Review Provides Updates on Off-Label Ruxolitinib for Alopecia, Other Conditions

Although topical ruxolitinib is being increasingly implemented off-label for different inflammatory skin conditions, a new systematic review suggests that there is consistent evidence of efficacy primarily for lichenoid or granulomatous dermatoses and mixed results for other conditions like alopecia areata.1

This systematic review was authored by a team of investigators who sought to assess off-label ruxolitinib utilization, given that data on the drug’s off-label use had mostly been limited to single case reports and series and only a few prospective studies. Marco Spadafora, from Dental Medicine and Morphological Sciences at the University of Modena and Reggio Emilia in Italy, led a team of researchers in this analysis.

“As previously reported for other topical JAK inhibitors, topical ruxolitinib may demonstrate efficacy in inflammatory and immune-mediated skin conditions other than vitiligo and atopic dermatitis,” Spadafora and colleagues wrote.1,2,3 “We reviewed the literature to provide the most up-to-date evidence-based data on the off-label use of topical ruxolitinib in dermatology.”

The investigative team utilized the MEDLINE/PubMed and Scopus databases for their comprehensive search of available research. They included all records available from inception through September 2024, though they only assessed analyses published in English. Studies lacking an English-language version were not included as a result of the investigators' limited resources for translating non-English texts.

The team imported all records they gathered into reference management software to consolidate the findings from both databases. Additionally, screenings of abstracts and titles were based on a set of criteria that were predefined, followed by full-text assessments of research that was deemed to be eligible for inclusion.

Only peer-reviewed journal articles were involved in the review; materials such as preprints, conference abstracts, books, and book chapters were not included in the review. Any review articles were also avoided in the investigative team's final analysis.

In their examination of off-label use of topical ruxolitinib articles, the team did not include already available studies on the medication's use in atopic dermatitis—a disease for which the cream is approved in the US but not yet in Europe or the UK. This was given the extensive body of evidence on its use for that particular disease.

Among the original 170 records screened by the team at first, 112 did not meet their criteria for inclusion. Ultimately, there were 28 studies that had been published between 2012 - 2024 included in the team's analysis. In the variety of findings highlighted by the investigators, data revealed that off-label implementation of ruxolitinib cream was shown to be most commonly reported for lichenoid and granulomatous dermatoses, as well as alopecia areata.

Despite the favorable efficacy outcomes and safety findings on topical ruxolitinib observed in the treatment of lichenoid and granulomatous conditions, the medication's effectiveness in alopecia areata yielded inconsistent results.

Unlike topical corticosteroids (TCS), known to be the most widely prescribed topical anti-inflammatory drugs, ruxolitinib was noted by the investigators as offering a more targeted mechanism of action that TCS. Extended utilization of medium- to high-potency corticosteroids was shown by the investigative team as potentially leading to adverse effects, including skin thinning (atrophy).

Overall, the team noted that topical ruxolitinib appears to be a potentially valuable medication for lichenoid and granulomatous dermatoses. Despite encouraging findings from oral ruxolitinib in alopecia areata, consistent evidence supporting the topical drug's formulation for this disease remains lacking.

The investigators further highlighted that there remains a notable lack of comparative analyses assessing ruxolitinib's performance against other topical treatments or alternative JAK/STAT pathway inhibitors.

“In conclusion, ruxolitinib appears to be a promising topical medication for various dermatologic conditions,” they wrote.1 “To date, there is insufficient data to assess the long-term efficacy and safety of off-label use. Future clinical trials should evaluate ruxolitinib cream as an alternative treatment for skin conditions that are unresponsive to first-line therapies.”

References

1. Spadafora, M., Morsia, S., Di Lernia, V.G., Kaleci, S., Pellacani, G. and Longo, C. (2025), Off-Label Use of Topical Ruxolitinib in Dermatology: A Systematic Literature Review and Current Perspectives. Exp Dermatol, 34: e70095. https://doi.org/10.1111/exd.70095.

2. L. Bokhari and R. Sinclair, “Treatment of Alopecia Universalis With Topical Janus Kinase Inhibitors - a Double Blind, Placebo, and Active Controlled Pilot Study,” International Journal of Dermatology 57 (2018): 1464–1470.

3. C. B. Bayart, K. L. DeNiro, L. Brichta, B. G. Craiglow, and R. Sidbury, “Topical Janus Kinase Inhibitors for the Treatment of Pediatric Alopecia Areata,” Journal of the American Academy of Dermatology 77 (2017): 167–170.