Researchers Recommend Refined Trial Design for Pediatric Vitiligo JAK Studies
Key Takeaways
- A new Journal of the American Academy of Dermatology correspondence raised methodological questions about a recent pediatric vitiligo trial combining oral JAK inhibitors with 308-nm excimer laser therapy.
- The authors pointed out that lesion-level analyses may overestimate treatment effects due to multiple lesions from the same patient are not statistically independent.
- The correspondence also noted combining baricitinib and tofacitinib into a single treatment arm may obscur potential differences between the two therpies.
Correspondence in the Journal of the American Academy of Dermatology asked for caution when interpreting findings from a recent trial evaluating oral Janus kinase (JAK) inhibitors in combination with 308-nm excimer laser therapy for pediatric progressive vitiligo.
In the letter, Erdem Topal, MD, and Ercan Yılmaz, MD, of Inonu University in Malatya, Turkey, commended Chang and colleagues for conducting one of the few prospective randomized studies in children with progressive vitiligo, an area where high-quality evidence is limited. They also noted two aspects of the study warrant additional consideration.
Patient-Level Outcomes May Better Reflect Clinical Benefit
The first concern centered on the study's analytical approach. While 188 patients were enrolled, efficacy outcomes were reported using 439 individual lesions. Since multiple lesions can originate from a single patient, the observations are not fully independent, the authors note. As a result, patients with more lesions may disproportionately influence estimates of treatment effect and statistical significance. They suggested that patient-level efficacy analyses may better reflect the clinical benefit experienced by individual patients and recommend additional clarification regarding the potential impact of lesion clustering.
The authors quesetioned the combintation of baricitinib and tofacitinib into a single intervention group. While the original investigators reported no significant differences between the two oral JAK inhibitors, few participants received tofacitinib, creating an imbalanced comparison the authors said may have been underpowered.
"These considerations do not diminish the clinical relevance of the study," the authors wrote. "Rather, they highlight important 58 issues that may guide the design of future trials. Studies evaluating a single JAK inhibitor and 59 emphasizing patient-level efficacy outcomes could provide a clearer understanding of the magnitude of 60 treatment benefit and facilitate more precise comparisons across therapeutic strategies. 61 We thank the authors for their contribution and look forward to further research in this evolving area of 62 pediatric vitiligo management."
Source
Topal E, Yilmaz E. JAAD. 2026: Doi:10.1016/j.jaad.2026.06.146