Alopecia Areata (AA) is a chronic, autoimmune mediated disorder that causes non-scarring hair loss on the scalp, eyebrows, eyelashes, and sometimes other parts of the body. It usually starts during childhood or early teen years, which makes it especially hard for patients emotionally and socially. Even though AA is quite common, there still isn't a treatment that works well all the time especially for moderate to severe cases. This study aimed to check how well and how safe oral tofacitinib, a type of Janus Kinase (JAK) inhibitor works in children diagnosed with moderate to severe alopecia areata.
Thirty patients aged between 4 and 17 years were enrolled at one pediatric dermatology center. All participants got oral tofacitinib at a dose of 5 mg twice daily for 12 months. The main outcome was how much the Severity of Alopecia Tool (SALT) score changed from the start until 4, 8, and 12 months after starting treatment. Other outcomes included how many patients had at least 50% improvement in their SALT scores, changes in quality of life using the Children’s Dermatology Life Quality Index (CDLQI), and tracking side effects and blood test results during the study.
Results showed a statistically significant reduction in mean SALT scores over time, with a mean baseline score of 68.2 decreasing to 52.1 at 4 months (23.6% improvement), 32.5 at 8 months (52.3% improvement), and 15.8 at 12 months (76.8% improvement). Notably, 63% of patients achieved at least 50% improvement at 8 months, increasing to 77% by the end of the 12-month treatment period. Four patients (13.3%) experienced complete regrowth of hair. In addition, CDLQI scores improved markedly, dropping from a mean of 14.6 at baseline to 4.2 after one year, reflecting substantial enhancement in quality of life.
Tofacitinib was generally well tolerated, with most adverse events being mild and transient in nature. The most commonly reported side effects included upper respiratory tract infections (20%), headache (13.3%), gastrointestinal discomfort (10%), and transient leukopenia (6.7%). No serious adverse events were recorded during the study period, and routine laboratory monitoring revealed only minor fluctuations in hemoglobin, white blood cell count, and liver enzymes, none of which required discontinuation of therapy.
In conclusion, this study shows that oral tofacitinib may be a safe and effective treatment option for children and teenagers with moderate to severe alopecia areata. The results support the need for more research using larger, multicenter, and randomized controlled trials to better understand the long-term effectiveness, best dosing strategy, and safety of tofacitinib in pediatric patients.
Although our findings are promising, they should be interpreted with caution due to the small sample size and open-label design, which can introduce bias. Also, the lack of a control group makes it hard to compare the results with other treatments or placebo. Therefore, future studies should aim to confirm these findings in more diverse patient populations and over longer follow-up periods.
Despite these limitations, this study adds to the growing body of evidence suggesting that JAK inhibitors like tofacitinib can offer meaningful clinical improvements in pediatric AA. Given the psychological impact of the disease on young patients, an effective and well-tolerated treatment is greatly needed. Our findings suggest that tofacitinib could play an important role in managing moderate to severe alopecia areata in children and adolescents, especially those who do not respond to traditional therapies.
International Journal of Dermatology, Venereology and Leprosy Sciences
