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Review
. 2025 Aug 11;8(8):CD015855.
doi: 10.1002/14651858.CD015855.

Non-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

Non-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, which carry significant morbidity and mortality. Treatment of IIM represents an area of unmet need. This review is an update of a review previously published in 2012, as new and promising data on non-targeted treatments have emerged.

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

Search methods: On 3 February 2023, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, Embase, MEDLINE, ClinicalTrials.gov and WHO ICTRP. We intended to check references and citations, and contact experts to identify additional studies, but lacked the resources.

Selection criteria: We included all randomised controlled trials (RCTs) or quasi-RCTs involving participants (adults and children) with IIM according to defined criteria. We included non-targeted immunosuppressants and immunomodulatory treatments alone or in combination, compared with a placebo, no treatment or another non-targeted immunosuppressant or immunomodulatory treatment. Our two primary outcomes were improvement of function or disability and improvement of muscle strength compared with baseline. By preference, we used the Health Assessment Questionnaire Disability Index (HAQ-DI) for disability and the Manual Muscle Test-8 (MMT8) score (adults or children) for muscle strength. Other outcomes were achievement of definitions of improvement (DOI) (the International Myositis Assessment and Clinical Studies (IMACS) Group or the more recent total improvement scores (TIS); for children, we reported achievement of improvement defined by the Paediatric Rheumatology International Trials Organisation (PRINTO)), cumulative corticosteroid dose, change in skin disease activity, serious adverse event and withdrawals for lack of benefit or adverse events.

Data collection and analysis: We followed standard Cochrane methodology. To assess the risk of bias, we used the domain-based Cochrane risk of bias 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 prioritised comparisons of the following with placebo, no treatment or standard care: immunoglobulin, azathioprine and methotrexate. We included other comparisons as additional tables. We assessed the certainty of evidence using the GRADE approach.

Main results: We identified 16 studies (789 participants). The risk of bias in all but one study was high or unclear. Intravenous immunoglobulin (IVIg), compared to placebo, probably improves disability and muscle strength in participants with refractory IIM (standardised mean difference (SMD) 0.86, 95% confidence interval (CI) 0.51 to 1.21 (disability) and 0.78, 95% CI 0.43 to 1.13 (muscle strength); 3 RCTs, 136 participants; both moderate-certainty evidence). IVIg has a higher response rate based on American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) criteria than placebo (risk ratio (RR) 1.80, 95% CI 1.26 to 2.56; 1 RCT, 95 participants; moderate-certainty evidence). IVIg, compared to placebo, improves skin symptoms (Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) total activity score 0 to 100; higher worse) in people with refractory DM (mean difference (MD) -8.20, 95% CI -11.91 to -4.49; 1 RCT, 95 participants; moderate-certainty evidence). There may be more serious adverse events with IVIg than with placebo (RR 1.91, 95% CI 0.50 to 7.30; 2 RCTs, 144 participants; very low-certainty evidence), but little or no difference between IVIg and placebo in withdrawals for either lack of benefit or adverse events (RR 1.02, 95% CI 0.24 to 4.33; 3 RCTs, 154 participants; very low-certainty evidence). For azathioprine versus placebo, one study showed little or no effect of azathioprine on improvement in muscle strength, but the evidence was very uncertain (RR 1.33, 95% CI 0.43 to 4.13; 1 RCT; 16 participants; very low-certainty evidence). The evidence was also very uncertain for cumulative steroid dose (MD 12.06 mg/kg, 95% CI -6.09 to 30.21; 1 RCT, 16 participants; very low-certainty evidence). This early study did not assess IMACS DOI or CDASI or measure function or disability. Serious adverse events and withdrawals for either lack of benefit or adverse events were not systematically reported. For methotrexate, there may be little or no improvement in adults with DM or PM in function (Amyotrophic Lateral Sclerosis Functional Rating Scale 0 to 40, higher better) (MD 1.24, 95% CI -1.60 to 4.08; 1 RCT, 27 participants; very low-certainty evidence), muscle strength (MMT scale 0 to 80, higher better) (MD -5.68, 95% CI -12.94 to 1.58; 1 RCT, 27 participants; very low-certainty evidence), achievement of IMACS DOI (RR 1.01, 95% CI 0.74 to 1.39; 1 RCT, 27 participants; very low-certainty evidence). Cumulative steroid dose was measured, but the data could not be analysed, and change in CDASI was not measured. In children with new-onset jDM on a background therapy of prednisone, a higher proportion may achieve minimal improvement according to the PRINTO criteria with methotrexate than with placebo (RR 1.40, 95% CI 1.01 to 1.96; 1 RCT, 93 participants; low-certainty evidence). Serious adverse events may occur slightly more frequently with methotrexate (RR 1.48, 95% CI 0.54 to 4.07; 2 RCTs, 124 participants; low-certainty evidence). There may be fewer withdrawals for lack of benefit or adverse events with methotrexate (RR 0.62, 95% CI 0.37 to 1.05; 3 RCTs, 151 participants; low-certainty evidence).

Authors' conclusions: Our review shows improvement in disability, muscle strength and skin symptoms following IVIg in people with refractory DM (for PM, these data are not reliable; other subtypes have not been investigated in RCTs). The improvements related to IVIg in DM may be clinically meaningful, but the absence of established minimal clinically important differences (MCIDs) for both disability and muscle strength in IIM does not facilitate interpretation. For the other agents, the small number of trials of immunosuppressive and immunomodulatory therapies is inadequate to decide whether these agents are beneficial in IIM (excluding IBM). Our review shows room for improvement in the conduct and reporting of clinical trials in IIM, as well as the need to further investigate MCIDs for important outcome measures in IIM.

Trial registration: ClinicalTrials.gov NCT04988087.

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Conflict of interest statement

JR: 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 a sub‐investigator of the ProDERM study and is a 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), 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.

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 the 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.

SA: SA is a subinvestigator for two studies, the Argenx and MYOJAK trials. 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 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 grants for the ARTEMIS, DETERMINE, NCT04999020, NCT05523167, Galarisso (NCT05695950) and MYOJAK (NCT04208464) trials and the Corbus Pharmaceuticals grant. 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 was funded to attend the EULAR e‐congress in 2020 by AbbVie, the EULAR congress in 2023 by Celltrion, and has also been funded by other drug companies to attend meetings in the past. 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. 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

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
Dalakas 1993 {published data only (unpublished sought but not used)}
    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 and Immunology 2005;29(3):255-69. - PubMed
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 (unpublished sought but not used)}
    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 2016.
    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.
JPRN‐UMIN000015469 {published data only (unpublished sought but not used)}
    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 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.
Leitman 1991 {published data only (unpublished sought but not used)}
    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 and unpublished data}
    1. Miller J, Walsh Y, Saminaden S, Lecky BRF, 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 JAL, Walsh Y, Saminaden S, Lecky BRF, 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.
NCT00335985 {published data only (unpublished sought but not used)}
    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 (first posted 8 June 2006).
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 (first posted 27 September 2018).
PRINTO {published data only (unpublished sought but not used)}
    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 and Rheumatology 2016;68:1717-8.
ProDERM {published data only (unpublished sought but not used)}
    1. Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie M, Griger Z, et al. A randomized, double-blind, placebo controlled 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 and 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 and 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 and 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 (unpublished sought but not used)}
    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 and 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.
SELAM {published data only (unpublished sought but not used)}
    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
Vencovsky 2000 {published data only (unpublished sought but not used)}
    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 (unpublished sought but not used)}
    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

References to studies excluded from this review

ARTEMIS {published data only}
    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 and 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
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
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 and Rheumatology 2021;73:910-3.
    1. NCT02347891. Belimumab in idiopathic inflammatory myositis. https://clinicaltrials.gov/ct2/show/NCT03215927 2015.
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 (unpublished sought but not used)}
    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.
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.
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.
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.
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
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
DETERMINE {published data only}
    1. Dan J, Sprow G, Concha J, Kodali N, Diaz D, Vazquez T, et al. Sensitivity of three skin-specific efficacy outcomes to detect patientand physician-reported improvement in overall skin disease in dermatomyositis. Arthritis and 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.
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
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.
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. https://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. https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-005772-34/SE 2013.
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.
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.
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‐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 dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-UMIN000016233 2015.
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
NCT00005571 {published data only}
    1. NCT00005571. Safety and effectiveness of h5G1.1-mAb for dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT00005571 2000.
NCT00033891 {published data only}
    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
NCT00035958 {published data only}
    1. NCT00035958. Understanding the pathogenesis and treatment of childhood onset dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT00035958 2002.
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}
    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}
    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 and 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}
    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.
NCT02043548 {published data only}
    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 and 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
NCT02466243 {published data only}
    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 and 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 and 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}
    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.
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
NCT02971683 {published data only}
    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 and Rheumatology 2022;74:4443-6.
    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.
NCT02980198 {published data only}
    1. EUCTR2016-000137-52-DE. Phase IIa exploratory study to assess the safety and effect of IFNa-Kinoid (IFN-K) in adult patients with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2016-000137-52-DE 2016.
    1. NCT02980198. Study of IFN-K in dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT02980198 2017.
NCT03092180 {published data only}
    1. NCT03092180. Optimizing treatment on idiopathic inflammatory myopathies. https://clinicaltrials.gov/ct2/show/NCT03092180 2017.
NCT03181893 {published data only}
    1. EUCTR2020-004228-41-IT. A placebo-controlled study to evaluate the efficacy, safety and tolerability of PF-06823859 in adult subjects with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2020-004228-41-IT 2021.
    1. NCT03181893. A study in adults with moderate to severe dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT03181893 2018.
NCT03192657 {published data only}
    1. NCT03192657. Basiliximab treating interstitial pneumonia of CADM. https://clinicaltrials.gov/ct2/show/NCT03192657 2017.
NCT03215927 {published data only}
    1. NCT03215927. Abatacept for the treatment of myositis-associated interstitial lung disease. https://clinicaltrials.gov/ct2/show/NCT03215927 2017. - PubMed
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.
NCT03981744 {published data only}
    1. JapicCTI-194822. A study of ustekinumab in participants with active polymyositis and dermatomyositis who have not adequately responded to one or more standard-of-care treatments. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-JapicCTI-194822 2019. - PMC - PubMed
    1. NCT03981744. A study of ustekinumab in participants with active polymyositis and dermatomyositis who have not adequately responded to one or more standard-of-care treatments. https://clinicaltrials.gov/ct2/show/NCT03981744 2019. - PMC - PubMed
NCT04025632 {published data only}
    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.
NCT04033926 {published data only}
    1. EUCTR2019-002605-22-CZ. 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-CZ 2019.
    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.
    1. NCT04033926. A phase 2 study of KZR-616 to evaluate safety and efficacy in patients with active polymyositis or dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT04033926 2020.
NCT04208464 {published data only}
    1. EUCTR2019-003868-42-GB. A trial of the drug baricitinib in the treatment of patients with muscle inflammatory conditions. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2019-003868-42-GB 2020.
    1. NCT04208464. A phase IIa trial of the oral JAK 1/2 inhibitor, baricitinib, in the treatment of adult IIM. https://clinicaltrials.gov/ct2/show/NCT04208464 2020.
NCT04237987 {published data only}
    1. NCT04237987. Low-dose interleukin-2 in combination with standard therapy on idiopathic inflammatory myopathy. https://clinicaltrials.gov/ct2/show/NCT04237987 2020.
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.
NCT04972760 {published data only}
    1. NCT04972760. Baricitinib in patients with relapsing or naïve dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT04972760 2021.
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.
NCT04988087 {published data only}
    1. EUCTR2020-004937-19-DE. A study to evaluate the safety, tolerability and efficacy of MHV370 in participants with Sjogren's Syndrome (SjS) or Mixed Connective Tissue Disease (MCTD). https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2020-004937-19-DE 2021.
NCT04999020 {published data only}
    1. Aggarwal R, Cavagna L, Iikuni N, Rakhade S. Design of a global phase 2/3 randomized, placebo-controlled trial of ravulizumab in adult dermatomyositis. Journal of Neuromuscular Diseases 2022;9:S165-6.
    1. Cavagna L, Aggarwal R, Iikuni N, Rakhade S. Design of a global phase 2/3 randomized, placebo-controlled trial evaluating the efficacy and safety of ravulizumab in adults with dermatomyositis. Annals of the Rheumatic Diseases 2022;81:1750-1.
    1. EUCTR2021-001200-15-FR. Ravulizumab versus placebo in adult participants with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=https://trialsearch.who.... 2021.
    1. NCT04999020. Ravulizumab versus placebo in adult participants with dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT04999020 2021.
    1. jRCT2031210681. Ravulizumab versus placebo in adult participants with dermatomyositis. https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-jRCT2031210681 2022.
NCT05379634 {published data only}
    1. NCT05379634. A study of nipocalimab in participants with active idiopathic inflammatory myopathies. https://clinicaltrials.gov/ct2/show/NCT05379634 2022.
NCT05437263 {published data only}
    1. NCT05437263. A phase 3 study to investigate the efficacy and safety of brepocitinib in adults with dermatomyositis. https://ClinicalTrials.gov/show/NCT05437263 2022.
NCT05495321 {published data only}
    1. NCT05495321. Interleukin-2 on active dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT05495321 2022.
NCT05650567 {published data only}
    1. NCT05650567. Study of M5049 in DM and PM participants (NEPTUNIA). https://clinicaltrials.gov/ct2/show/NCT05650567 2023.
NCT05669014 {published data only}
    1. NCT05669014. A phase 2 proof of concept study to evaluate the efficacy and safety of daxdilimab in participants with dermatomyositis (DM) or anti-synthetase inflammatory myositis (ASIM). https://clinicaltrials.gov/ct2/show/NCT05669014 2022.
NCT05695950 {published data only}
    1. NCT05695950. A study evaluating the effects of GLPG3667 given as oral treatment for up to 24 weeks in adults with dermatomyositis. https://clinicaltrials.gov/ct2/show/NCT05695950 2023.
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
RECITAL {published data only}
    1. EUCTR2012-003633-42-GB. A trial of rituximab compared to usual best care in patients with interstitial (inflammatory or scarring conditions) lung disease due to systemic autoimmune (connective tissue) diseases. https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2012-003633-42-GB 2013.
    1. ISRCTN16474148. Rituximab versus cyclophosphamide in connective tissue disease-ILD. https://trialsearch.who.int/Trial2.aspx?TrialID=ISRCTN16474148 2015.
    1. Maher T, Tudor V, Saunders P, Gibbons M, Fletcher S, Parfrey H, et al. Rituximab versus cyclophosphamide for the treatment of connective tissue disease associated interstitial lung disease (RECITAL): a subgroup analysis of a multi-centre randomised controlled trial. Arthritis and Rheumatology 2022;74:5-7.
    1. Maher TM, Tudor V, Saunders P, Gibbons M, Fletcher S, Parfrey H, et al. Rituximab versus cyclophosphamide for the treatment of connective tissue disease associated interstitial lung disease (RECITAL): a multi-centre randomised controlled trial. American Journal of Respiratory and Critical Care Medicine 2022;205(1):A4779.
    1. Maher TM, Tudor VA, Saunders P, Gibbons MA, Fletcher SV, Denton CP, et al. Rituximab versus intravenous cyclophosphamide in patients with connective tissue disease-associated interstitial lung disease in the UK (RECITAL): a double-blind, double-dummy, randomised, controlled, phase 2b trial. Lancet Respiratory Medicine 2023;11(1):45-54. - PubMed
RIM {published data only}
    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 and 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
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 and Nerve 2016;53(1):58-66. - 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.
Takada 2002 {published data only}
    1. Takada K, Bookbinder S, Furie R, Oddis C, Mojcik C, Bombara M, et al. A pilot study of eculizumab in patients with dermatomyosilis. Arthritis and Rheumatism 2002;46(Suppl):S489.
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.
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

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.
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.
NCT00001261 {published data only}
    1. NCT00001261. Intravenousimmunoglobulin (IVIg) for the treatment of inflammatory myopathies. https://ClinicalTrials.gov/ct2/show/NCT00001261 (first received 4 November 1999).
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 (first received 28 February 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 (first received 5 August 2019).
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.
Tian 2008 {published data only}
    1. Tian JG, Chen JIW, Li FXX. Efficacy and safety of the combined treatment with intravenous immunoglobulin and oral glucocorticoid in the elderly with dermatomyositis. Chinese Journal of Geriatrics 2008;27:588-90.

References to ongoing studies

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.
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.
NCT03770663 {published data only}
    1. NCT03770663. Cyclophosphamide and azathioprine vs tacrolimus in antisynthetase syndrome-related interstitial lung disease. https://clinicaltrials.gov/ct2/show/NCT03770663 (first received 10 December 2018).
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 (first received 16 May 2022).
NCT05505409 {published data only}
    1. NCT05505409. Efficacy and safety of pirfenidone in CTD-ILD. https://clinicaltrials.gov/ct2/show/NCT05505409 (first received 17 August 2022).
NCT05523167 {published data only}
    1. NCT05523167. A study to investigate the efficacy and safety of efgartigimod PH20 SC in adult participants with active idiopathic inflammatory myopathy. https://clinicaltrials.gov/ct2/show/NCT05523167 (first received 31 August 2022).
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.

Additional references

Aali 2021
    1. Aali G, Shokraneh F. No limitations to language, date, publication type, and publication status in search step of systematic reviews. Journal of Clinical Epidemiology 2021;133:165-7. [DOI: 10.1016/j.jclinepi.2021.02.002] [PMID: ] - DOI - PubMed
ACTS Study Group 1996
    1. No authors listed. The Amyotrophic Lateral Sclerosis Functional Rating Scale. Assessment of activities of daily living in patients with amyotrophic lateral sclerosis. The ALS CNTF treatment study (ACTS) phase I-II Study Group. Archives of Neurology 1996;53(2):141-7. - PubMed
Aggarwal 2017
    1. Aggarwal R, Rider LG, Ruperto N, Bayat N, Erman B, Feldman BM, Oddis CV, et al, International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation. 2016 American College of Rheumatology/European League Against Rheumatism criteria for minimal, moderate, and major clinical response in adult dermatomyositis and polymyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative. Annals of the Rheumatic Diseases 2017;76(5):792-801. - PMC - PubMed
Ahlstrom 1993
    1. Ahlstrom G, Gunnarsson LG, Leissner P, Sjoden PO. Epidemiology of neuromuscular diseases, including the postpolio sequelae, in a Swedish county. Neuroepidemiology 1993;12(5):262-9. - PubMed
Ahmed 2020
    1. Ahmed S, Chakka S, Concha J, Krain R, Feng R, Werth VP. Evaluating important change in cutaneous disease activity as an efficacy measure for clinical trials in dermatomyositis. British Journal of Dermatology 2020;182(4):949-54. - PMC - PubMed
Allenbach 2018
    1. Allenbach Y, Mammen AL, Benveniste O, Stenzel W, Amato A, Aussey A, et al. 224th ENMC International Workshop: Clinico-sero-pathological classification of immune-mediated necrotizing myopathies Zandvoort, The Netherlands, 14–16 October 2016. Neuromuscular Disorders 2018;28(1):87-99. - PubMed
Allenbach 2017
    1. Allenbach Y, Mammen A, Benveniste O, Stenzel W, on behalf of the Immune-Mediated Necrotizing Myopathies Working Group. 224th ENMC International Workshop: Clinico-sero-pathological classification of immune-mediated necrotizing myopathies Zandvoort, The Netherlands, 14–16 October 2016. Neuromuscular Disorders 2017;28(1):87-99. - PubMed
Barba 2019
    1. Barba T, Fort R, Cottin V, Provencher S, Durieu I, Jardel S, et al. Treatment of idiopathic inflammatory myositis associated interstitial lung disease: a systematic review and meta-analysis. Autoimmunity Reviews 2019;18(2):113-22. - PubMed
Barnes 2006
    1. Barnes PJ. How corticosteroids control inflammation: Quintiles Prize Lecture 2005. British Journal of Pharmacology 2006;148(3):245-54. - PMC - PubMed
Benveniste 2015
    1. Benveniste O, Stenzel W, Hilton-Jones D, Sandri M, Boyer O, Van Engelen BG. Amyloid deposits and inflammatory infiltrates in sporadic inclusion body myositis: the inflammatory egg comes before the degenerative chicken. Acta Neuropathologica 2015;129:611–24. [DOI: 10.1007/s00401-015-1384-5] - DOI - PMC - PubMed
Betteridge 2019
    1. Betteridge Z, Tansley S, Shaddick G, Chinoy H, Cooper RG, New RP, et al, UKMyonet contributors. Frequency, mutual exclusivity and clinical associations of myositis autoantibodies in a combined European cohort of idiopathic inflammatory myopathy patients. Journal of Autoimmunity 2019;101:48-55. - PMC - PubMed
Bohan 1975a
    1. Bohan A, Peter JB. Polymyositis and dermatomyositis (first of two parts). New England Journal of Medicine 1975;292(7):344-7. - PubMed
Bohan 1975b
    1. Bohan A, Peter JB. Polymyositis and dermatomyositis (second of two parts). New England Journal of Medicine 1975;292(8):403-7. - PubMed
Bredahl 2021
    1. Bredahl EC, Eckerson JM, Tracy SM, McDonald TL, Drescher KM. The role of creatine in the development and activation of immune responses. Nutrients 2021;13(3):751. - PMC - PubMed
Bruce 2003
    1. Bruce B, Fries JF. The Stanford health assessment questionnaire: dimensions and practical applications. Health and quality of life outcomes 2003;1:1-6. - PMC - PubMed
Bruce 2003a
    1. Bruce B, Fries JF. The Stanford Health Assessment Questionnaire: a review of its history, issues, progress, and documentation. Journal of Rheumatology 2003;30(1):167-178. - PubMed
Castrillo‐Viguera 2010
    1. Castrillo-Viguera C, Grasso DL, Simpson E, Shefner J, Cudkowicz ME. Clinical significance in the change of decline in ALSFRS-R. Amyotrophic Lateral Sclerosis 2010;11(1-2):178-80. - PubMed
Chung 2007
    1. Chung YL, Alexanderson H, Pipitone N, Morrison C, Dastmalchi M, Ståhl-Hallengren C, et al. Creatine supplements in patients with idiopathic inflammatory myopathies who are clinically weak after conventional pharmacologic treatment: six-month, double-blind, randomized, placebo-controlled trial. Arthritis & Rheumatology 2007;57(4):694-702. [DOI: 10.1002/art.22687] [PMID: ] - DOI - PubMed
Convery 1977
    1. Convery FR, Minteer MA, Amiel D, Connett KL. Polyarticular disability: a functional assessment. Archives of Physical Medicine and Rehabilation 1977;58:494-9. - PubMed
Covidence [Computer program]
    1. Covidence. Version accessed prior to 14 February 2025. Melbourne, Australia: Veritas Health Innovation, 2023. Available at www.covidence.org.
Cronstein & Aune 2020
    1. Cronstein BN, Aune TM. Methotrexate and its mechanisms of action in inflammatory arthritis. Nature Reviews Rheumatology 2020;16:145-54. - PubMed
Dalakas 1991
    1. Dalakas MC. Polymyositis, dermatomyositis, and inclusion-body myositis. New England Journal of Medicine 1991;325(21):1487-98. - PubMed
Dalakas 2001
    1. Dalakas MC. Progress in inflammatory myopathies: good but not good enough. Journal of Neurology, Neurosurgery and Psychiatry 2001;70(5):569-73. - PMC - PubMed
Deeks 2021
    1. Deeks JJ, Higgins JP, Altman DG, editor(s). Chapter 10: Analysing data and undertaking meta-analyses. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from training.cochrane.org/handbook.
Drahota 2023 [Computer program]
    1. RevMan calculator. Drahota A, Beller E. Available from: https://training.cochrane.org/resource/revman-calculator, accessed 2 June 2023.
Feldman 1995
    1. Feldman BM, Ayling-Campos A, Luy L, Stevens D, Silverman ED, Laxer RM. Measuring disability in juvenile dermatomyositis: validity of the Childhood Health Assessment Questionnaire. Journal of Rheumatology 1995;22(2):326-31. - PubMed
Goswami 2022
    1. Goswami RP, Haldar SN, Chatterjee M, Vij P, Kooi AJ, Lim J, et al. Efficacy and safety of intravenous and subcutaneous immunoglobulin therapy in idiopathic inflammatory myopathy: a systematic review and meta-analysis. Autoimmunity Reviews 2022;21(2):102997. - PubMed
GRADEpro GDT [Computer program]
    1. GRADEpro GDT: GRADEpro Guideline Development Tool. Version accessed 27 July 2025. Hamilton (ON): McMaster University and Evidence Prime, 2022. Available from gradepro.org.
Higgins 2011
    1. Higgins JP, Altman DG, Sterne JA, editor(s). Chapter 8: Assessing risk of bias in included studies. In: Higgins JP, Green S, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 (updated March 2011). The Cochrane Collaboration, 2011. Available from https://www.cochrane.org/authors/handbooks-and-manuals/handbook/archive/....
Higgins 2021a
    1. Higgins JP, Li T, Deeks JJ, editor(s). Chapter 6: Choosing effect measures and computing estimates of effect. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from https://www.cochrane.org/handbook.
Higgins 2021b
    1. Higgins JP, Eldridge S, Li T, editor(s). Chapter 23: Including variants on randomized trials. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from https://www.cochrane.org/handbook.
Hoa 2017
    1. Anh-Tu Hoa S, Hudson M. Critical review of the role of intravenous immunoglobulins in idiopathic inflammatory myopathies. Seminars in Arthritis and Rheumatism 2017;46(4):488-508. - PubMed
Hoogendijk 2004
    1. Hoogendijk JE, Amato AA, Lecky BR, Choy EH, Lundberg IE, Rose MR, et al. 119th ENMC international workshop: trial design in adult idiopathic inflammatory myopathies, with the exception of inclusion body myositis, 10-12 October 2003, Naarden, The Netherlands. Neuromuscular Disorders 2004;14(5):337-45. - PubMed
Huber 2001
    1. Huber AM, Hicks JE, Lachenbruch PA, Perez MD, Zemel LS, Rennebohm RM, et al, for the Juvenile Dermatomyositis Disease Activity Collaborative Study Group. Validation of the Childhood Health Assessment Questionnaire in the juvenile idiopathic myopathies. Journal of Rheumatology 2001;28(5):1106-11. - PubMed
Ingelfinger 2023
    1. Ingelfinger F, Sparano C, Bamert D, Reyes-Leiva D, Sethi A, Rindlisbacher L, et al. Azathioprine therapy induces selective NK cell depletion and IFN-γ deficiency predisposing to herpesvirus reactivation. Journal of Allergy and Clinical Immunology 2023;151(1):P280-6. - PubMed
Kamperman 2022
    1. Kamperman RG, Kooi AJ, Visser M, Aronica E, Raaphorst J. Pathophysiological mechanisms and treatment of dermatomyositis and immune mediated necrotizing myopathies: a focused review. International Journal of Molecular Sciences 2022;23(8):4301. - PMC - PubMed
Khoo 2023
    1. Khoo T, Lilleker JB, Thong BY, Leclair V, Lamb JA, Chinoy H. Epidemiology of the idiopathic inflammatory myopathies. Nature Reviews Rheumatology 2023;19(11):695-712. - PubMed
Lam 2011
    1. Lam CG, Manlhiot C, Pullenayegum EM, Feldman BM. Efficacy of intravenous Ig therapy in juvenile dermatomyositis. Annals of the Rheumatic Diseases 2011;70(12):2089-94. - PubMed
Laskin 2017
    1. Laskin BL, Jiao J, Baluarte HJ, Amaral A, Furth SL, Akimova T, et al. The effects of tacrolimus on T-cell proliferation are short-lived: a pilot analysis of immune function testing. Transplant Direct 2017;3(8):e199. - PMC - PubMed
Leclair 2021
    1. Leclair V, Notarnicola A, Vencovsky J, Lundberg IE. Polymyositis: does it really exist as a distinct clinical subset? Current Opinion in Rheumatology 2021;33(6):537-43. - PubMed
Lim 2020
    1. Lim J, Eftimov F, Verhamme C, Brusse E, Hoogendijk J, Saris C, et al. Intravenous immunoglobulins as first-line treatment in idiopathic inflammatory myopathies: a pilot study. Rheumatology 2020;60(4):1784-92. [DOI: 10.1093/rheumatology/keaa459] - DOI - PMC - PubMed
Loarce‐Martos 2020
    1. Loarce-Martos J, Lilleker JB, Parker M, McHugh N, Chinoy H. Polymyositis: is there anything left? A retrospective diagnostic review from a tertiary myositis centre. Rheumatology 2020;60(7):3398–403. [DOI: 10.1093/rheumatology/keaa801] [PMID: ] - DOI - PubMed
Loarce‐Martos 2021
    1. Loarce-Martos J, Lilleker JB, Parker M, McHugh N, Chinoy H. Polymyositis: is there anything left? A retrospective diagnostic review from a tertiary myositis centre. Rheumatology 2021;60(7):3398-403. - PubMed
Lundberg 2017
    1. Lundberg IE, Tjärnlund A, Bottai M, Werth VP, Pilkington C, Visser M, et al. 2017 European League Against Rheumatism/American College of Rheumatology Classification Criteria for Adult and Juvenile Idiopathic Inflammatory Myopathies and Their Major Subgroups. Arthritis & Rheumatology 2017;69(12):2271-82. [PMID: ] - PMC - PubMed
Mammen 2015
    1. Mammen AL, Tiniakou E. Intravenous immune globulin for statin-triggered autoimmune myopathy. New England Journal of Medicine 2015;373(17):1680-2. - PMC - PubMed
Mammen 2020
    1. Mammen AL, Allenbach Y, Stenzel W, Benveniste O, De Bleecker J, Boyer O, et al. 239th ENMC international workshop: classification of dermatomyositis, Amsterdam, the Netherlands, 14–16 December 2018. Neuromuscular Disorders 2020;30(1):70-92. - PubMed
Mariampillai 2018
    1. Mariampillai K, Granger B, Amelin D, Guiguet M, Hachulla E, Maurier F, et al. Development of a new classification system for idiopathic inflammatory myopathies based on clinical manifestations and myositis-specific autoantibodies. JAMA Neurology 2018;75(12):1528-37. - PMC - PubMed
Marie 2013
    1. Marie I, Hatron PY, Cherin P, Hachulla E, Diot E, Vittecoq O, et al. Functional outcome and prognostic factors in anti-Jo1 patients with antisynthetase syndrome. Arthritis Research & Therapy 2013;15:1-10. - PMC - PubMed
Matsuda 2000
    1. Matsuda M, Koyasu S. Mechanisms of action of cyclosporine. Immunopharmacology 2000;47(2-3):119-25. - PubMed
McKenzie 2021
    1. McKenzie JE, Brennan SE. Chapter 12: Synthesizing and presenting findings using other methods. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from https://www.cochrane.org/handbook.
Mecoli 2019
    1. Mecoli CA, Park JK, Alexanderson H, Regardt M, Needham M, Groot I, et al. Perceptions of patients, caregivers, and healthcare providers of idiopathic inflammatory myopathies: an international OMERACT study. Journal of Rheumatology 2019;46(1):106-11. - PMC - PubMed
Meyer 2015
    1. Meyer A, Meyer N, Schaeffer M, Gottenberg J-E, Geny B, Sibilia J. Incidence and prevalence of inflammatory myopathies: a systematic review. Rheumatology 2015;54(1):50-63. - PubMed
Meyer 2020
    1. Meyer A, Troyanov Y, Drouin J, Oligny-Longpré G, Landon-Cardinal O, Hoa S, et al. Statin-induced anti-HMGCR myopathy: successful therapeutic strategies for corticosteroid-free remission in 55 patients. Arthritis Research & Therapy 2020;22(1):1-10. - PMC - PubMed
Oddis 2005
    1. Oddis CV, Rider LG, Reed AM, Ruperto N, Brunner HI, Koneru B, et al, International Myositis Assessment and Clinical Studies Group. International consensus guidelines for trials of therapies in the idiopathic inflammatory myopathies. Arthritis and Rheumatism 2005;52(9):2607-15. - PubMed
Page 2021a
    1. Page MJ, McKenzie JE, Bossuyt PM, Boutron I, Hoffmann TC, Mulrow CD, et al. The PRISMA 2020 statement: an updated guideline for reporting systematic reviews. BMJ 2021;372:n71. [DOI: 10.1136/bmj.n71] - DOI - PMC - PubMed
Page 2021b
    1. Page MJ, Higgins JP, Sterne JA. Chapter 13: Assessing risk of bias due to missing results in a synthesis. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from https://www.cochrane.org/handbook.
Pineda 2006
    1. Pineda AA. Developments in the apheresis procedure for the treatment of inflammatory bowel disease. Inflammatory Bowel Diseases 2006;12:S10-4. - PubMed
Ponyi 2005
    1. Ponyi A, Borgulya G, Constantin T, Váncsa A, Gergely L, Dankó K. Functional outcome and quality of life in adult patients with idiopathic inflammatory myositis. Rheumatology 2005;44(1):83-8. [DOI: 10.1093/rheumatology/keh404] - DOI - PubMed
Raaphorst 2025
    1. Raaphorst J, Gullick NJ, Shokraneh F, Brassington R, Min M, Ali SS, et al. Targeted immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies. Cochrane Database of Systematic Reviews 2025, Issue 8. Art. No: CD015854. [DOI: 10.1002/14651858.CD015854] - DOI - PMC - PubMed
Ramanathan 2015
    1. Ramanathan S, Langguth D, Hardy TA, Garg N, Bundell C, Rojana-Udomsart A, et al. Clinical course and treatment of anti-HMGCR antibody–associated necrotizing autoimmune myopathy. Neurology-Neuroimmunology & Neuroinflammation 2015;2(3):e96. [DOI: 10.1212/NXI.0000000000000096] - DOI - PMC - PubMed
Review Manager 2020 [Computer program]
    1. Review Manager (RevMan). Version 5.4. The Cochrane Collaboration, 2020.
RevMan Web 2022 [Computer program]
    1. Review Manager Web (RevMan Web). Version 4.21.1. The Cochrane Collaboration, accessed 13 December 2022. Available at revman.cochrane.org.
Rider 2003
    1. Rider LG, Giannini EH, Harris-Love M, Joe G, Isenberg D, Pilkington C, et al, and International Myositis Assessment and Clinical Studies Group. Defining clinical improvement in adult and juvenile myositis. Journal of Rheumatology 2003;30(3):603-17. - PubMed
Rider 2004
    1. Rider LG, Giannini EH, Brunner HI, Ruperto N, James-Newton L, Reed AM, et al, International Myositis Assessment and Clinical Studies Group. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis and Rheumatism 2004;50(7):2281-90. - PubMed
Rider 2010
    1. Rider LG, Koziol D, Giannini EH, Jain MS, Smith MR, Whitney-Mahoney K, et al. Validation of manual muscle testing and a subset of eight muscles for adult and juvenile idiopathic inflammatory myopathies. Arthritis Care & Research 2010;62(4):465-72. [DOI: 10.1002/acr.20035] - DOI - PMC - PubMed
Rider 2011
    1. Rider LG, Werth VP, Huber AM, Alexanderson H, Rao AP, Ruperto N, et al. Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), physician global damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI). Arthritis Care & Research 2011;63 No. S11:S118-57. - PMC - PubMed
Rietveld 2019
    1. Rietveld A, Lim J, De Visser M, Van Engelen B, Pruijn G, Benveniste O, et al. Autoantibody testing in idiopathic inflammatory myopathies. Practical Neurology 2019;19(4):284-94. - PubMed
Rose 2015
    1. Rose MR, Jones K, Leong K, Walter MC, Miller J, Dalakas MC, et al. Treatment for inclusion body myositis. Cochrane Database of Systematic Reviews 2015, Issue 6. Art. No: CD001555. [DOI: 10.1002/14651858.CD001555.pub5] - DOI - PMC - PubMed
Schmidt 2018
    1. Schmidt J. Current classification and management of inflammatory myopathies. Journal of Neuromuscular Diseases 2018;5(2):109-29. [PMID: ] - PMC - PubMed
Schünemann 2021
    1. Schünemann HJ, Vist GE, Higgins JP, Santesso N, Deeks JJ, Glasziou P, Akl EA, Guyatt GH. Chapter 15: Interpreting results and drawing conclusions. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions version 6.2 (updated February 2021). Cochrane, 2021. Available from https://www.cochrane.org/handbook.
Tanboon 2020
    1. Tanboon J, Uruha A, Stenzel W, Nishino I. Where are we moving in the classification of idiopathic inflammatory myopathies? Current Opinion in Neurology 2020;33(5):590-603. [PMID: ] - PubMed
Tiao 2017
    1. Tiao J, Feng R, Berger EM, Brandsema JF, Coughlin CC, Khan N, et al. Evaluation of the reliability of the Cutaneous Dermatomyositis Disease Area and Severity Index and the Cutaneous Assessment Tool-Binary Method in juvenile dermatomyositis among paediatric dermatologists, rheumatologists and neurologists. British Journal of Dermatology 2017;177(4):1086-92. - PMC - PubMed
Toyoda 2003
    1. Toyoda M, Pao A, Petrosian A, Jordan SC. Pooled human gammaglobulin modulates surface molecule expression and induces apoptosis in human B cells. American Journal of Transplantation 2003;3(2):156-66. - PubMed
van der Meulen 2003
    1. Meulen MF, Bronner IM, Hoogendijk JE, Burger H, Venrooij WJ, Voskuyl AE, et al. Polymyositis: an overdiagnosed entity. Neurology 2003;61(3):316-21. - PubMed
Winters 2012
    1. Winters JL. Plasma exchange: concepts, mechanisms, and an overview of the American Society for Apheresis guidelines. Hematology. American Society of Hematology. Education Program 2012;2012(1):7-12. - PubMed
Xiong 2023
    1. Xiong A, Qiang Y, Cao Y, Shuai Y, Chen H, Xiang Q, et al. The therapeutic efficacy and safety of intravenous immunoglobulin in dermatomyositis and polymyositis: a systematic review and meta-analysis. Modern Rheumatology 2023;33(3):533-42. - PubMed
Yassaee 2010
    1. Yassaee M, Fiorentino D, Okawa J, Taylor L, Coley C, Troxel AB, et al. Modification of the cutaneous dermatomyositis disease area and severity index, an outcome instrument. British Journal of Dermatology 2010;162(3):669-73. - PMC - PubMed

References to other published versions of this review

Choy 2005
    1. Choy EHS, Winer J, Lecky B, Hoogendik J. Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis. Cochrane Database of Systematic Reviews 2005, Issue 2. Art. No: CD003643. [DOI: 10.1002/14651858.CD003643] - DOI - PubMed
Choy 2005b
    1. Choy EHS, Hoogendijk JE, Lecky B, Winer JB. Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis. Cochrane Database of Systematic Reviews 2005, Issue 3. Art. No: CD003643. [DOI: 10.1002/14651858.CD003643.pub2] - DOI - PubMed
Choy 2009
    1. Choy EHS, Hoogendijk JE, Lecky B, Winer JB, Gordon P. Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis. Cochrane Database of Systematic Reviews 2009, Issue 4. Art. No: CD003643. [DOI: 10.1002/14651858.CD003643.pub3] - DOI - PubMed
Gordon 2012
    1. Gordon PA, Winer JB, Hoogendijk JE, Choy EHS. Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis. Cochrane Database of Systematic Reviews 2012, Issue 8. Art. No: CD003643. [DOI: 10.1002/14651858.CD003643.pub4] - DOI - PMC - PubMed
Raaphorst 2023
    1. Raaphorst J, Gullick NJ, Pipitone N, Shokraneh F, Brassington R, Ali S Sasha, et al. Immunosuppressive and immunomodulatory therapies for idiopathic inflammatory myopathies. Cochrane Database of Systematic Reviews 2023, Issue 1. Art. No: CD014510. [DOI: 10.1002/14651858.CD014510] - DOI

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