Second-line agents in myositis: 1-year factorial trial of additional immunosuppression in patients who have partially responded to steroids

作者：Ibrahim Fowzia^*; Choy Ernest; Gordon Patrick; Dore Caroline J; Hakim Alan; Kitas George; Isenberg David; Griffiths Bridget; Lecky Bryan; Chakravarty Kuntal; Winer John; Danko Katalin; Cooper Robert G; White Alao Beverley; Scott David L

来源：Rheumatology, 2015, 54(6): 1050-1055.

DOI：10.1093/rheumatology/keu442

• myositis and muscle disease
• rheumatic diseases
• DMARDs therapies
• immunosuppressant therapies
• clinical trials and methods
• basic and clinical sciences
• quality of life
• psychology and social phenomena

Objective. Ciclosporin and MTX are used in idiopathic inflammatory myopathies (DM and PM) when patients incompletely respond to glucocorticoids. Their effectiveness is unproved in randomized controlled trials (RCTs). We evaluated their benefits in a placebo-controlled factorial RCT.
Methods. A 56-week multicentre factorial-design double-blind placebo-controlled RCT compared steroids alone, MTX (15-25mg weekly) plus steroids, ciclosporin (1-5mg/kg/day) plus steroids and all three treatments. It enrolled adults with myositis (by Bohan and Peter criteria) with active disease receiving corticosteroids.
Results. A total of 359 patients were screened and 58 randomized. Of the latter, 37 patients completed 12 months of treatment, 7 were lost to follow-up and 14 discontinued treatment. Patients completing 12 months of treatment showed significant improvement (P<0.001 on paired t-tests) in manual muscle testing (14% change), walking time (22% change) and 9% change). Intention to treat and completer analyses indicated that ciclosporin monotherapy, MTX monotherapy and ciclosporin/MTX combination therapy showed no significant treatment effects in comparison with placebo.
Conclusion. Neither MTX nor ciclosporin (by themselves or in combination) improved clinical features in myositis patients who had incompletely responded to glucocorticoids.

• 出版日期2015-6