Apremilast may be safe, efficacious option in recalcitrant cutaneous dermatomyositis

Apremilast may be an efficacious and safe treatment option for patients with recalcitrant cutaneous dermatomyositis, according to a study.

“Recalcitrant cutaneous [dermatomyositis (DM)] significantly impacts patient quality of life, with no standardized treatment approach," Carole Bitar, MD, dermatologist at Tulane Medical Center, and colleagues wrote. “We present the results of a phase 2a, open-label, single-arm nonrandomized controlled trial studying the efficacy and safety of apremilast as an add-on treatment in recalcitrant cutaneous DM.”

Enrolled patients from a single tertiary referral center included eight adults with a cutaneous disease activity severity index (CDASI) score of moderate to severe, defined as greater than 14, despite treatment with steroids. Patients were administered 30 mg of apremilast twice daily for up to 6 months. Clinical follow-up visits were performed at 1, 3, 6 and 7 months after therapy discontinuation.

Of eight patients, seven achieved the primary outcome of an overall response rate of a decrease in CDASI score of at least 4 points at 3 months after treatment. The mean CDASI score decreased by 12.9 points from baseline, or 56.7% (P < .001). Patient’s dermatology life quality index rating also decreased from 15.1 at enrollment to 6.3 after 3 months.

“To our knowledge, this is the first study to investigate the role of apremilast in DM, suggesting its mechanism of action in recalcitrant DM,” Bitar and colleagues wrote. “Larger clinical trials are needed to study apremilast in the treatment of naïve DM and as a monotherapy.”