Topical 1.5% ruxolitinib cream for facial refractory segmental vitiligo: a retrospective single-center case series

This study hypothesized that topical 1.5% ruxolitinib cream is effective and safe for facial segmental vitiligo (SV), especially during the progressive stage.
In a retrospective review of 40 patients with treatment-resistant facial SV, all applied the cream twice daily for 24 weeks. Patients were grouped as progressive-stage (n=25) or stable-stage (n=15). The main goal was to see how many achieved ≥75% improvement on the Vitiligo Area Scoring Index (VASI75) by week 24.
The results showed that 45% of all patients (18/40) reached this goal, with an average VASI score improvement of 71.3%.
This study found that topical 1.5% ruxolitinib cream is an effective and safe treatment for refractory facial segmental vitiligo (SV). The most notable finding was the significant difference in response based on disease activity: 64% of progressive-stage patients achieved substantial repigmentation (VASI75), compared to only 13% of stable-stage patients. Improvement continued over time, with significantly better results at 24 weeks than at 12 weeks. The treatment was well-tolerated, with only minor side effects. These results suggest that ruxolitinib works best in active, inflammatory disease. For clinicians, this supports its use especially for progressive SV, with the understanding that longer treatment may be needed for maximum benefit and that stable disease may respond less effectively. The favorable safety profile makes it suitable for medium-term facial use.
Limitations to Consider:
The retrospective design and lack of a control group limit the strength of the conclusions.
The small sample size may introduce bias and limits generalizability.
Therefore, these results should be seen as promising real-world evidence that needs confirmation in larger, prospective, randomized controlled trials.

本研究假设，1.5%鲁索替尼乳膏（ruxolitinib cream）外用治疗面部节段型白癜风（segmental vitiligo, SV）安全有效，尤其适用于进展期患者。
本研究对40例难治性面部节段型白癜风患者开展回顾性分析，所有患者均每日外用该乳膏2次，疗程为24周。研究人员将患者按疾病分期分为进展期组（n=25）与稳定期组（n=15）。本研究的主要观测指标为：至24周时，白癜风面积评分指数（Vitiligo Area Scoring Index, VASI）改善≥75%（VASI75）的患者占比。
结果显示，总人群中45%的患者（18/40）达成该观测指标，平均VASI评分改善率达71.3%。
本研究证实，1.5%鲁索替尼乳膏外用是治疗难治性面部节段型白癜风的安全有效方案。本研究最显著的发现为，基于疾病活动度的治疗应答存在显著差异：进展期组64%的患者实现了显著复色（VASI75），而稳定期组仅为13%。治疗应答随时间持续提升，24周时的疗效显著优于12周。该治疗耐受性良好，仅出现轻微不良反应。上述结果提示，鲁索替尼在活动性炎症性白癜风中疗效最佳。对于临床医师而言，本研究支持将该药物用于进展期SV患者，同时需注意：需延长治疗周期以获得最大获益，而稳定期疾病的应答效果相对较差。其良好的安全性使其适用于面部中长期外用治疗。
需注意的研究局限性：
本研究采用回顾性设计且未设置对照组，因此结论的证据强度有限。
样本量较小可能引入偏倚，且限制了研究结果的外推性。
因此，本研究结果可视为颇具前景的真实世界证据，仍需通过更大规模的前瞻性随机对照试验加以验证。