Role of JAK Inhibitors for Vitiligo Remains Unclear

The use of topical JAK inhibitors for treating vitiligo appears promising, but existing data do not show significant improvement in outcomes, according to a recently published study.

Alzahra A. Mohammed, of Centre for Translational Medicine, Semmelweis University, Budapest, Hungary, and colleagues conducted a series of meta-analyses to evaluate the efficacy and safety of JAK inhibitors in patients with vitiligo. The analyses included data from 19 studies. They conducted three separate meta-analyses.

The first included data from three randomized controlled trials (RCTs) of the topical JAK inhibitor ruxolitinib that suggested that patients treated with the JAK inhibitor were more likely to achieve Facial Vitiligo Area Scoring Index 75 (F-VASI75) than those treated with vehicle cream (risk ratio [RR]=3.47; 95% CI, 0.98-12.22). The researchers described the improvements seen with the JAK inhibitor as a “clinically notable but statistically nonsignificant” improvement.

The second meta-analysis included data from four single-arm trials, showing a 43.79% average improvement in the Vitiligo Area Scoring Index (VASI) among patients treated with a JAK inhibitor (95% CI, 0.71-0.93; P < 0.001).

Finally, an analysis of a cohort of patients from case reports and case series indicated significant repigmentation increases of 48.7% in patients treated with JAK inhibitors (P=0.0018) and 63.7% in those treated with JAK inhibitors plus narrowband ultraviolet B (P=0.061).

“Although these findings underscore the potential therapeutic benefits of oral JAK inhibitors, it is essential to consider their associated risks, including global immune suppression, cardiovascular events, and hematopoietic disorders such as thrombocytopenia and anemia,” the researchers wrote. “Topical JAK inhibitors, by contrast, generally exhibit a more favorable safety profile; however, they are not entirely devoid of adverse effects, albeit typically mild, including pruritus, application-site acne, and folliculitis.”

Mohammed and colleagues concluded that “future RCTs are essential to evaluate long-term safety, refine optimal application protocols and establish standardized outcome measures for combination therapies, ultimately improving treatment strategies for vitiligo.”

Mohammed AA, et al. Dermatol Ther (Heidelb). 2025 May 7. doi:10.1007/s13555-025-01397-z.