Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream

Background
Efficacy of ruxolitinib cream, a topical Janus kinase (JAK) 1/JAK2 inhibitor, was demonstrated in a phase 2 trial in patients with vitiligo.

Objective
To characterize circulating inflammatory biomarker profiles in patients who demonstrated ≥50% improvement in facial Vitiligo Area Scoring Index scores (F-VASI50) by Week 24 (Group 1) and those who did not (Group 2).

Design
This was a post hoc analysis of a multicenter, randomized, double-blind, vehicle-controlled, phase 2 study in which screening was conducted between June 7, 2017 and March 21, 2018.

Population
Patients aged 18–75 years with vitiligo including depigmentation affecting ≥0.5% of body surface area (BSA) on the face and ≥3% of BSA on nonfacial areas were eligible.

Intervention
Patients applied 1.5% ruxolitinib cream to lesions once or twice daily for 52 weeks.

Main outcomes and measures
Patients were grouped by achievement of F-VASI50 at Week 24. Proteomic analysis was performed on baseline serum samples.

Results
Mean±SE F-VASI in Group 1 (n=30) versus Group 2 (n=27) improved by 79.9%±4.0% versus 1.1%±7.3% and 91.9%±1.5% versus 25.1%±13.4% at Weeks 24 and 52, respectively. Broad proteomic analysis revealed 76 proteins (out of 1104 tested) that were differentially expressed between Groups 1 and 2 at baseline (P<0.05). Ten distinct proteins were upregulated in Group 1; 64 were elevated in Group 2. Conclusion This analysis identified potential differences between patients who achieved F-VASI50 response at 24 weeks and those who did not that require deeper scientific interrogation and may be important in stratifying therapeutic benefit for patients with vitiligo.