Background: Upadacitinib, a selective Janus kinase (JAK) 1 inhibitor, has demonstrated promising efficacy in alopecia areata (AA), particularly in patients unresponsive to conventional therapies. As JAK inhibitors gain prominence, understanding upadacitinib's role in AA management is critical.
Summary: This scoping review synthesizes data from 24 publications, including 64 AA patients treated with upadacitinib (15-45 mg daily). Most patients experience substantial or complete hair regrowth within 1-4 months. The most common AA subtypes include alopecia universalis (n = 28), ophiasis (n = 15), and alopecia totalis (n = 8). Upadacitinib was generally well tolerated, with mild adverse events such as transient acneiform eruptions, leukopenia, and creatine phosphokinase elevations. Many patients with comorbid autoimmune conditions, such as atopic dermatitis (59.4%) and inflammatory bowel disease, also reported improvement. Despite these promising findings, limitations include small cohort sizes, variability in prior treatments, and reliance on case reports. Two ongoing studies are underway: a US Phase 3 clinical trial (M23-716) assessing long-term efficacy and safety, and a real-world observational study in China (NCT06573593) comparing upadacitinib with other JAK inhibitors. These studies underscore the need for larger, controlled trials to establish standardized treatment protocols and long-term safety outcomes.
Key messages: (i) Upadacitinib demonstrates good efficacy in treating AA. (ii) Its safety profile supports potential off-label use. (iii) Larger studies are essential to validate current findings and optimize management.
Keywords: Alopecia areata; Hair loss; Janus kinase 1 inhibitors; Scoping review; Upadacitinib.
Hair loss from conditions like alopecia areata (AA) can significantly impact confidence and emotional well-being. Upadacitinib, a Janus kinase 1 (JAK1) inhibitor, has shown promise for patients unresponsive to other treatments. This review includes findings from 24 studies covering 64 AA patients treated with upadacitinib (15–45 mg daily). Most experienced substantial or complete hair regrowth within 1–4 months, particularly those with severe forms like alopecia universalis or ophiasis. Upadacitinib was generally well tolerated, with mild and temporary side effects such as acne, minor changes in blood counts, and occasional liver enzyme elevations. Many patients with coexisting autoimmune diseases, including atopic dermatitis and inflammatory bowel disease, also saw improvements. While results are promising, larger studies are needed to confirm long-term safety and effectiveness. Ongoing trials aim to address these gaps, positioning upadacitinib as a potential treatment option for AA.
© 2025 The Author(s). Published by S. Karger AG, Basel.