Emerging treatments
Tumor necrosis factor-alpha (TNF-alpha) inhibitors
Infliximab and etanercept exert their effects by blocking the actions of the proinflammatory cytokine TNF-alpha. Small case series and case reports describe improvement in muscle disease with infliximab, and in skin and muscle disease with etanercept.[180][181][182][183][184] A pilot randomized, double-blind placebo-controlled trial in adults with DM reported a corticosteroid-sparing effect and no major safety concerns for patients treated with etanercept.[185] However, a pilot prospective study in patients with refractory juvenile DM found that etanercept did not improve symptoms, and in some patients exacerbated disease.[186] Further trials are needed.
Tofacitinib
Tofacitinib is a Janus kinase (JAK) inhibitor. One open-label study of 10 patients with refractory DM treated with tofacitinib found that 50% of patients experienced a moderate improvement in disease activity, and 50% a minimal improvement in disease activity (according to American College of Rheumatology/European League Against Rheumatism myositis response criteria) after 12 weeks of treatment. The authors of the study noted that the response was predominantly in the skin.[187] Further randomized controlled trials using JAK inhibitors for treating DM are needed.
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