Oral Tofacitinib Effective for Pediatric Patients with Alopecia Areata

Tofacitinib treatment of alopecia areata may be efficacious for pediatric patients due to rapid regrowth among responders, new findings suggest, though the medication’s durability necessitates additional research.1

These findings followed the conclusion of a recent study authored by Rahul Mahajan, from the department of dermatology, venereology, and leprology at the Post Graduate Institute of Medical Education and Research in India. Mahajan and colleagues noted the need for additional information on the safety and efficacy of oral tofacitinib in pediatric patients with alopecia areata.

“Case reports and small case series highlight the safe and effective use of tofacitinib in children with [alopecia areata],” Mahajan and colleagues wrote. “This retrospective chart review adds to the limited body of evidence, evaluating efficacy and safety of oral tofacitinib monotherapy in moderate to severe [alopecia areata] in children and adolescents and addressing management strategies for suboptimal responses.”1,2,3

Analysis of Tofacitinib Treatment of Children with Alopecia Areata

From January 2021 - December 2023, the investigative team conducted their retrospective chart review, looking at patients with alopecia areata who were under the age of 16 and met their criteria for eligibility. Specifically, they required the potential participants to have a Severity of Alopecia Tool (SALT) score that exceeded 20, and to have undergone at least 6 months of oral tofacitinib monotherapy.

Administration of tofacitinib was not given to subjects weighing less than 10 kg. There was variance in the prescribed dosage based on patients’ weights, with those weighing 40 kg or more receiving 5 mg twice each day and those under 40 kg being given 2.5 mg. Given the unavailability of an oral solution, the team made necessary adjustments for tablet division.

The investigators gathered data regarding SALT scores taken at baseline, follow-up results, demographic and clinical factors, prior medication utilization, and reports of adverse events (AEs). Descriptive and inferential methods were implemented in the team’s statistical analysis, with significance being set at P = .05.

They also consistently monitored subjects, allowing for minimization of risk. Off-label tofacitinib implementation was guided by clinical discretion, and alternative therapies were considered in cases of reported AEs.

The research team concluded that 52 of the 378 pediatric alopecia areata cases evaluated in their assessment initiated treatment with oral tofacitinib. The team also highlighted that 16 were excluded, resulting in a final cohort of 36 individuals.

Among these subjects, there was a mean age of 10.11 years, with 17 being male and 19 female. At baseline, it was reported that the mean SALT score among participants was 73.11. Additionally, the investigative team found that the condition’s average duration at the time of presentation was 2.97 years, ranging from two months to seven years.

They reported that the mean time required to achieve specific levels of regrowth in patients’ hair was as follows: SALT50 in 2.81 months, SALT75 in 4.08 months, SALT90 in 5.71 months, and complete regrowth (SALT100) in 6.74 months. The analysis also demonstrated that total of 72.2% of the 36 subjects succeeded in achieving SALT50 and SALT75.

The research team noted that 66.7% of these individuals attained SALT90 and 52.8% attained SALT100. Overall, the duration of tofacitinib treatment averaged 8.69 months, spanning from 6 to 16 months.

Four of the 10 non-responders who had not attained a SALT75 score had alopecia universalis. Additionally, 2 exhibited patchy alopecia areata, 2 were shown to have ophiasis, and 2 had subtotalis and totalis, respectively.

Those who did not achieve SALT75 scores were noted by the team as having had higher SALT scores at baseline, a lengthier median disease duration, an increase in the prevalence of atopy, and more commonly-reported involvement in the nail region.

Two of the 9 patients who did report AEs canceled their involvement due to urinary tract infection and leukopenia, respectively. Among the other individuals reporting AEs, the events were reported as mild and did not necessitate cessation of medication.

“Tofacitinib shows promise for managing moderate to severe pediatric [alopecia areata], with rapid regrowth and favorable response,” they wrote. “However, durability of response and safety warrant further research.”1

References

1. Mahajan, R., Mehta, H., De, D. and Handa, S. (2025), Oral Tofacitinib for the Management of Moderate to Severe Alopecia Areata in Children and Adolescents: A Retrospective Study of 36 Patients. Pediatric Dermatology. https://doi.org/10.1111/pde.15882.

2. J. Huang, T. Li, Z. Tan, et al., “Effectiveness of Tofacitinib in Pre-Adolescent Alopecia Areata: A Retrospective Case Series and Literature Review,” Acta Dermato-Venereologica 103 (2023): adv13418.

3. S. Youssef and L. A. Bordone, “Clinical Response to Oral Tofacitinib in Pediatric Patients With Alopecia Areata,” JAAD Case Reports 31 (2023): 83–88.