MINNEAPOLIS – The first study to evaluate tofacitinib’s effectiveness at treating severe alopecia areata in the pediatric population found that the janus kinase inhibitor was effective for more than half of the patients, and well tolerated by all.
Of a case series of 13 pediatric patients who had alopecia areata (AA) and were treated with tofacitinib, 9 (68%) experienced “clinically significant” regrowth of hair, with mean improvement in the Severity of Alopecia Tool (SALT) score of 88% for these responders. The nonresponding group, all of whom had alopecia universalis or totalis, saw essentially no response, with a 1% reduction in SALT score.
Lucy Y. Liu, a medical student at Yale University, New Haven, Conn., presented the findings at the annual meeting of the Society for Pediatric Dermatology.
Ms. Liu and her coinvestigators reported that all of the patients had severe AA by SALT scoring, with an overall mean pretreatment SALT score of 74. Eight of the patients (62%) had alopecia universalis, and two (15%) had alopecia totalis.
The patients ranged in age from 12 to 17 years, with a median age of 15. All but three were male, and patients were an average 9 years old at onset of AA. For patients with alopecia totalis or universalis, the duration of the current episode was a median 1.75 years.
Five patients (38%) had atopic dermatitis, while 1 (8%) had thyroid disease. Three patients (23%) had family members with AA; all but one patient, however, had a family history of autoimmune disease of some sort.
Patients were given tofacitinib 5 mg orally twice daily for 5 months. One patient developed new patches of alopecia during treatment, so the dosing for that patient was increased to 10 mg/5 mg daily.
Adverse events for participants included headaches, upper respiratory infections, and “mild, transient increases in transaminases,” wrote Dr. Lieu and her collaborators. No serious adverse events were reported.
Previous work at Yale had shown that tofacitinib reversed alopecia universalis in a patient who received the medication for plaque psoriasis, and that topical treatment with ruxolitinib, another janus kinase inhibitor, was effective in treating alopecia universalis.
Study limitations included the small sample size and the relatively short duration of follow-up, an important consideration because relapse has been observed after tofacitinib treatment in AA. Still, “Tofacitinib is a promising therapy for the treatment of severe AA in adolescents,” wrote Ms. Liu and her colleagues, recommending randomized clinical trials for further exploration of efficacy and safety in the pediatric population.
On Twitter @karioakes