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The following is a summary of “Off-Label Use of Topical Ruxolitinib in Dermatology: A Systematic Literature Review and Current Perspectives,” published in the April 2025 issue of Experimental Dermatology by Spadafora et al. 

JAK inhibitors were used to treat various inflammatory skin diseases, but systemic formulations were linked to a higher risk of major adverse events (AEs). Ruxolitinib 1.5% cream, a selective topical JAK1, and JAK2 inhibitor, had been approved by the EMA and MHRA for non-segmental vitiligo and by the FDA for both vitiligo and atopic dermatitis, with off-label use reported for several skin conditions, though data were mainly limited to case reports and a few prospective studies with mixed outcomes.  

Researchers conducted a retrospective study to examine the potential efficacy of topical Ruxolitinib for treating various skin diseases in an off-label setting.  

They searched the MEDLINE (PubMed) and Scopus databases from inception to September 2024 using the keywords “ruxolitinib cream and dermatology” and “topical ruxolitinib and dermatology.” Reviews, irrelevant articles, books, book chapters, and articles not in English were excluded from the analysis.  

The results showed that 170 studies were screened, with 112 excluded based on criteria and 58 assessed for eligibility. Of these, 28 studies published between 2012 and 2024 were selected. Ruxolitinib cream was mainly used off-label for treating lichenoid and granulomatous dermatoses, as well as alopecia areata. Topical ruxolitinib was effective and safe for the former skin conditions, but its efficacy in alopecia areata was controversial and showed as a promising treatment option for lichenoid and granulomatous dermatoses.  

Investigators concluded that despite exciting results from the oral formulation, no consistent data were described for topical ruxolitinib in alopecia areata. 

Source: onlinelibrary.wiley.com/doi/10.1111/exd.70095