Efficacy and safety of oral tofacitinib in vitiligo and its variants: A retrospective case series

Background: Vitiligo is a chronic inflammatory skin disorder characterized by patchy depigmentation due to melanocyte loss, often leading to psychiatric morbidities. Emerging therapies like JAK inhibitors, like tofacitinib, target the autoimmune process underlying vitiligo by inhibiting the JAK-STAT pathway, potentially offering more effective and sustained repigmentation with fewer side effects. There is limited data on the dosing, efficacy, and safety of tofacitinib in treating vitiligo, particularly within the Indian population, beyond a few case reports and clinical trials. Aim: This study aims to retrospectively analyze the safety and efficacy of tofacitinib in vitiligo patients. Methods: This retrospective case series reviewed oral tofacitinib use in vitiligo patients from the Department of Dermatology, Cutis Hospital, Bengaluru, India, between January 2020 and August 2022. Data were collected on patient demographics, treatment history, clinical assessments, and laboratory investigations. Tofacitinib was administered at 5 mg twice daily, often combined with NB-UVB phototherapy. Results: The study included 20 patients (11 males and 9 females) aged 24–57 years. Disease duration varied from 1 to 20 years. Nine patients had more than 25% reduction in Vitiligo Area Severity Index when compared to baseline. No significant side effects were recorded, although some patients showed an increase in cholesterol and triglycerides. Conclusion: Tofacitinib shows promise as a targeted therapy for vitiligo with a favorable safety profile. Despite limited data, initial results indicate significant efficacy, particularly when combined with phototherapy. Further research is needed to validate these findings, especially in the Indian population, to establish comprehensive treatment guidelines for JAK inhibitors in vitiligo.