A new clinical tool may improve how physicians assess the severity of a painful and often debilitating skin disorder, according to a study recently published in JAMA Dermatology.
Epidermolysis Bullosa Simplex (EBS) is the most common subtype of Epidermolysis Bullosa (EB), a group of inherited skin conditions characterized by blistering and skin fragility. Despite its prevalence, there is no U.S. Food and Drug Administration-approved treatment for EBS, and clinicians have lacked a standardized method to measure disease severity, until now.
“We don’t have therapies for these patients, so studies like this are about finding ways to improve quality of life and find treatments in the future,” said Amy Paller, MS, MD, ’81, ’83 GME, the chair and Walter J. Hamlin Professor of Dermatology, who was a co-author of the study. “Previously, drug companies conducting clinical trials had no specific tool to measure this.”
In the current study, investigators from medical sites across the country analyzed a repository of 130 clinical photographs from 80 genetically confirmed EBS patients, capturing a range of disease severities across five anatomical regions. Nine board-certified dermatologists scored the images using a 5-point scale, assessing features such as blistering, erosions and keratoderma.
They then compared the scales and found there was strong agreement among the scoring dermatologists. Using this consensus, the investigators then developed the Epidermolysis Bullosa Simplex Disease Activity and Response Tool (EBSdart), a scale designed specifically to evaluate the core features of EBS.
The new tool will provide a reliable and disease-specific severity scale, which will enable more accurate monitoring of disease progression and response to future therapies, Paller said.
“Having this standardized tool imparts greater accuracy to the results of clinical trials related to EBS. We hope that our results using this new tool will reflect more meaningful outcomes and a faster track towards FDA approval and commercialization of treatments,” Paller said.
Building off the new scoring system, Paller and her collaborators are conducting multiple clinical trials using the tool, which they hope will accelerate the development of new treatments for the condition.
The study was supported by a Pediatric Dermatology Research Alliance fellowship.
