OBJECTIVES: Muscular dystrophies (MD) are a family of genetic muscle diseases manifesting with progressive weakness and loss of muscle mass. The disease poses a considerable burden not only on patients but also on their caregivers. This study evaluates the socioeconomic impact of MD, primarily from the perspective of caregivers, and assesses the accessibility of health and social care services for MD patients in the Czech Republic.

METHODS: A cross-sectional questionnaire-based study was conducted among Czech MD patients and their caregivers. The questionnaire consisted of six sections: 1)patients’ demographic and clinical characteristics, 2)caregivers’ demographics, 3)disease-related activities, 4)out-of-pocket costs, 5)use of social care resources, and 6)use of medical devices.

RESULTS: A total of 80 patients completed the questionnaire, representing 23% of the total MD population in the Czech Republic and majority having Duchenne muscular dystrophy (79%). The mean age of the patients was 13 years, while the caregivers had a mean age of 43 years. The majority of caregivers (85%) were females, mostly mothers of the patients. The study revealed a significant time burden for caregivers, with an average of nearly four hours spent daily assisting with disease-related activities. Additionally, caregivers spent an average of 29 hours per month in health facilities or traveling to them. Only 17 caregivers (20%) were able to work full-time, with seven caregivers (8%) on maternal leave. The remaining caregivers (72%) either worked part-time or were unemployed, and many cited the patient's care as the reason. Moreover, only 31 caregivers (39%) had regular assistance from another caregiver. MD also incurred substantial out-of-pocket costs: €1320/year on traveling to health facilities and €852/year on medical devices, among other expenses.

CONCLUSIONS: The study highlights the significant socioeconomic burden faced by MD patients and their caregivers and underscores the critical importance of ensuring the availability of adequate health and social care services.