【摘要】 目的 评估1.5%芦可替尼乳膏治疗中国非节段型白癜风患者的长期疗效与安全性，并探讨疗效与临床表型的相关性。方法 2023年8月―2025年5月采用单中心观察性研究，患者每日2次外涂1.5%芦可替尼乳膏，依据患者最后1次随访时间节点，将患者分为已治疗12周、24周、40周和52周4个队列，分别评估患者面部白癜风面积评分指数(facial vitiligo area scoring index, F-VASI),以F-VASI较基线下降超过50%/75%/90%(F-VASI 50/75/90)作为疗效评估标准，同时评估不良事件发生率。根据疾病分期、发病年龄、面部白斑面积、家族史等临床特征进行亚组分析。结果 共纳入996名面部非节段型白癜风患者。在治疗24周的队列中，F-VASI 50、F-VASI 75和F-VASI 90患者比例分别为51.05%、29.13%和16.82%;在治疗52周的队列中，F-VASI 50、F-VASI 75和F-VASI 90患者比例分别为69.67%、50.82%和40.98%。亚组分析发现，治疗早期家族史阴性的患者疗效显著(OR=0.52,P=0.034);面部白斑面积<0.5%的患者疗效更优(P<0.05),F-VASI 50患者在12周时显现差异(P<0.01),F-VASI 75/90患者的差异自24周开始显著(P<0.01),到40周和52周时，F-VASI 50/75/90患者与F-VASI<50患者相比差异均显著(均P<0.05);治疗24周的患者在进展期、晚发型(≥12岁发病)疗效显著(P<0.05)。用药期间最常见的不良反应为用药部位的皮肤瘙痒和痤疮，未出现严重的不良反应。结论 本研究为中国人群首次大样本、长周期的1.5%芦可替尼乳膏治疗面部非节段型白癜风患者真实世界研究，结果证实该药治疗安全有效，尤其是家族史阴性、面部小面积患者复色显著。临床表型可作为疗效预测因素，为个体化治疗提供参考。更多还原

【Abstract】 Objective To evaluate the long-term efficacy and safety of 1.5% ruxolitinib cream in Chinese patients with non-segmental vitiligo and to explore the correlation between efficacy and clinical phenotypes. Methods From August 2023 to May 2025, a single-center observational study was conducted. Patients applied 1.5% ruxolitinib cream twice daily. Based on the last follow-up timepoint, patients were stratified into four cohorts: 12-week, 24-week, 40-week, and 52-week treatment groups. Efficacy was assessed using the facial vitiligo area scoring index(F-VASI), with F-VASI 50/75/90(≥50%, ≥75%, and ≥90%) improvement from baseline F-VASI, respectively. Adverse events(AEs) were recorded at the same time. Subgroup analyses were performed according to disease activity, age of onset, facial affected area, family history and other clinical features. Results A total of 996 patients with facial non-segmental vitiligo were ultimately enrolled in this study. In the 24-week cohort, F-VASI 50, F-VASI 75, and F-VASI 90 response rates were 51.05%, 29.13%, and 16.82%, respectively. By week 52, these rates were 69.67%, 50.82%, and 40.98%, respectively. Subgroup analysis revealed that patients with a negative family history showed significantly better efficacy in the early treatment phase(OR=0.52, P=0.034), and patients with a baseline facial affected area of <0.5% achieved superior outcomes in four cohorts(P<0.05). The divergence in treatment response was observed at week 12 for F-VASI 50 responders(P<0.01), extended to F-VASI 75/90 responders by week 24(P<0.01), and became consistently significant for F-VASI 50/75/90 at both week 40 and 52 compared to F-VASI <50(P<0.05); in the 24-week cohort, patients with active disease and late-onset(onset age ≥12 years) showed significantly better efficacy(P<0.05). The most common adverse reactions during treatment were application site pruritus and acne; no serious adverse reactions were reported. Conclusions This study represents the first large-scale, long-term real-world investigation of 1.5% ruxolitinib cream in Chinese patients with non-segmental vitiligo. The results confirm that this treatment is safe and effective, with significant repigmentation particularly in patients with a negative family history and those with small facial affected areas. Clinical phenotypes can serve as predictors of treatment response, providing a reference for individualized therapy.更多还原