Novel treatment found for disfiguring cutaneous sarcoidosis

A new study published in NEJM has reported successful treatment of a patient with disfiguring sarcoidosis with a drug approved for rheumatoid arthritis.

For several months, a 48-year-old female patient was treated with arthritis medication tofacitinib, a Jak inhibitor which blocks a pathway known as Jak-STAT, twice-daily. The patient with cutaneous sarcoidosis had not previously had a response to medications and had not received systemic glucocorticoids. The researchers observed that her skin lesions nearly disappeared. This treatment resulted in clinical and histologic remission of her skin disease. They also performed RNA sequencing on biopsied skin from the patient before and during treatment.

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Brett King, M.D., has pioneered the use of Jak inhibitors to treat other intractable skin diseases, including vitiligo, alopecia areata, and eczema.

" A frequently awful disease, which to date has no reliably effective therapy, may now be targeted with Jak inhibitors. We have a relatively safe medicine that works. Before treatment, we were able to show that the Jak-STAT pathway is activated," King said.

"During treatment, not only does her skin disease go away, but there is no activation of the pathway, he added."

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"We plan to evaluate the activation of the Jak-STAT pathway in the lung fluid and blood of over 200 patients with pulmonary and multiorgan sarcoidosis," said co-author Nkiruka Emeagwali. These are big steps toward understanding a disease that has been a mystery for years, the researchers said.

Sarcoidosis is an inflammatory disease that affects multiple organs in the body. While some sarcoidosis patients recover without treatment, others suffer damage to the lungs, heart, lymph nodes, skin, and other organs. Current treatments, including steroids, are not reliably effective for the skin and can cause serious side effects.

For reference log on to https://www.nejm.org/doi/full/10.1056/NEJMoa1805958