ABSTRACT
BACKGROUND: The symptoms of post-polio syndrome (PPS) and its resulting disabilities can affect quality of life and the ability to perform daily activities. No study has comprehensively analysed how various patient-reported outcome measures (PROMs) are associated with objectively assessed physical function in patients with PPS. AIM: To investigate health-related quality of life (HRQOL), self-reported impairments and activities of daily living during 6 months and evaluate their association with clinical muscle function outcomes in individuals with PPS. METHODS: Twenty-seven patients with PPS were included in the study. At baseline and 6 months, patients were administered PROMs measuring HRQOL (WHOQOL-BREF), self-reported impairments related to PPS (SIPP-RS) and activities of daily living (IBM-FRS). Clinical muscle function outcomes included 6 min walking distance (6MWD) and motor function measure (MFM). RESULTS: There were no changes in self-reported impairments (25.52 to 24.93, p = 0.40), activities of daily living (33.89 to 33.30, p = 0.20), 6MWD (391.52 to 401.85, p = 0.30) and MFM (83.87 to 85.46, p = 0.14) during 6 months, while the HRQOL psychological health decreased during this period (76.85 to 72.38, p = 0.05). A strong association was found between activities of daily living and clinical muscle function outcomes (6MWD: ß = 0.02, 95% CI: 0.02;0.03, t = 6.88, p < 0.01; MFM: ß = 0.25, 95% CI: 0.17;0.33, t = 6.69, p < 0.01). Self-reported impairments and HRQOL domains were not associated with the clinical muscle outcomes. CONCLUSIONS: Study findings indicate that objectively measured walking and motor abilities do not reflect patient's perspectives of their HRQOL and impairment due to PPS. More research is needed to assess changes over time and capture clinically meaningful changes in individuals with PPS and to increase the understanding of how the patient's perspective of disability measured by PROMs is related to objectively measured walking and motor abilities. TRIAL REGISTRATION: ClinicalTrials.gov Identifier ( NCT02801071 ) registered June 15, 2016.
ABSTRACT
This cross-sectional study assessed health-related quality of life (HRQOL) in ambulant and nonambulant patients with Duchenne muscular dystrophy, and explored the association between health-related quality of life and clinically assessed motor function. The Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale and PedsQL Neuromuscular module were completed by 34 parent-child dyads. Association between PedsQL scores and overall motor abilities and the transfers and standing posture domain measured by the Motor Function Measure were examined. Child self-reported and parent proxy-reported mean PedsQL scores for children with Duchenne muscular dystrophy were lower than those for healthy children for the physical and psychosocial health-related quality of life. Fifty-six percent of patients reported clinically impaired psychosocial health-related quality of life scores. Several aspects of the generic and disease-specific health-related quality of life in patients with Duchenne muscular dystrophy were positively associated to overall motor function and transfers and standing posture domain. Associations remained stable when adjusted for age and corticosteroid use. The Motor Function Measure is clinically meaningful in the context of a patient's day-to-day life.