Efficacy and safety of the oral Janus kinase inhibitor baricitinib in the treatment of adults with alopecia areata: Phase 2 results from a randomized controlled study

Background: There are no treatments approved by the Food and Drug Administration for alopecia areata. Objective: To evaluate the efficacy and safety of baricitinib in patients with >= 50% scalp hair loss in a phase 2 study of adults with alopecia areata (BRAVE-AA1). Methods: Patients were randomized 1:1:1:1 to receive placebo or baricitinib 1 mg, 2 mg, or 4 mg once daily. Two consecutive interim analyses were performed after all patients completed weeks 12 and 36 or had discontinued treatment prior to these time points. The primary endpoint was the proportion of patients achieving a Severity of Alopecia Tool (SALT) score <= 20 at week 36. Logistic regression was used with nonresponder imputation for missing data. Results: A total of 110 patients were randomized (placebo, 28; baricitinib 1-mg, 28; 2-mg, 27; 4-mg, 27). The baricitinib 1-mg dose was dropped after the first interim analysis based on lower SALT30 response rate. At week 36, the proportion of patients achieving a SALT score of <= 20 was significantly greater in baricitinib 2-mg (33.3%, P = .016) and 4-mg (51.9%, P = .001) groups versus placebo (3.6%). Baricitinib was well tolerated with no new safety findings. Limitations: Small sample size limits generalizability of results. Conclusion: These results support the efficacy and safety of baricitinib in patients with >= 50% scalp hair loss.