Clinical Trials in Myositis: Where Do We Stand?

Abstract

There are currently no universally accepted management guidelines for idiopathic inflammatory myopathies (IIM), with only limited clinical evidence available to guide treatment decisions. However, the situation is changing and the number of clinical trials is growing, thanks to the improvement in our understanding of disease pathogenesis and availability of novel immunomodulatory drugs. We provide an overview of completed and ongoing interventional clinical trials evaluating the efficacy of pharmacotherapy in various forms of adult IIMs. Unfortunately, the results of many trials are not very encouraging and no single drug seems to be a real breakthrough, reflecting the difficulty associated with studying such rare and heterogenous group of diseases. Several drugs have proven good efficacy in certain IIM subgroups and there are promising novel candidates raising hope for the future. The efficacy of intravenous immunoglobulins in dermatomyositis has been clearly confirmed. Encouraging reports, albeit very preliminary, are coming from trials with JAK inhibitors. We eagerly await the results of a large clinical trial with abatacept or the cannabinoid receptor modulator lenabasum.