Epidermolysis Bullosa Simplex Disease Activity and Response Tool (EBSdart): Scoring Clinical Severity and Outcomes in Epidermolysis Bullosa Simplex
Stanford University School of Medicine
Stanford University School of Medicine
Overview
Epidermolysis Bullosa (EB) is a devastating hereditary blistering disease divided into subtypes based on unique mutations and location of affected skin areas. While there have been major breakthroughs for severe dystrophic EB, there is currently no FDA approved treatment for EB simplex (EBS), the most common subtype. Several early-phase EBS clinical trials have failed to obtain approval, which is partially due to a lack of appropriate scoring instruments to measure changes in disease severity. While tools to measure EB severity exist, such measurements are not suitable for EBS, creating challenges in presenting data to the FDA that reflects patients’ clinical outcomes during studies. Our aim is to develop and validate a new instrument for EBS clinical research by modifying existing tools, incorporating clinical experience from recent studies, and create standardized photographic representations of different disease severities before sending test photographs to key EBS opinion leaders for refinement and reliability analysis.
Status
This project was funded through a 2022 PeDRA Research Fellowship Grant and has resulted in one publication titled, Development and Validation of a Scale to Assess Epidermolysis Bullosa Simplex Severity, published in JAMA Dermatology in 2025. Learn more about this project in a PeDRA Pearls interview with the investigators, where they discuss their mentorship relationship and the process behind creating this assessment tool—and its significance for patients.
