592 Patient-reported outcome measures for chronic itch in epidermolysis bullosa: An exploratory analysis of a randomized controlled trial

Epidermolysis Bullosa (EB) is a rare genodermatosis characterized by skin fragility, blistering, and chronic pruritus. This study evaluated whether existing patient-reported instruments can capture the real-life burden of itch specific to EB. To date, no single instrument considered gold standard for EB-related itch. Using longitudinal data from completed phase 2 trial with 24 participants (NCT03836001), associations Itch Numeric Rating Scale (NRS) Dermatology Life Quality Index (DLQI), ItchyQoL, Leuven (LIS), Patient-Reported Outcomes Information System (PROMIS) were linear mixed model (LMM). LMM predicted following score changes each 1-point increase in Average NRS: 0.45 points DLQI (-0.2-1.1;P=0.2), 2.3 ItchyQoL (95% CI: 0.5-4.1; P=0.01), 3 out 100 LIS-Consequences (1.0-5.1; P=0.005). Predicted T-score four PROMIS domains 1.4 Itch-Interference (0.2-2.5; P=0.03), Mood Sleep (0.9-3.8; P=0.005), 2.9 Scratching Behavior (1.6-4.2; P=0.0005), 1.2 Activity Clothing (-0.06-2.6; P=0.06). Worst NRS was similar relation other instruments. In instrument, median value scratching item highest, sleep disturbance also common. Our results showed that LIS, are well suited assessing patient-centered clinical research practice. PROMIS, particular, may be used as an EB-specific assessment tool, it has assess customizable. warrants further validation larger prospective EB cohorts enable more appropriate method its impact on quality life.