Huang Fan, Hu Dingyuan, Fan Huaying, Hu Binyi, Liu Yian, Dong Wenliang, Liu Xiangxing, Li Yanting, Yan Diqin, Ding Rui, Niu Suping, Chen Liming, Nie Xiaoyan, Fang Yi
Clinical Trial Institution, Peking University People's Hospital, Beijing, China.
Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing, China.
Clin Pharmacol Ther. 2025 Mar;117(3):659-669. doi: 10.1002/cpt.3538. Epub 2024 Dec 23.
Although several case reports and small clinical trials have reported promising outcomes with Janus kinase (JAK) inhibitors for vitiligo, high-quality evidence and guidelines are lacking. We evaluated the efficacy and safety of JAK inhibitors for the treatment of vitiligo using a meta-analysis of randomized controlled trials (RCTs). We searched the PubMed, Embase, and Cochrane Library databases up to August 2023, with additional studies from ClinicalTrials.gov and company websites. We assessed outcomes, including percentage improvement in total vitiligo area score index (TVASI) and facial vitiligo area score index (FVASI); the proportion of patients achieving 50% improvement in TVASI (TVASI50) and 50% and 75% improvement in FVASI (FVASI50 and FVASI75); the risk of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), infections, and skin-related adverse events (AEs). Five studies with 1,550 participants were included. JAK inhibitors were associated with a higher proportion of TVASI50 (relative risk [RR] 2.67, 95% confidence interval [CI] 1.24-5.78) and FVASI75 (RR 3.97, 95%CI 2.62-6.02) responders than placebo. JAK inhibitors significantly increased the risk of skin-related AEs (RR 1.96, 95% CI 1.29-2.98) compared with placebo. However, the risk of TEAEs, SAEs, and infections was not significantly different between the JAK inhibitor and placebo groups. Subgroup analysis showed that JAK1 and JAK1/2 inhibitors were more effective than JAK3 inhibitors. However, there was insufficient evidence to suggest that the route of administration affects the efficacy and safety of JAK inhibitors in vitiligo. These findings indicate that JAK inhibitors are effective in repigmentation and well tolerated in patients with vitiligo.
尽管有几例病例报告和小型临床试验表明,Janus激酶(JAK)抑制剂治疗白癜风取得了令人鼓舞的结果,但仍缺乏高质量的证据和指南。我们通过对随机对照试验(RCT)进行荟萃分析,评估了JAK抑制剂治疗白癜风的疗效和安全性。我们检索了截至2023年8月的PubMed、Embase和Cochrane图书馆数据库,并从ClinicalTrials.gov和公司网站获取了其他研究。我们评估了各项结果,包括白癜风总面积评分指数(TVASI)和面部白癜风面积评分指数(FVASI)的改善百分比;TVASI改善50%(TVASI50)以及FVASI改善50%和改善75%(FVASI50和FVASI75)的患者比例;治疗中出现的不良事件(TEAE)、严重不良事件(SAE)、感染和皮肤相关不良事件(AE)的风险。纳入了五项研究,共1550名参与者。与安慰剂相比,JAK抑制剂治疗的TVASI50(相对风险[RR]2.67,95%置信区间[CI]1.24 - 5.78)和FVASI75(RR 3.97,95%CI 2.62 - 6.02)应答者比例更高。与安慰剂相比,JAK抑制剂显著增加了皮肤相关AE的风险(RR 1.96,95%CI 1.29 - 2.98)。然而,JAK抑制剂组和安慰剂组之间TEAE、SAE和感染的风险没有显著差异。亚组分析表明,JAK1和JAK1/2抑制剂比JAK3抑制剂更有效。然而,没有足够的证据表明给药途径会影响JAK抑制剂治疗白癜风的疗效和安全性。这些发现表明,JAK抑制剂在白癜风患者的色素再生方面有效且耐受性良好。