Presentation (CTI)

OBJECTIVES: Epidermolysis bullosa (EB) is a rare genetic condition causing blistering fragile skin and mucous membranes. Our study explores the socioeconomic burden of the disease and its impact on the quality-of-life (QoL) of patients and their caregivers.
METHODS: A cross-sectional questionnaire-based study was performed among Czech patients and their caregivers followed in the clinical center specialized in EB. We collected demographic, clinical and QoL data: EQ-5D-5L, disease specific QOLEB and ZBI (Zarit burden interview-caregiver burden), out-of pocket costs, foundation or patient organization costs and social security system costs. Productivity loss was assessed using patient’s working/school attendance status and questionnaire WPAI-GH. Disease-related impact on activities was assessed for both the patient and the caregiver.
RESULTS: Fifty EB patients completed the questionnaires (15% of the Czech EB population). The mean EB patient age was 24.9 years, 54.0% participants were male, mean utility from EQ-5D was 0.641 and mean QOLEB score was 13.2, corresponding to a moderate impact on the QoL of EB patients. EB patients on average absented from school 2.5 hours/week and total productivity loss for adults was 29.6%.Twenty-six caregivers for EB patients participated in the study with mean age 40.3 years and most were women (96.2%). The study revealed that disease impacted 64% of caregivers in their ability to work. ZBI scores were equal to 23.6 corresponding to mild to moderate burden. Total monthly out-of-pocket costs related to EB were 2,067 CZK (€85) mainly related to medication, cosmetics and medical supplies (56.7%), transport (28.3%) and rehabilitation (10.8%). Mean annual social security system costs spent on EB patients were 144,500 CZK (€5,967).
CONCLUSIONS: Our study revealed a significant societal burden imposed on EB patients and their caregivers. These costs should therefore not be omitted while assessing innovative and effective EB treatments.