A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo

Vitiligo is a multifactorial depigmentation disorder characterized by the destruction of melanocytes, resulting in loss of pigmentation of the skin.¹ An autoimmune process plays an important role in the disease pathogenesis, with CD8+ T cells in vitiligo lesions, producing a variety of cytokines, such as interferon‐gamma (IFN‐γ).² Treatment up until now has been with the use of topical corticosteroids, immunomodulators, and narrow band-ultraviolet (UV) B, none of which is approved by the US Food and Drug Administration (FDA) for the treatment of vitiligo.¹ Monobenzone is an FDA-approved treatment for the depigmentation of vitiligo, offering patients with more extensive disease an option for treatment by inducing melanocyte necrosis giving patients a more uniform skin tone.³

Ruxolitinib 1.5% cream, a topical Janus kinase (JAK) inhibitor, was approved by the FDA in July 2022 for treatment of nonsegmental vitiligo in patients ages 12 and up.⁴ Topical ruxolitinib is the first drug FDA-approved for repigmentation in patients with vitiligo.⁴ Ruxolitinib can be used topically up to 60 grams in one week, or 100 grams over 2 weeks on ≤10% body surface area (BSA).⁵ The purpose of this review is to describe the pharmacology, clinical trials, safety, and efficacy of ruxolitinib in the treatment of vitiligo.

A systematic review was performed using the terms ruxolitinib OR opzelura in MEDLINE (PubMed) and EMBASE databases. Available studies were considered for inclusion if they were written in English and related to pharmacology, clinical trials, adverse events (AEs), and safety prior to July 2022. References of the included sources were also searched to identify additional studies for inclusion. ClinicalTrials.gov was searched to identify ongoing or unpublished studies.