Vitiligo, a chronic skin disease affecting 1-2% of the global population, is associated
with significant impairment in quality of life. Current pharmacological treatment
options have limited efficacy and considerable side effects. Recent studies have shown
promising results when using Janus kinase inhibitors (JAKis). Despite these favourable
findings, there remains a critical need for comprehensive data on the efficacy and
safety of JAKi in the treatment of vitiligo.Three databases were searched for studies
on patients with vitiligo treated with oral or topical JAKi, with or without conventional
therapy. Placebo or vehicle cream were comparators in randomised controlled trials
(RCTs). Outcomes included a 75% improvement in Facial-Vitiligo Area Scoring Index
(F-VASI), mean Vitiligo Area Scoring Index (VASI) improvement, repigmentation percentage
and adverse events. We performed three analyses: one using RCT data, one from case
reports and a novel cohort of JAKi-treated patients from case reports. The protocol
is registered with PROSPERO (CRD42023445503).Among the 35 articles identified, 19
were included in the statistical analyses. A meta-analysis of three randomised controlled
trials (RCTs) on topical Janus kinase inhibitors (JAKis) suggested that patients treated
with JAKi were more likely to achieve Facial Vitiligo Area Scoring Index 75 (F-VASI75)
than those using vehicle cream (risk ratio (RR) 3.47, 95% confidence interval (CI)
0.98-12.22), with no significant difference in adverse events between groups (RR 1.27;
95% CI 0.88-1.82). A meta-analysis of four single-arm trials showed a 43.8% mean Vitiligo
Area Scoring Index (VASI) improvement (95% CI 0.71-0.93). A cohort (n = 28) from case
reports and series revealed significant repigmentation increases of 48.7% and 63.7%
(p = 0.0018; p < 0.001) in patients treated with JAKi alone or with narrowband ultraviolet
B (UVB). However, data were insufficient to determine if combination treatments were
superior to JAKi alone.Our systematic review evaluated the efficacy and safety of
JAKi for vitiligo using data from RCTs, single-arm trials and case reports. While
topical ruxolitinib showed promising but non-significant results in RCTs, single-arm
trials and case studies highlighted significant repigmentation, particularly with
oral JAKis combined with other therapies. Oral JAKis showed effectiveness but require
caution due to potential adverse effects such as immune suppression and cardiovascular
risks. Furthermore, it is important to acknowledge that a considerable proportion
of patients do not respond to these therapies. Additional RCTs are needed to address
long-term safety, optimise application strategies and establish standardised endpoints
for combination therapies.
