Efficacy of Ruxolitinib Cream in Nonsegmental Vitiligo Confirmed in Real-World Study

Encouraging real-world evidence supports the safety and efficacy of ruxolitinib cream for adults with nonsegmental vitiligo (NSV).1 

Published in the International Journal of Dermatology, the study showed that 15 mg/g of the topical formulation of the Janus kinase (JAK) inhibitor led to rapid and clinically meaningful repigmentation within just 3 months of treatment, including among patients with disease impacting their face.

By week 12 of treatment with ruxolitinib cream, the majority of patients with nonsegmental vitiligo experienced significant repigmentation. | Image credit: etonastenka - stock.adobe.com

The results were both statistically significant and consistent with outcomes seen in phase 3 trials, confirming that real-world use matches controlled study performance. The findings, wrote the researchers, underscore the potential of ruxolitinib cream to become a first-line topical therapy for NSV, especially for patients with facial involvement, where cosmetic concerns are greatest.

Still, the authors caution that the study was limited by its small sample size, single-center design, and relatively short duration. Long-term studies are needed to confirm the durability of repigmentation, relapse rates after discontinuation, and outcomes in patients with more extensive disease.

Vitiligo is an autoimmune disorder that destroys melanocytes and causes patchy depigmentation of the skin. Conventional therapies, including narrowband UVB phototherapy, corticosteroids, and calcineurin inhibitors, offer modest benefit and carry high relapse rates, with nearly 40% of patients relapsing within a year of discontinuing treatment.

Ruxolitinib, a JAK1/2 inhibitor, works by blocking interferon-gamma signaling and downstream chemokines such as CXCL10, which recruit cytotoxic lymphocytes to skin lesions. Phase 3 randomized trials have already demonstrated its efficacy in repigmenting vitiligo lesions, with the treatment gaining an FDA nod for patients aged 12 years and older in 2022,2 but data from daily clinical practice remain limited. 

Researchers at a clinic in southern Italy conducted a single-center retrospective observational study of 50 adult patients with NSV.1 All had vitiligo affecting 0.5% to 10% of their body surface area, with facial involvement. Patients were screened to rule out other autoimmune diseases that might complicate JAK inhibitor therapy.

Participants applied ruxolitinib cream twice daily for 12 weeks. Effectiveness was assessed using the Facial Vitiligo Area Scoring Index (F-VASI), Total Vitiligo Area Scoring Index (T-VASI), and patient-reported outcomes, including the Vitiligo Noticeability Scale (VNS) and quality-of-life measures.

By week 12, the majority of patients experienced significant repigmentation. Median F-VASI dropped from 1.0 at baseline to 0.34 (P < 0.001), with 40% achieving F-VASI 75 (≥75% improvement) and 10% achieving F-VASI 90 (≥90% improvement). During that period, median T-VASI decreased from 2.09 to 0.95 (P < 0.001), with 42% of patients achieving T-VASI 50 (≥50% overall improvement). Additionally, about 1 in 4 patients reported visibly noticeable improvements on the VNS.

The speed of response to treatment, wrote the researchers, may help improve adherence compared to older therapies, which often require longer treatment courses with uncertain results.

Beyond repigmentation, patients reported meaningful improvements in psychosocial well-being. Scores on the Dermatology Life Quality Index dropped significantly, and Vitiligo Quality of Life scores improved by an average of 26 points.

Notably, the study also tracked alexithymia, a personality trait marked by difficulty expressing emotions. Alexithymia scores improved over the course of treatment, suggesting that visible skin improvement may positively impact emotional self-awareness and coping.

The safety profile of the topical treatment mirrored that of phase 3 clinical trials, with no serious side effects reported with the treatment, and systemic absorption remained minimal. Given that many NSV patients had previously failed or relapsed on topical corticosteroids, calcineurin inhibitors, or phototherapy, the cream represents a valuable new option.

References

1. Cristallo M, Zaza I, Francesca D, et al. Effectiveness and safety of ruxolitinib cream 15 mg/g in patients with non-segmental vitiligo: a real-life study. Int J Dermatol. Published online September 1, 2025. doi:10.1111/ijd.70049
2. FDA approves topical treatment addressing repigmentation in vitiligo in patients aged 12 and older. FDA. Last updated July 22, 2022. Accessed September 12, 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-topical-treatment-addressing-repigmentation-vitiligo-patients-aged-12-and-older