Efficacy and safety of upadacitinib in adults and adolescents for treatment of non-segmental vitiligo (Viti-Up): results of two phase 3 randomised controlled studies.
Two phase 3 placebo-controlled trials found that once-daily upadacitinib increased clinically meaningful facial and total-body repigmentation at 48 weeks in adults and adolescents with non-segmental vitiligo, without new safety signals reported during the study period.
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Two phase 3 placebo-controlled trials found that once-daily upadacitinib increased clinically meaningful facial and total-body repigmentation at 48 weeks in adults and adolescents with non-segmental vitiligo, without new safety signals reported during the study period.
Research significance
The trials support upadacitinib as a potential systemic treatment for non-segmental vitiligo; any relevance to pediatric cancer treatment, oncology-associated depigmentation, or antitumor biology would be unsupported inference because no oncology population or outcomes were studied.
Source abstract
BACKGROUND: Non-segmental vitiligo is an autoimmune inflammatory disease characterised by loss of melanocytes leading to skin depigmentation. No systemic therapies have been approved, and additional therapeutic options are needed. Here, we report the efficacy and safety of upadacitinib, a selective once-daily Janus kinase inhibitor, in adults and adolescents with non-segmental vitiligo. METHODS: Viti-Up includes two phase 3, global, multicentre, randomised, double-blind, placebo-controlled studies conducted at 138 institutions in 18 countries in Asia, Europe, North America, and South America. Eligible patients were adults and adolescents aged 12 years or older with non-segmental vitiligo involving the face and body with a Facial Vitiligo Scoring Index (F-VASI) of 0·5 or greater and Total Vitiligo Area Scoring Index (T-VASI) of 5 to less than 50. Patients were randomly assigned (2:1) to receive either upadacitinib 15 mg or placebo orally once daily. The coprimary efficacy endpoints for both studies were T-VASI 50 (≥50% reduction in T-VASI score from baseline) at week 48 and F-VASI 75 (≥75% reduction in F-VASI score from baseline) at week 48 (ie, clinically meaningful repigmentation). Efficacy endpoints were analysed in the intention-to-treat population; safety endpoints were assessed in all randomly assigned patients who received at least one dose of study drug during the 48-week double-blind period. Viti-Up is registered on ClinicalTrials.gov, NCT06118411, and is ongoing. FINDINGS: Between Dec 19, 2023, and Oct 14, 2024, 750 individuals were assessed for eligibility. 308 individuals in Viti-Up-1 (206 assigned to upadacitinib and 102 assigned to placebo; 156 [51%] female and 152 [49%] male) and 306 in Viti-Up-2 (205 assigned to upadacitinib and 101 assigned to placebo; 151 [49%] female and 155 [51%] male) were randomly assigned. At week 48, a greater proportion receiving upadacitinib versus placebo reached the coprimary endpoints of T-VASI 50 (Viti-Up-1: upadacitinib 40 [19%] participants vs placebo six [6%]; Viti-Up-2: upadacitinib 44 [21%] vs placebo six [6%]; p<0·001) and F-VASI 75 (Viti-Up-1: upadacitinib 52 [25%] vs placebo six [6%]; Viti-Up-2: upadacitinib 48 [23%] vs placebo seven [7%]; p<0·001). There were no reports of adjudicated major adverse cardiovascular events, adjudicated venous thromboembolic events, adjudicated gastrointestinal perforations, active tuberculosis, lymphoma, non-melanoma skin cancer, or opportunistic infections other than herpes zoster in either study. Safety data were consistent with the overall upadacitinib safety profile. INTERPRETATION: Upadacitinib 15 mg daily provides clinically meaningful repigmentation of both the face and total body for adults and adolescents with non-segmental vitiligo, with no new safety signals identified. FUNDING: AbbVie.