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

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

Glucocorticoids are the first choice drugs for the treatment of idiopathic inflammatory myopathies. The investigators of this study have tried to evaluate the effectiveness of second-line immunosuppressive agents like Cyclosporine and MTX in the patients who did not respond to glucocorticoids.

Cyclosporine 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.

ABSTRACT: 
Background: 

Cyclosporine 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 multicenter factorial-design double-blind placebo-controlled RCT compared steroids alone, MTX (15-25 mg weekly) plus steroids, cyclosporine (1-5 mg/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 function (9% change). Intention to treat and complete analyses indicated that cyclosporine monotherapy, MTX monotherapy and cyclosporine/MTX combination therapy showed no significant treatment effects in comparison with placebo.

Conclusion: 

Neither MTX nor cyclosporine (by themselves or in combination) improved clinical features in myositis patients who had incompletely responded to glucocorticoids.

Rheumatology (Oxford) 2015 Jun; 54(6):1050-5