A Real-world Study on the Influencing Factors of Efficacy of Ruxolitinib Cream in Vitiligo

A Real-world Study on the Influencing Factors of the Efficacy of Ruxolitinib Cream in the Treatment of Vitiligo

Vitiligo is a common chronic autoimmune skin disease that can occur on any part of the body and severely impacts patients' quality of life. Current treatment for vitiligo primarily include topical medications, phototherapy, systemic therapy and surgical interventions, but their efficacy is limited, often failing to achieve ideal therapeutic outcomes, and are associated with a high recurrence rate. This study is an investigator-initiated real-world research on the application of ruxolitinib cream for vitiligo treatment, with the primary objective of exploring the factors influencing the efficacy of ruxolitinib cream in treating vitiligo.

Vitiligo is a common chronic autoimmune skin disease characterized by skin depigmentation, resulting in white patches that can occur on any part of the body and significantly impact patients' quality of life. Currently, treatment options for vitiligo primarily include topical medications, phototherapy, systemic therapy and surgery; however, their efficacy is limited, making it difficult to achieve optimal therapeutic outcomes,with a high recurrence rate. Studies have demonstrated that the JAK-STAT signaling pathway plays a critical role in autoimmune diseases and is involved in the pathogenesis of vitiligo. The JAK inhibitor ruxolitinib cream can improve skin lesions in patients with vitiligo. This study is an investigator-initiated real-world study on the use of ruxolitinib cream for the treatment of vitiligo, with the primary objective of investigating the factors influencing the efficacy of ruxolitinib cream in vitiligo treatment. Primary outcome measure: At Week 24, the clinical characteristic differences between the groups achieving and not achieving T-VASI50 [50% improvement of total vitiligo area scoring index (T-VASI)]. Secondary outcome measures:

(1) At Week 12 and Week 24, the proportion of patients scoring 4(slightly noticeable) or 5(no longer noticeable) on the Vitiligo Noticeability Scale (VNS), as well as the distribution of patients within each category; (2) Change from baseline in vitiligo-specific quality-of-life score (vitiligo-specific quality-of-life, VitiQoL) at Week 12 and Week 24; (3) Proportion achieving T-VASI 50/75/90 at Week 12 and Week 24; (4) Proportion achieving F-VASI 50/75/90[facial vitiligo area scoring index(F-VASI)] at Week 12 and Week24.

Inclusion Criteria:

1. Age12 years or older, any gender;

2. Signed"Informed Consent Form";

3. Clinically diagnosed with non-segmental vitiligo;

4. Vitiligo area less than 10% of body surface area;

5. Agreed to discontinue all other vitiligo-related treatments from the screening visit until the final follow-up visit;

6. Female subjects must not plan for pregnancy or oocyte donation from screening until 4 weeks after the last dose and must voluntarily adopt highly effective physical contraception; male subjects must not plan for fertility or sperm donation during the same period and must voluntarily adopt highly effective physical contraception. Methods include:

   • Barrier methods: Condom or barrier cap (diaphragm or cervical cap);
   • Intrauterine device (IUD) or intrauterine system (IUS);
   • Surgical sterilization (bilateral oophorectomy with/without hysterectomy, total hysterectomy, bilateral tubal ligation, or vasectomy) performed ≥6 months prior to the first dose in the subject or their partner;
   • Other investigator-confirmed highly effective physical contraception.

Exclusion Criteria:

1. Allergy to ruxolitinib cream;
2. Pregnant or lactating women;
3. Any condition deemed inappropriate for study participation by the investigator.