Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib

Brooke Rothstein, Deep Joshipura, Ami Saraiya, Rana Abdat, Huda Ashkar, Yana Turkowski, Vaneeta Sheth, Victor Huang, Shiu Chung Au, Courtney Kachuk, Nicole Dumont, Alice B. Gottlieb, David Rosmarin

Research output: Contribution to journalArticle

32 Citations (Scopus)

Abstract

Background Existing therapies for vitiligo are limited in efficacy and can be associated with undesirable side effects. Topical Janus kinase inhibitors may offer a new therapeutic option for vitiligo. Objective We sought to assess the role of topical ruxolitinib 1.5% cream, a Janus kinase inhibitor, in vitiligo treatment. Methods This 20-week, open-label, proof-of-concept trial of twice-daily topical ruxolitinib 1.5% cream was conducted in 12 patients with a minimum of 1% affected body surface area of vitiligo. The primary outcome was percent improvement in Vitiligo Area Scoring Index from baseline to week 20. Results Of 12 patients screened, 11 were enrolled and 9 completed the study (54.5% men; mean age, 52 years). Four patients with significant facial involvement at baseline had a 76% improvement in facial Vitiligo Area Scoring Index scores at week 20 (95% confidence interval, 53-99%; P = .001). A 23% improvement in overall Vitiligo Area Scoring Index scores was observed in all enrolled patients at week 20 (95% confidence interval, 4-43%; P = .02). Three of 8 patients responded on body surfaces and 1 of 8 patients responded on acral surfaces. Adverse events were minor, including erythema, hyperpigmentation, and transient acne. Limitations Limitations of the study include the small sample size and open-label study design. Conclusions Topical ruxolitinib 1.5% cream provided significant repigmentation in facial vitiligo and may offer a valuable new treatment for vitiligo.

Original languageEnglish (US)
Pages (from-to)1054-1060.e1
JournalJournal of the American Academy of Dermatology
Volume76
Issue number6
DOIs
StatePublished - Jun 1 2017
Externally publishedYes

Fingerprint

Janus Kinases
Vitiligo
Therapeutics
Confidence Intervals
INCB018424
Hyperpigmentation
Body Surface Area
Acne Vulgaris
Erythema
Sample Size
Extremities

Keywords

  • facial vitiligo
  • Janus kinase inhibitor
  • ruxolitinib
  • topical application
  • VASI
  • vitiligo

ASJC Scopus subject areas

  • Dermatology

Cite this

Rothstein, B., Joshipura, D., Saraiya, A., Abdat, R., Ashkar, H., Turkowski, Y., ... Rosmarin, D. (2017). Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib. Journal of the American Academy of Dermatology, 76(6), 1054-1060.e1. https://doi.org/10.1016/j.jaad.2017.02.049

Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib. / Rothstein, Brooke; Joshipura, Deep; Saraiya, Ami; Abdat, Rana; Ashkar, Huda; Turkowski, Yana; Sheth, Vaneeta; Huang, Victor; Au, Shiu Chung; Kachuk, Courtney; Dumont, Nicole; Gottlieb, Alice B.; Rosmarin, David.

In: Journal of the American Academy of Dermatology, Vol. 76, No. 6, 01.06.2017, p. 1054-1060.e1.

Research output: Contribution to journalArticle

Rothstein, B, Joshipura, D, Saraiya, A, Abdat, R, Ashkar, H, Turkowski, Y, Sheth, V, Huang, V, Au, SC, Kachuk, C, Dumont, N, Gottlieb, AB & Rosmarin, D 2017, 'Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib', Journal of the American Academy of Dermatology, vol. 76, no. 6, pp. 1054-1060.e1. https://doi.org/10.1016/j.jaad.2017.02.049
Rothstein B, Joshipura D, Saraiya A, Abdat R, Ashkar H, Turkowski Y et al. Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib. Journal of the American Academy of Dermatology. 2017 Jun 1;76(6):1054-1060.e1. https://doi.org/10.1016/j.jaad.2017.02.049
Rothstein, Brooke ; Joshipura, Deep ; Saraiya, Ami ; Abdat, Rana ; Ashkar, Huda ; Turkowski, Yana ; Sheth, Vaneeta ; Huang, Victor ; Au, Shiu Chung ; Kachuk, Courtney ; Dumont, Nicole ; Gottlieb, Alice B. ; Rosmarin, David. / Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib. In: Journal of the American Academy of Dermatology. 2017 ; Vol. 76, No. 6. pp. 1054-1060.e1.
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AU - Turkowski, Yana

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N2 - Background Existing therapies for vitiligo are limited in efficacy and can be associated with undesirable side effects. Topical Janus kinase inhibitors may offer a new therapeutic option for vitiligo. Objective We sought to assess the role of topical ruxolitinib 1.5% cream, a Janus kinase inhibitor, in vitiligo treatment. Methods This 20-week, open-label, proof-of-concept trial of twice-daily topical ruxolitinib 1.5% cream was conducted in 12 patients with a minimum of 1% affected body surface area of vitiligo. The primary outcome was percent improvement in Vitiligo Area Scoring Index from baseline to week 20. Results Of 12 patients screened, 11 were enrolled and 9 completed the study (54.5% men; mean age, 52 years). Four patients with significant facial involvement at baseline had a 76% improvement in facial Vitiligo Area Scoring Index scores at week 20 (95% confidence interval, 53-99%; P = .001). A 23% improvement in overall Vitiligo Area Scoring Index scores was observed in all enrolled patients at week 20 (95% confidence interval, 4-43%; P = .02). Three of 8 patients responded on body surfaces and 1 of 8 patients responded on acral surfaces. Adverse events were minor, including erythema, hyperpigmentation, and transient acne. Limitations Limitations of the study include the small sample size and open-label study design. Conclusions Topical ruxolitinib 1.5% cream provided significant repigmentation in facial vitiligo and may offer a valuable new treatment for vitiligo.

AB - Background Existing therapies for vitiligo are limited in efficacy and can be associated with undesirable side effects. Topical Janus kinase inhibitors may offer a new therapeutic option for vitiligo. Objective We sought to assess the role of topical ruxolitinib 1.5% cream, a Janus kinase inhibitor, in vitiligo treatment. Methods This 20-week, open-label, proof-of-concept trial of twice-daily topical ruxolitinib 1.5% cream was conducted in 12 patients with a minimum of 1% affected body surface area of vitiligo. The primary outcome was percent improvement in Vitiligo Area Scoring Index from baseline to week 20. Results Of 12 patients screened, 11 were enrolled and 9 completed the study (54.5% men; mean age, 52 years). Four patients with significant facial involvement at baseline had a 76% improvement in facial Vitiligo Area Scoring Index scores at week 20 (95% confidence interval, 53-99%; P = .001). A 23% improvement in overall Vitiligo Area Scoring Index scores was observed in all enrolled patients at week 20 (95% confidence interval, 4-43%; P = .02). Three of 8 patients responded on body surfaces and 1 of 8 patients responded on acral surfaces. Adverse events were minor, including erythema, hyperpigmentation, and transient acne. Limitations Limitations of the study include the small sample size and open-label study design. Conclusions Topical ruxolitinib 1.5% cream provided significant repigmentation in facial vitiligo and may offer a valuable new treatment for vitiligo.

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