Trial of Etanercept in DM Highlights Challenges of Rare-Disease Research

Dermatomyositis (DM), a subtype of inflammatory myopathy, is a rare disease; estimates of its prevalence range from about 1 to 6 per 100,000. Patients with DM are typically treated with prednisone. Searching for more-effective and less noxious therapies, researchers undertook this randomized, double-blind pilot trial of etanercept, a soluble tumor necrosis factor alpha (TNF-α) receptor fusion protein that inactivates TNF-α. Selection criteria initially targeted newly diagnosed and treatment-naive patients, but were subsequently modified (because of slow recruitment) to also include those with refractory and steroid-dependent disease. After screening 153 patients, 16 patients were enrolled (5 newly diagnosed and 11 with refractory disease); …