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Upadacitinib Shows Promising Efficacy in Pediatric Refractory Alopecia Areata With Favorable Safety Profile

A small retrospective study suggests that upadacitinib may offer an effective treatment option for pediatric patients with refractory alopecia areata (AA), demonstrating notable improvements in hair regrowth with an acceptable safety profile.

“JAK inhibitors are recommended as first-line systemic therapy for severe AA, yet systemic options for pediatric patients—especially refractory cases—remain limited,” the investigators noted. “Upadacitinib… may offer a potential option for this unmet need.” 

Researchers retrospectively reviewed 12 pediatric patients with refractory AA (baseline Severity of Alopecia Tool [SALT] score 42–100) treated with upadacitinib between December 2023 and February 2025. Patients received weight-based dosing (7.5 mg/day if <30 kg; 15 mg/day if ≥30 kg) for at least 24 weeks. Outcomes were assessed using SALT scores and response thresholds, including SALT50, SALT75, and SALT90. 

Clinical improvements were observed across the cohort. The median SALT score decreased from 69.0 at baseline to 28.0 at week 24, indicating substantial hair regrowth. Additionally, 1 patient (16.7%) achieved complete or near-complete eyebrow and eyelash regrowth, and 1 patient (33.3%) demonstrated nail recovery. 

Treatment was generally well tolerated. Mild adverse events occurred in 25% of patients, including folliculitis and upper respiratory infections, with no serious safety signals reported. 

“This study showed that upadacitinib was effective and well tolerated in younger children with refractory AA who had failed prior systemic therapies,” the authors concluded. “Larger prospective studies are needed to validate these findings.” 

These findings highlight the potential role of upadacitinib as a systemic option in pediatric AA, particularly for patients with limited treatment alternatives.

Reference

Tao K, Miao Y, Hu R, et al. Upadacitinib for the treatment of refractory alopecia areata in pediatric patients: a retrospective study. Presented at: American Academy of Dermatology Annual Meeting; March 27–31, 2026; Denver, Colorado.

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