500 - Patient-reported outcomes following 24 weeks of treatment with upadacitinib in adults with non-segmental vitiligo: results from a phase 2, randomized, double-blind, dose-ranging study

Abstract

Abstract Introduction & Objectives Vitiligo has a considerable negative impact on patients’ health-related quality of life (HRQoL). Patients often experience social stigma, low self-esteem, anxiety, and depression. Assessment of HRQoL is therefore recommended as part of the evaluation of disease severity and treatment benefits. Accordingly, the impacts of treatment with upadacitinib (UPA), an oral Janus kinase inhibitor, on a range of patient-reported HRQoL outcomes were evaluated as part of a phase 2, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study in adults with non-segmental vitiligo. Materials & Methods Adults aged 18–65 years with non-segmental vitiligo with a Facial Vitiligo Area Scoring Index score ≥0.5 and a Total Vitiligo Area Scoring Index score ≥5 at baseline were included in this 52-week study (NCT04927975). During a 24-week, double-blind, placebo-controlled treatment period (period 1), patients were randomly assigned to receive UPA 6 mg (UPA6), UPA 11 mg (UPA11), UPA 22 mg (UPA22), or placebo once daily (QD). Patient-reported HRQoL outcomes assessed at the week 24 primary analysis timepoint (ie, end of period 1) included change from baseline in vitiligo QoL (VitiQoL) total score (lower scores reflect better HRQoL), achievement of a Vitiligo Noticeability Scale (VNS) response of 4 (“a lot less noticeable”) or 5 (“no longer noticeable”), change from baseline in Dermatology Life Quality Index (DLQI; lower scores reflect better HRQoL), change from baseline in Hospital Anxiety and Depression Scale (HADS) anxiety and depression scores (where lower scores reflect less anxiety and/or depression), and achievement of a Patient’s Global Impression of Change-Vitiligo (PaGIC-V) response of 1 (“much better”) or 2 (“a little better”). Results A total of 185 patients were enrolled in the study (UPA6 n=49; UPA11, n=47; UPA22, n=43; placebo n=46). Mean reductions in VitiQoL scores from baseline to week 24 with UPA22 (−6.6) and UPA6 (−7.5) were numerically larger than with placebo (−5.5), although not statistically significant. At week 24, more patients treated with UPA22 reported a VNS response of “a lot less noticeable” or “no longer noticeable” than with placebo (11.6% vs 0%, respectively; P < .05). Mean reductions in DLQI scores from baseline to week 24 were significantly larger with UPA22 than with placebo (−2.2 vs −0.6; P < .05). HADS anxiety scores decreased from baseline to week 24 in the UPA6 and UPA22 groups, with similar reductions in HADS depression scores observed for the UPA11 and UPA22 groups; changes in HADS scores were not significantly different vs placebo. A significantly greater proportion of patients achieved a PaGIC-V response of “much better” or “a little better” with any UPA dose vs placebo: UPA6 (34.7%), UPA11 (55.3%), and UPA22 (60.5%) vs placebo (19.6%; P < .05 vs placebo for all UPA doses). Conclusion Among adults with non-segmental vitiligo, 24 weeks of treatment with UPA at all doses resulted in improvements compared to placebo in patient impressions of disease improvement based on PaGIC results. These initial findings suggest that UPA22 may improve patient HRQoL and perceptions of vitiligo noticeability; longer term data from the extension phase of this study will further clarify the impact of UPA treatment on QoL.