Efficacy and safety of repository corticotropin injection for refractory cutaneous dermatomyositis: a prospective, open-label study

Abstract

Objectives: Cutaneous dermatomyositis (DM) is often refractory to multiple medications. Repository corticotropin injection (RCI) is FDA-approved for DM, but little is known about its efficacy and safety for treating cutaneous DM. We conducted a prospective, open-label trial assessing efficacy and safety of RCI for treating refractory cutaneous DM. Methods: DM patients with moderate-to-severe cutaneous activity [Cutaneous Dermatomyositis Disease Area and Severity Index activity (CDASI-A)] >14 despite prior treatment with ≥2 systemic agents were enrolled. Patients were initiated on 80 u RCI twice weekly for 6 months. Primary outcomes included significant decreases in CDASI-A and Physician’s Global Assessment (PGA) scores at 6 months. Results: Of 19 patients enrolled, 15 patients (11 females, four males) with DM (seven classic, eight amyopathic) completed 6 months of RCI treatment. Patients were treated with a median 3.0 systemic medications prior to enrolment and were taking a median of 2.0 systemic medications at enrolment. Median baseline CDASI-A score was 19.0 and median PGA activity score was 2.5/10. For patient-reported outcomes, baseline median patient global skin score (PtGSS) was 3.0/10 and median dermatology life quality index (DLQI) score was 7.0/10. At 6 months, there were statistically significant improvements in CDASI-A scores (median ¼ 10.0), PGA scores (median ¼ 0.8/10), PtGSS scores (median ¼ 7.0) and DLQI scores (median ¼ 2.0), among others. Adverse effects were mild. Conclusions: RCI treatment resulted in statistically significant and clinically meaningful improvement in cutaneous DM activity and quality of life. Our results suggest RCI is an effective, safe and well-tolerated treatment for patients with refractory cutaneous dermatomyositis. Clinical Trial Registration: This clinical trial was registered with ClinicalTrials.gov (ClinicalTrials.gov Identifier: NCT01906372).