Evaluation of apremilast, an oral phosphodiesterase 4 inhibitor, for refractory cutaneous dermatomyositis: A phase 1b clinical trial.

Abstract

Dermatomyositis, an idiopathic inflammatory myopathy, is characterized by cutaneous itchy manifestations, which are frequently refractory and recurrent even after intensive immunosuppressive treatments. To evaluate the effectiveness and safety of apremilast, an oral phosphodiesterase 4 inhibitor, in treating skin-dominant dermatomyositis in which myositis and interstitial lung disease are absent or in remission, we performed this prospective, single-arm, interventional study. A total of five Japanese patients (one male and four females, median [range] age, 64 [37-71] years) with refractory dermatomyositis-associated cutaneous manifestations were recruited and treated with a 12-week course of oral apremilast. Among five enrolled patients, three experienced diarrhea with full-dose apremilast (30 mg twice daily), two of whom withdrew from the study and recovered quickly afterwards. A total of three evaluable female patients (median [range] age, 65 [64-71] years) received apremilast treatment for 12 weeks. A 39.4% reduction from baseline Cutaneous Dermatomyositis Disease Area and Severity Index total activity score, but not the damage score, at week 12 was observed in all three patients. Visual analog scale of itching, and quality of life by Dermatology Life Quality Index were slightly improved in one and two apremilast-treated patients, respectively. As apremilast was effective, with expected and recoverable digestive adverse events (diarrhea), in patients with refractory and recurrent dermatomyositis-associated cutaneous manifestations in this first phase Ib study, it can be suggested as a possible treatment when aggressive immunosuppressive therapies with high-dose systemic corticosteroid and/or immunosuppressive agents for other manifestations, myositis, and interstitial lung disease, are not required.

• Authors: Risa Konishi, Ryota Tanaka, Sae Inoue, Yuki Ichimura, Toshifumi Nomura, Naoko Okiyama
• Journal: The Journal of dermatology (J Dermatol) Vol. 49 Issue 1 Pg. 118-123 (Jan 2022) ISSN: 1346-8138 [Electronic] England
• PMID: 34605053 (Publication Type: Clinical Trial, Phase I, Journal Article)
• Copyright: © 2021 Japanese Dermatological Association.

Chemical References

• Anti-Inflammatory Agents, Non-Steroidal
• Phosphodiesterase 4 Inhibitors
• Thalidomide
• apremilast

Topics

• Aged
• Anti-Inflammatory Agents, Non-Steroidal (therapeutic use)
• Dermatomyositis (complications, drug therapy)
• Double-Blind Method
• Female
• Humans
• Male
• Middle Aged
• Phosphodiesterase 4 Inhibitors (therapeutic use)
• Prospective Studies
• Psoriasis (drug therapy)
• Quality of Life
• Severity of Illness Index
• Thalidomide (analogs & derivatives)
• Treatment Outcome