Compliance of comprehensive care in children with Duchenne muscular dystrophy: Real-world experience of a pediatric neuromuscular diseases center

ABSTRACT BACKGROUND Although international care considerations for Duchenne muscular dystrophy (DMD) were revised in 2018, compliance in clinical practice remains unknown. The objective of this study was to investigate the parental compliance with care considerations in a tertiary hospital in Türkiye. METHODS A questionnaire was administered to parents of patients with DMD to assess compliance with recommended frequencies of visits to healthcare providers and clinical tests. The data collected from the questionnaires were analyzed using cross-tables to explore if there were differences between ambulant and non-ambulant patients. RESULTS Sixty-one participants completed the questionnaire, most of whom were parents of ambulant patients (n= 44, 72.1%). The majority of parents complied with the recommended frequency of cardiac assessments. However, suboptimal compliance was observed in the rest of the assessments, including the PPSV23 and influenza vaccination rates, which were notably low across all patient groups. Compliance with anthropometric assessments and the use of corticosteroids with proper posology was significantly higher in ambulant patients. Compliance with respiratory and orthopedic evaluations was significantly higher among non-ambulant patients. CONCLUSION This study showed several areas in which compliance is insufficient. To improve compliance, future work should focus on home-based assessments, educational programs for patients, parents, and physicians, as well as raising awareness about the importance and timing of recommended vaccinations