Tofacitinib, an oral Janus kinase (JAK)-1/3 inhibitor approved for treating rheumatoid arthritis, was beneficial in treating cutaneous dermatomyositis in three patients who had failed traditional therapies, according to a report published in JAMA Dermatology.

Dr. Drew J.B. Kurtzman of the department of dermatology at Brigham and Women’s Hospital and Harvard University, Boston, and his associates evaluated the responses of three patients with multidrug-resistant cutaneous dermatomyositis to treatment with twice-daily oral tofacitinib (Xeljanz). Previous treatment included hydroxychloroquine, methotrexate, mycophenolate mofetil, and intravenous immunoglobulin. Two of the patients received tofacitinib as monotherapy, and one continued using hydroxychloroquine.

Response was measured with the validated Cutaneous Dermatomyositis Disease Area and Severity Index ( CDASI ) activity score. Secondary endpoints included tolerability, adverse events, and need for concurrent therapies. The results were published online in a research letter on April 27 ( JAMA Dermatol. 2016. April 27. doi: 10.1001/jamadermatol.2016.0866 ).

They were treated for a mean of 9.6 months. Significant improvements were noted in all three patients, with clinical responses observed after only 4 weeks. CDASI activity scores decreased in all three patients. The mean improvement was 12 points, which exceeded the 4- to 5-point change reflective of a clinically significant response, the authors pointed out.

Because the two patients on tofacitinib monotherapy showed a decrease to an activity score of 14 or lower, they were considered to have improved from moderate to severe disease, to mild disease. Although the remaining patient experienced a lesser degree of improvement, the response to treatment was still deemed clinically significant.

In addition, all three patients reported less pruritus, and the two patients with classic dermatomyositis reported subjective improvements in strength and fatigue. No adverse events were reported for any of the patients. The greatest degree of improvement was seen in the one patient receiving the highest dose of tofacitinib, suggesting that the effect of tofacitinib on refractory cutaneous dermatomyositis may have been dose dependent, Dr. Kurtzman and his associates wrote.

Noting that larger studies on efficacy and long-term safety are needed to validate their findings, they added, “given the limited number of agents available for the management of recalcitrant skin disease, alternative options are needed. By mitigating the signaling cascades that are likely responsible for dermatomyositis, tofacitinib and other JAK inhibitors may represent a rational therapeutic approach.”

Tofacitinib was approved in 2012 for adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate.

No external funding source was disclosed. All but one of the seven authors declared no conflicts of interest. The remaining author’s disclosure included serving as a consultant for Biogen IDEC, Amgen, Janssen, AbbVie, Eli Lilly, and Momenta, and as speaker for AbbVie and Eli Lilly.