PORTLAND – Twice-daily topical therapy with the Janus kinase (JAK) inhibitor ruxolitinib led to significant improvements in facial vitiligo in a small, uncontrolled, open-label, proof-of-concept study.
Four patients with significant baseline facial involvement improved by an average of 76% on the facial Vitiligo Area Scoring Index, or VASI (95% confidence interval, 53%-99%, P = .001), Brooke Rothstein reported at the annual meeting of the Society for Investigative Dermatology. The results suggest that topical JAK inhibition might help treat facial vitiligo, while potentially sparing patients from the side effects of oral therapy, said Ms. Rothstein, a medical student at Tufts University, Boston, who conducted the study under the mentorship of David Rosmarin, MD, of the department of dermatology at Tufts.
The study included 11 patients with vitiligo affecting at least 1% of body surface area. In all, 54% were male and the average age was 52 years. Patients applied ruxolitinib 1.5% phosphate cream to affected areas twice daily for 20 weeks. The primary outcome was percent improvement in VASI from baseline, Ms. Rothstein said.
By week 20, eight (73%) patients responded to treatment. Overall VASI scores improved by 23% (95% CI, 4%-43%; P = .02) when considering all patients and affected body regions. Three of eight patients responded on the body, and one of these eight patients also improved on acral surfaces, but these improvements were modest – less than 10%, compared with baseline, which was statistically insignificant.
Adverse events were generally mild and included erythema, hyperpigmentation, and transient acne, Ms. Rothstein reported. Despite the small sample size and open-label design of this study, the findings support further studies of topical JAK inhibition in vitiligo and add to mounting evidence that targeting interferon-gamma and its associated chemokines might stimulate repigmentation of skin in affected patients, she concluded.
This study also was published online in the Journal of the American Academy of Dermatology (J Am Acad Dermatol. 2017 Apr 5. doi: 10.1016/j.jaad.2017.02.049). The work was partially supported by Incyte, manufacturer of ruxolitinib (Jakafi), which supplied the study drug and reviewed the manuscript, but did not have final approval or control over the decision to submit for publication. An Alpha Omega Alpha Carolyn L. Kuckein Student Research Fellowship also helped support the work. Ms. Rothstein and her coinvestigators reported having no financial conflicts of interest.
Ruxolitinib, in a tablet formulation, is approved by the Food and Drug Administration for treating myelofibrosis and polycythemia vera.