Patient‐reported outcomes and quality of life in dominant dystrophic epidermolysis bullosa: A global cross‐sectional survey

• DOI: 10.1111/pde.14802
• Publication Date: 2021-09-13T10:53:25Z
• Authors: Shivali Fulchand; Nicole Harris; Shufeng Li; Melissa Barriga; Emily Gorell; Mark De Souza; Dedee Murrell; M. Peter Marinkovich; Vamsi Krishna Yen...; Jean Y. Tang
• Keywords: 03 medical and health sciences; Cross-Sectional Studies; 0302 clinical medicine; Quality of Life; Humans; Patient Reported Outcome Measures; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica; 3. Good health

AbstractIntroductionDystrophic epidermolysis bullosa is a debilitating skin condition, without curative treatment. Previous research has focused on the recessive variant, which is known to cause severe disease. Limited work focusing on the clinical manifestations and outcomes of dominant dystrophic epidermolysis bullosa is found (DDEB).MethodsAnalysis of an online survey of 42 DDEB patients.ResultsSelf‐reported severity of disease did not correlate with size of the wound or number of dressing changes, but did correlate with severity of pain reported in the last 12 months (3.4 mild vs 6.8 severe disease, P = 0.0002). Patients with severe DDEB also reported more severe internal disease symptoms, such as difficulty swallowing (62.5%, P = 0.01) and greater analgesic use during dressing changes (4.4% mild vs 81.3% severe, P = <0.001).DiscussionPatient perception of disease severity in DDEB appears to be most impacted by pain, presence of chronic open wounds, difficulty swallowing, difficulty walking, and anal strictures. As research on DDEB increases, future studies focused on these symptoms might be the most impactful for DDEB patients. However, distinguishing DDEB from other subtypes remains a challenge.