Patient-reported outcomes in neuromuscular disorders – health-related quality of life and psychosocial adjustment in post-polio syndrome and Duchenne muscular dystrophy

Abstract

Neuromuscular disorders (NMDs) have a large impact on many aspects of life. Disabilities caused by an impaired muscle function often can lead to a wide range of secondary problems in daily life and affect psychosocial aspects such as the quality of life and psychosocial adjustment. Due to improvements in symptomatic treatments and increased life expectancy in patients with NMDs, these problems become more and more evident. In order to develop appropriate intervention programs for patients with NMDs, it is very important to evaluate patients’ quality of life and to understand its association to physical functioning thereby improving health outcomes. Furthermore, patients that are at high risk of psychosocial impairments should be detected to be able to start an early intervention. The aim of the present work is to investigate patient-reported outcomes, such as the health-related quality of life (HRQOL) and psychosocial adjustment in patients with post-polio syndrome (PPS) and Duchenne muscular dystrophy (DMD) and their association to motor abilities. First, a prospective observational study in patients with PPS is performed focusing on HRQOL, self-reported impairments and activities of daily living and their association with clinical muscle function outcomes. Afterwards, in a cross-sectional study we extensively study the HRQOL in ambulant and non-ambulant patients with DMD and its association to motor function. Finally, we focus on the psychosocial adjustment in children with DMD and its possible association to parental stress and other sociodemographic and disorder-related items.