E, Gordon P, White-Alao B, Ibrahim F, Kowalczyk A, Hakim A, et al. One year randomised controlled trial of second line agents in myositis (SELAM): late additional immunosuppression is ineffective in patients who have partially responded to steroids. Arthritis and Rheumatism 2011;63(10):S83.
    1. Choy E, White-Alao B, Ibrahim F, Kowalczyk A, Gordon P, Hakim A, et al. One-year randomised controlled trial of second line agents in myositis (SELAM): additional immunosuppression is ineffective in patients who have partially responded to steroids. Rheumatology 2011;50:iii33‐4.
    1. ISRCTN40085050. Second line agents in myositis. https://www.isrctn.com/ISRCTN40085050 2002.
    1. Ibrahim F, Choy E, Gordon P, Dore CJ, Hakim A, Kitas G, et al. Second-line agents in myositis: 1-year factorial trial of additional immunosuppression in patients who have partially responded to steroids. Rheumatology 2015;54(6):1050‐5. - PMC - PubMed
SLCTR/2015/010 {published data only}
    1. SLCTR/2015/010. Tolerability and effectiveness of every-other-day atorvastatin dosing in patients with statin related muscle disease. https://trialsearch.who.int/Trial2.aspx?TrialID=SLCTR/2015/010 2015.
TIM Dutch {published data only}
    1. EUCTR2020-001710-37-NL. Intravenous immunoglobulin and prednisone vs. prednisone in newly diagnosed myositis: a double blind randomized clinical trial. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2020-001710-37-NL 2021.
    1. Kamperman R, Walter H, De Visser M, Bogaards H, De Borgie C, Colen-De Koning J, et al. Design of 'time is muscle': RCT on IVIg add-on to prednisone in newly diagnosed myositis. Journal of Neuromuscular Diseases 2022;9:S264-5.
    1. NL9722. IVIG in myositis: Time is Muscle (TIM). https://trialsearch.who.int/Trial2.aspx?TrialID=NL9722 2021.
Torley 1991 {published data only}
    1. Torley HI, Madhok R, Capell HA, Brouwer RM, Maddison PJ, Black CM, et al. A double blind, randomised, multicentre comparison of two doses of intravenous iloprost in the treatment of Raynaud's phenomenon secondary to connective tissue diseases. Annals of the Rheumatic Diseases 1991;50(11):800-4. - PMC - PubMed
Unger 2013 {published data only}
    1. Unger L, Kampf S, Aringer M, Luthke K. Real life experience suggests differential effects of rituximab on refractory dermatomyositis and polymyositis. Annals of the Rheumatic Diseases 2013;72:A650.
Vencovsky 2000 {published data only}
    1. Vencovsky J, Jarosova K, Machacek S, Studynkova J, Kafkova J, Bartunkova J, et al. Cyclosporine A versus methotrexate in the treatment of polymyositis and dermatomyositis. Scandinavian Journal of Rheumatology 2000;29(2):95-102. - PubMed
Villalba 1998 {published data only}
    1. Villalba L, Hicks JE, Adams EM, Sherman JB, Gourley MF, Leff RL, et al. Treatment of refractory myositis: a randomized crossover study of two new cytotoxic regimens. Arthritis and Rheumatism 1998;41(3):392‐9. - PubMed
Wang 2012 {published data only}
    1. Wang DX, Shu XM, Tian XL, Chen F, Zu N, Ma L, et al. Intravenous immunoglobulin therapy in adult patients with polymyositis/dermatomyositis: a systematic literature review. Clinical Rheumatology 2012;31(5):801-6. - PubMed

References to studies awaiting assessment

BIM {published data only}
    1. Chadha P, Narain S, Nandkumar P, Sison C, Schiopu E, Levine T, et al. Safety and efficacy of belimumab in the treatment of adult idiopathic inflammatory myositis (polymyositis and dermatomyositis). Arthritis & Rheumatology 2021;73:910-3.
    1. NCT02347891. Belimumab in idiopathic inflammatory myositis. https://clinicaltrials.gov/study/NCT02347891 2015.
ChiCTR2000030591 {published data only}
    1. ChiCTR2000030591. An open-label, randomized controlled trial for baratinib in the treatment of dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2000030591 2020.
ChiCTR2000036282 {published data only}
    1. ChiCTR2000036282. A prospective, double-blind, positive control study of abatacept in the treatment of high-risk patients with dermatomyositis and rapidly progressing interstitial pneumonia. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2000036282 2020.
ChiCTR2000036709 {published data only}
    1. ChiCTR2000036709. Study on early warning of rapid progressive interstitial lung disease in dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2000036709 2020.
EUCTR2006‐000078‐65‐SE {published data only}
    1. EUCTR2006-000078-65-SE. Rituximab in the treatment of refractory adult and juvenile dermatomyositis (DM) and adult polymyositis (PM) - rituximab in myositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2006-000078-65-SE 2006.
EUCTR2021‐000898‐83‐IT {published data only}
    1. EUCTR2021-000898-83-IT. Phase II placebo controlled study, to evaluate the efficacy and safety of begelomab in combination with standard immunosuppressive therapy and/or corticosteroids in the treatment of patients with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2021-000898-83-IT 2021.
NCT00005571 {published data only}
    1. NCT00005571. Safety and effectiveness of h5G1.1-mAb for dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT00005571 2000.
NCT02980198 {published data only}
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    1. EUCTR2019-002605-22-GB. A study to evaluate the safety and efficacy of KZR-616 for the treatment of patients with active inflammations of the muscle and the skin (rheumatic disease). https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2019-002605-22-GB 2019.
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References to ongoing studies

NCT04208464 {published data only}
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NCT04972760 {published data only}
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NCT05669014 {published data only}
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References to other published versions of this review

Raaphorst 2023
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