309 Losartan treatment improves recessive dystrophic Epidermolysis bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) manifests with blistering and erosions of the skin mucous membranes due to mutations in COL7A1. The repetitive wounding healing processes lead extensive cutaneous scarring. scarring is driven by inflammatory processes, particularly TGF-b signaling pathways, resulting deposition extracellular matrix, especially collagen. There currently no effective or specific treatment for RDEB. Losartan, an angiotensin II type 1 receptor antagonist, inhibitor signaling. Previous preclinical studies hypomorphic Col7A1 mice recapitulating features RDEB have suggested that losartan may improve clinical In this case series, we assessed effects on histopathologic seven patients RDEB; 3F/4M, age 18.1+9.1 years. diagnosis was based characteristic presence biallelic loss-of-function Daily oral administration (0.7 mg/kg) six weeks resulted subjective improvement features, as judged treating physicians patients, severity disease improved Birmingham Epidermolysis Bullosa Severity (BEBS) score (30.5+12.8 vs. 23.3+10.4, before after treatment; p=0.018), accompanied quality life, determined EB-QoL questionnaire (24.0+8.1 17.7+5.5; p=0.018). Histopathology selected lesions revealed increased number mast cells, enhanced microvasculature mid lower dermis. width collagen bundles dermis be decreased four samples changed from dense loose appearance. summary, series reports beneficial a potentially novel treatment, along histopathological alterations. Larger randomized trials are required determine long-term efficacy