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Medically reviewed by Dr. Shani S. Saks, D.O. on August 2, 2023

Two phase 3 trials report significant repigmentation of vitiligo lesions over 52 weeks, though adverse events were common.

Vitiligo is a chronic autoimmune disease characterized by skin depigmentation. Ruxolitinib, an inhibitor of Janus kinase 1 and 2, achieved repigmentation during a phase 2 trial with adult vitiligo patients. This study, published in The New England Journal of Medicine, evaluated the efficacy and safety of ruxolitinib cream in treating nonsegmental vitiligo through two phase 3 randomized, controlled trials (TRuE-V1 and TRuE-V2) conducted in North America and Europe.

The trials included 674 patients aged 12 years or older. All participants had nonsegmental vitiligo that covered 10% or less of their total body surface area. Patients were randomly assigned in a 2:1 ratio to apply either 1.5% ruxolitinib cream or vehicle control twice daily on all vitiligo-affected areas of their face and body for the first 24 weeks, after which, all patients were instructed to apply 1.5% ruxolitinib cream until week 52. 

The primary endpoint noted was a decrease of at least 75% from baseline in the facial Vitiligo Area Scoring Index (F-VASI), or F-VASI75 response, at week 24. Key secondary endpoints included improved responses on the Vitiligo Noticeability Scale.

Primary and Secondary Endpoints Achieved but With High Rate of Adverse Events

Among the 674 enrolled patients, 330 were assigned to TRuE-V1, and 344 were assigned to TRuE-V2. 

  • In TRuE-V1, 29.8% of patients in the ruxolitinib-cream group achieved a F-VASI75 response at week 24, compared to 7.4% in the vehicle group (relative risk, 4.0; 95% confidence interval [CI], 1.9 to 8.4; P<0.001). 
  • In TRuE-V2, the percentages were 30.9% and 11.4%, respectively (relative risk, 2.7; 95% CI, 1.5 to 4.9; P<0.001). 
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Ruxolitinib cream demonstrated superiority over vehicle control for key secondary endpoints. 

Adverse side effects were reported in 54.8% of patients in TRuE-V1 and 62.3% in TRuE-V2, with the most common side effects being: 

  • Application-site acne (6.3% and 6.6%, respectively)
  • Nasopharyngitis (5.4% and 6.1%, respectively)
  • Application-site pruritus (5.4% and 5.3%, respectively). 

Conclusion and Recommendations

Application of ruxolitinib cream resulted in significant repigmentation of vitiligo lesions compared to vehicle control through 52 weeks in two phase 3 trials. However, the treatment was associated with application-site acne and pruritus. 

The findings from these phase 3 trials provide promising evidence of the efficacy of ruxolitinib cream in the repigmentation of vitiligo lesions. The substantial improvement observed in F-VASI75 response and key secondary endpoints indicates the potential clinical benefit of this treatment option for patients with nonsegmental vitiligo. However, the occurrence of adverse events, such as application-site acne and pruritus, underscores the need for careful monitoring and management of side effects during treatment.

To effectively establish ruxolitinib cream as a viable long-term treatment option, larger and longer trials are warranted to gain a comprehensive understanding of its efficacy and safety profile. As the research continues to evolve, the hope is that ruxolitinib cream will provide a promising therapeutic approach for patients with vitiligo, ultimately improving their quality of life and offering a novel avenue for managing this challenging autoimmune skin disorder.

 

Source:

Rosmarin, D., Passeron, T., Pandya, A. G., Grimes, P. E., Harris, J. E., Desai, S. R., Lebwohl, M., Ruer-Mulard, M., Seneschal, J., Wolkerstorfer, A., Kornacki, D., Sun, K., Butler, K., & Ezzedine, K. (2022). Two phase 3, randomized, controlled trials of ruxolitinib cream for vitiligo. The New England Journal of Medicine, 387(16), 1445–1455. https://doi.org/10.1056/nejmoa2118828 

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