Natural History Study of Recessive Dystrophic Epidermolysis Bullosa Wounds and Patient Reported Outcomes Using Mobile Application Home Photography

• DOI: 10.21203/rs.3.rs-3876810/v1
• Publication Date: 2024-03-26T18:16:53Z
• Authors: Shivali Fulchand; Jaron Nazaroff; Nicki Harris; Jodi So; Edward Eid; Shufeng Li; Ying Lu; Jean Tang

Abstract Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare, blistering genetic disease where wounding and wound pain are the most commonly reported problems by patients. The natural history of RDEB wounds has not been prospectively studied, thus limiting design clinical trial endpoints. Objective: To determine differences in spontaneous closure between chronic open vs recurrent wounds. Methods: We conducted prospective observational study which participants used mobile application to upload weekly photographs multiple target for up 6 months associated itch (Photo Cohort). also utilized separate dataset placebo-treated from previously completed (Validation Cohort) validate characteristics rates 13 single academic center were enrolled Photo Cohort while data 57 was leveraged Validation . Results: For Cohort, 734 photos collected 69 wounds, 42 (73.7%) Chronic larger, more painful, much less likely experience (17% 100% P<0.001) with higher time-to-closure (25.7 weeks vs. 5.7 weeks, P<0.0001) than during follow-up. Baseline size sole predictor (12.8, 95% CI: 3.3-48, P<0.01., Findings recapitulated those (26% had 86% P<0.001, 14.6 8 wounds). In multivariable models, type 29-fold greater likelihood close spontaneously compared (P=0.0045). Conclusions: tend be have lower probability closure, longer distinction types can predict guide selection observation trials.