Seneschal Julien, Guyon Mathilde, Merhi Ribal, Mazereeuw-Hautier Juliette, Andreu Nicolas, Cazenave Sarah, Ezzedine Khaled, Passeron Thierry, Boniface Katia
CHU de Bordeaux, Department of Dermatology and Pediatric Dermatology, National Reference Center for Rare Skin Diseases, Hôpital Saint-André, UMR 5164, Bordeaux, France.
CNRS, Immuno ConcEpT, UMR 5164, University Bordeaux, Bordeaux, France.
JAMA Dermatol. 2025 Apr 1;161(4):375-382. doi: 10.1001/jamadermatol.2024.5737.
Vitiligo is a chronic autoimmune disorder leading to skin depigmentation and reduced quality of life (QOL). Patients with extensive and very active disease are the most difficult to treat.
To assess the efficacy and adverse events of baricitinib combined with narrowband UV-B in adults with severe, active, nonsegmental vitiligo.
DESIGN, SETTING, AND PARTICIPANTS: This academic, multicenter, double-blind, noncomparative randomized clinical trial was conducted at 4 dermatology departments between July 2021 and April 2023 and included adult patients with extensive and active nonsegmental vitiligo. The study was designed to evaluate the effect of baricitinib plus narrowband UV-B based solely on the results from this experimental group. The placebo group was used as a calibration group. Data were analyzed from August to November 2023.
Participants were randomized 3:1 to baricitinib, 4 mg per day, or placebo for 36 weeks alone for the first 12 weeks and then in combination with narrowband UV-B twice a week from weeks 12 to 36.
The primary outcome was mean percentage change in total Vitiligo Area Scoring Index (VASI) score from baseline to week 36 (baricitinib group). The prespecified aim of the study was to show that the reduction in the baricitinib plus narrowband UV-B was significantly greater than 42.9%, a repigmented surface threshold previously observed in patients treated with narrowband UV-B alone. Adverse events and secondary outcomes of change in disease activity and QOL were assessed. Post hoc analyses were additionally performed.
Of 49 included patients, 35 (71%) were female, and the median (IQR) age was 49.9 (38.4-59.8) years. A total of 37 patients were randomized to the baricitinib group and 12 to the placebo group. The mean change in total VASI at week 36 was -44.8% (95% CI, -58.4% to -31.3%) for the baricitinib group and -9.2% (95% CI, -27.7% to 24.7%) for the placebo group. This was not significantly greater than the sufficient repigmented surface threshold of 42.9%. Post hoc analyses showed a significant difference at week 36 for total VASI score in the baricitinib plus narrowband UV-B group compared with placebo plus narrowband UV-B (-44.8% vs -9.2%, respectively; P = .02). There was a greater improvement in disease activity and QOL in the baricitinib group vs placebo group and no significant difference in the number of adverse events.
This proof-of-concept randomized clinical trial confirmed the efficacy of baricitinib combined with narrowband UV-B in the treatment of patients with extensive and active vitiligo.
ClinicalTrials.gov Identifier: NCT04822584.
白癜风是一种慢性自身免疫性疾病，会导致皮肤色素脱失并降低生活质量（QOL）。患有广泛性且病情非常活跃的疾病的患者最难治疗。
评估巴瑞替尼联合窄谱中波紫外线（NB-UVB）治疗重度、活动性、非节段性白癜风成人患者的疗效和不良事件。
设计、设置和参与者：这项学术性、多中心、双盲、非对照随机临床试验于2021年7月至2023年4月在4个皮肤科进行，纳入了患有广泛性和活动性非节段性白癜风的成年患者。该研究旨在仅根据该实验组的结果评估巴瑞替尼加窄谱中波紫外线的效果。安慰剂组用作校准组。于2023年8月至11月进行数据分析。
参与者按3:1随机分为巴瑞替尼组（每日4毫克）或安慰剂组，前12周单独用药36周，然后从第12周开始至第36周每周联合窄谱中波紫外线治疗两次。
主要结局是从基线到第36周（巴瑞替尼组）白癜风总面积评分指数（VASI）总分的平均变化百分比。该研究预先设定的目标是表明巴瑞替尼加窄谱中波紫外线的色素再生率显著高于42.9%，这是之前仅接受窄谱中波紫外线治疗的患者所观察到的色素再生表面阈值。评估了不良事件以及疾病活动度和生活质量变化的次要结局。还进行了事后分析。
纳入的49例患者中，35例（71%）为女性，年龄中位数（四分位间距）为49.9（38.4 - 59.8）岁。共有37例患者随机分配至巴瑞替尼组，12例至安慰剂组。巴瑞替尼组在第36周时VASI总分的平均变化为-44.8%（95%置信区间，-58.4%至-31.3%），安慰剂组为-9.2%（95%置信区间，-27.7%至24.7%）。这并不显著高于足够的色素再生表面阈值42.9%。事后分析显示，在第36周时，巴瑞替尼加窄谱中波紫外线组的VASI总分与安慰剂加窄谱中波紫外线组相比有显著差异（分别为-44.8%对-9.2%；P = 0.02）。与安慰剂组相比，巴瑞替尼组的疾病活动度和生活质量改善更大，不良事件数量无显著差异。
这项概念验证随机临床试验证实了巴瑞替尼联合窄谱中波紫外线治疗广泛性和活动性白癜风患者的疗效。
ClinicalTrials.gov标识符：NCT04822584。
