Efficacy and safety of tofacitinib in patients with total and universal alopecia- A retrospective evaluation of 69 patients

Background Alopecia areata (AA) is a chronic autoimmune dermatosis with significant challenges in the treatment of severe cases. Recently, tofacitinib has been a promising cure for moderate to severe AA. Yet, its long-term efficacy and safety in the treatment of alopecia totalis (AT) and alopecia universalis (AU) remain underexplored, and determinants of its effectiveness are poorly understood. Aim To evaluate the long-term efficacy and safety of tofacitinib in AT and AU patients and explore the factors that may influence treatment outcomes. Methods We retrospectively assessed the efficacy and safety of tofacitinib in 69 AT and AU patients between January 2020 and June 2024. We also explored risk factors affecting the efficacy of tofacitinib using binary logistic regression analysis. Results After a median treatment of 6 months with tofacitinib, 47.8% (33/69) of patients had a severity of alopecia tool (SALT) score ≤ 20, and 26.1% (18/69) had complete hair regrowth. Patients showed more pronounced SALT score reductions and significant efficacy at weeks 24 and 36, compared to week 12 (P < 0.01). Binary logistic regression analysis revealed that younger initial age of AA (odds ratio (OR) = 1.063, 95% confidence interval (CI) 1.011-1.117, P = 0.017) and shorter treatment duration (OR = 1.249, 95% CI 1.065-1.465, P = 0.006) were significant risk factors associated with poorer efficacy of tofacitinib. Acneiform rash and folliculitis were the primary adverse effects. Limitation The single-center, retrospective study may be limited by data deficiencies and a small sample size. Conclusion Tofacitinib demonstrated significant efficacy in treating patients with AT and AU within a minimum of 6 months. Severe adverse reactions were not observed. Notably, the age of AA onset and the duration of tofacitinib treatment may be significant factors influencing its therapeutic outcomes.