Related Experiment Video
Updated: Jul 24, 2026

Single-cell Analysis of Immunophenotype and Cytokine Production in Peripheral Whole Blood via Mass Cytometry
Published on: June 26, 2018
Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment
Michael D Howell1, Fiona I Kuo1, Beth Rumberger1
1Incyte, Wilmington, Delaware, USA.
Background:
Efficacy of ruxolitinib cream, a topical Jak1/Jak2 inhibitor, was demonstrated in a phase 2 trial in patients with vitiligo.
Objective:
This study aimed to characterize circulating inflammatory biomarker profiles in patients who demonstrated ≥50% improvement in facial Vitiligo Area Scoring Index scores by week 24 (group 1) and those who did not (group 2).
Design:
This was a posthoc 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 between 18 and 75 years with vitiligo, including depigmentation affecting ≥0.5% of body surface area on the face and ≥3% of body surface area 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 ≥50% improvement in facial Vitiligo Area Scoring Index at week 24. Proteomic analysis was performed on baseline serum samples.
Results:
Mean ± standard error facial Vitiligo Area Scoring Index 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 (of 1,104 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 ≥50% improvement in facial Vitiligo Area Scoring Index 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.
Trial Registration:
The original study was registered at ClinicalTrials.gov, NCT03099304.
Insights
Ruxolitinib cream effectively treated vitiligo, with responders showing distinct baseline inflammatory biomarker profiles. These protein differences may help predict treatment success for vitiligo patients.
Area of Science:
- Dermatology
- Immunology
- Proteomics
Background:
- Ruxolitinib cream, a topical Janus kinase (JAK) inhibitor, has shown efficacy in vitiligo treatment.
- Understanding patient response to treatment is crucial for personalized medicine.
Purpose of the Study:
- To investigate circulating inflammatory biomarker profiles in vitiligo patients based on their response to ruxolitinib cream.
- To identify potential biomarkers that differentiate patients achieving significant facial repigmentation.
Main Methods:
- Posthoc analysis of a phase 2, randomized, double-blind, vehicle-controlled trial.
- Patients (18-75 years) with vitiligo applied 1.5% ruxolitinib cream or vehicle.
- Serum proteomic analysis was performed on baseline samples from patients grouped by ≥50% improvement in facial Vitiligo Area Scoring Index (VASI) at week 24.
Main Results:
- 76 out of 1,104 tested proteins were differentially expressed at baseline between responders (n=30) and non-responders (n=27).
- Ten proteins were upregulated in responders (Group 1), while 64 were elevated in non-responders (Group 2).
- Facial VASI improved significantly more in Group 1 compared to Group 2 at weeks 24 and 52.
Conclusions:
- Baseline proteomic profiles differ significantly between vitiligo patients who respond well and those who do not respond to ruxolitinib cream.
- These identified protein differences warrant further investigation for potential use in stratifying treatment benefits.
- Biomarker discovery may aid in predicting therapeutic outcomes for vitiligo patients treated with topical JAK inhibitors.

