Researchers report impressive results in a small open trial of the JAK inhibitor tofacitinib in cutaneous sarcoidosis: 6 out of 10 patients improved so much that they reached a zero disease activity level, and all patients improved on average by 83% using a scoring system.
“Not only did patients improve, but in many cases they were able to get out of the basal immunosuppressive regimen, including prednisone i methotrexate. “They would completely eliminate prednisone or, in some cases, substantially reduce it,” the study investigated. William Damsky, doctor, doctor, reported to Experience of virtual meetings of the American Academy of Dermatology.
Sarcoidosis is a common disease that affects approximately one in 25 black women and is thought to contribute to the deaths of about 4,000 people in the United States each year, said Damsky of the Department of Dermatology, Yale University, New Haven. Conn. famous patient is comic Bernie Mac, who died of the disease in 2008.
“About a third of patients have skin involvement,” Damsky said, and the skin may be the only manifestation of the disease. There is no therapy approved by the Food and Drug Administration for cutaneous sarcoidosis, he added. Prednisone, the first-line therapy in cutaneous manifestations, is only approved for pulmonary sarcoidosis.
“There is often an attempt at partial or total transition to other therapies, including methotrexate and TNF-alpha blockers. But this has been a mixed success,” he said. This is not always possible, “so many patients end up taking prednisone.”
Earlier, a Yale team prescribed 5 mg tofacitinib (Xeljanz) for several patients with severe cutaneous sarcoidosis and who got impressive results, Damsky said, including a patient with pulmonary sarcoidosis who also improved. He noted that there are case reports in the medical literature with similar findings.
These positive results inspired the new study. The researchers recruited 10 patients with cutaneous sarcoidosis (9 with involvement of internal organs) with an instrument of activity and cutaneous morphology of sarcoidosis (CSAMI) score of 10 or higher. Nine patients were over fifty, one was 63 years old and five were men. Patients ’skin colors ranged from skin types I to Fitzpatrick’s VI, and they had all been taking at least two medications, usually methotrexate and prednisone.
Patients received 5 mg of tofacitinib twice daily for 6 months. “Everyone improved during the study and six patients had a complete response, which we defined as a zero-activity CSAMI score,” Damsky said. “It’s really pretty remarkable to see that.” Overall, patients saw an 83% improvement in CSAMI scores.
In terms of safety, “all patients completed the study,” he said. “Tofacitinib was well tolerated and there were no adverse effects or serious events.”
A one-month supply of 5 mg twice daily tofacitinib pills would cost between $ 4,900 and $ 5,100 with free coupons, according to information accessed on April 24, 2021 on GoodRx.com. Generic is not available.
In an interview, Sotonye Imolojemu, MD, of the dermatology department, Brigham and Women’s Hospital, Boston, praised the study and said that “tofacitinib is a reasonable treatment for refractory or extensive cutaneous sarcoidosis,” although it will be useful to obtain results from randomized controlled trials.
He warned that the drug “is a potent immunosuppressant, so the risk of infection should be discussed with patients before prescribing them. Detection of chronic infections such as viral hepatitis, tuberculosis, i HIV it should be completed before the start of treatment. Blood counts, liver function, and lipid panels should be monitored regularly. Vaccines needed for those who are immunosuppressed should be administered as much as possible and age-appropriate cancer screening should be kept up to date. “
The study was funded by Pfizer, the Dermatology Foundation and the Yale Department of Dermatology. Damsky revealed research support (Pfizer), consulting fees (Eli Lilly, Pfizer, TWi Biotechnology) and licensing fees (EMD Millipore / MillporeSigma). Imadonemu has no disclosures.
This article originally appeared on MDedge.com, which is part of the Medscape professional network.