A retrospective analysis of the efficacy and safety of oral tofacitinib in active vitiligo treatment

Abstract

Oral Janus Kinase (JAK) inhibitors represent a significant advancement in the treatment of vitiligo; however, large-scale clinical data on their optimal treatment duration and efficacy remain limited. This study aimed to evaluate the efficacy and safety of the oral JAK inhibitor tofacitinib in patients with active vitiligo over a 3-month period. A retrospective analysis was conducted on patients diagnosed with active vitiligo between June 2023 and January 2024 who received oral tofacitinib for at least 3 months. Data were extracted from patient records. Vitiligo activity was assessed based on the Vitiligo Disease Activity (VIDA) score in combination with specific clinical manifestations, while treatment efficacy was evaluated based on the Vitiligo Area Severity Index (VASI). Safety data were reviewed retrospectively throughout the treatment period. A total of 137 patients were included in the study. The VIDA score improved from 2.75 ± 1.15 at baseline to 1.63 ± 0.66 at treatment discontinuation, with 59.1% (81/137) of patients achieving disease stabilization. The baseline VASI score was 3.79 (1.54-4.0), which decreased to 2.68 (1.0-2.92), reflecting an overall 29.3% improvement. After 3 months of treatment, 98 patients continued to take tofacitinib. Patients who received treatment for 6–9 months demonstrated significantly greater repigmentation (42.27% improvement) compared to those treated for less than 6 months (P < 0.05). No significant differences in efficacy were observed between non-segmental and segmental vitiligo. Mild adverse reactions were reported in 13.8% of patients, primarily including gastrointestinal discomfort, acne, elevated liver enzymes, and palpitations, with no severe adverse events recorded. In conclusion, oral tofacitinib administered for 3 months effectively stabilized vitiligo progression, while treatment extending to 6–9 months results in greater repigmentation and maintained a favorable safety profile. Further long-term controlled studies are warranted to validate these findings and optimize treatment strategies.

Trial registration: http://www.chictr.org.cn. identifier: ChiCTR2400092326.