Baricitinib is effective for severe alopecia areata, but long-term safety is not known

Reference

King B, Ohyama M, Kwon O, et al, for the BRAVE-AA Investigators. Two phase 3 trials of baricitinib for alopecia areata. N Engl J Med 2022;386(18):1687-1699.

Synopsis

This is a combined report of 2 industry-sponsored phase 3 trials that evaluated the Janus kinase inhibitor baricitinib for the treatment of severe alopecia areata (AA). Although not FDA approved for this indication, the medication is available in the doses studied in this trial for the treatment of rheumatoid arthritis and COVID-19. Severe AA was defined as a Severity of Alopecia Tool (SALT) score of 50 or higher, where 100 is complete hair loss of the scalp. Patients also had to be experiencing their current episode of AA for between 6 months and 8 years, with no significant improvement in the last 6 months. Patients in the 2 studies (n = 654 and n = 546) were randomized in a 3:2:2 ratio to receive baricitinib 4 mg once daily, baricitinib 2 mg once daily, or placebo for 36 weeks. The groups were comparable at baseline: the mean SALT score was 85, the patients' average age was 38 years, and approximately 60% were women. Analysis was by intention to treat and 90% of the patients completed the studies. For the first trial, the primary outcome of a SALT score of 20 or less was achieved in 39% in the 4-mg group, 23% in the 2-mg group, and 6% in the placebo group (numbers needed to treat [NNTs] = 3 and 6, respectively). Results were similar in the second study: 36%, 19%, and 3% (NNTs = 3 and 6, respectively). Safety is an important concern, as Janus kinase inhibitors are associated with an increased risk of tuberculosis and other infections, lymphoma, lung cancer, and venous thrombosis. Across both trials, serious adverse events occurred in 2.7% of the 4-mg group, 2.4% in the 2-mg group, and 1.7% of the placebo group. There were no deaths. However, the study is too short to fully ascertain the risks of long-term use.