Quality of life of individuals with neuromuscular disease: a national cross-sectional survey
Year: 2026
Type: article
Abstract: To describe health utility scores among individuals living with neuromuscular disorders (NMDs) and examine sociodemographic, clinical, and economic factors associated with these outcomes. We conducted a nationwide, cross-sectional survey of individuals with NMDs and caregivers enrolled with Muscular Dystrophy Canada. Health-related quality of life (HRQoL) was assessed in adults using the Health Utilities Index Mark 3 (HUI3) and in minors using parent-proxy responses to the Pediatric Quality of Life Inventory Neuromuscular Module (PedsQL-NMM). Multivariable generalized linear models were used. The sample included 850 adults (mean [SD] age, 56 [16] years, 52% female) and 94 minors (mean [SD] age, 11 [5] years, 44% female). Mean HUI3 score was 0.27 (SD:0.31) and mean PedsQL-NMM score was 45.9 (SD: 18.7). Among adults, lower HUI3 was associated with moderate/severe financial toxicity (RR, 0.43; 95% CI: 0.35, 0.53), longer disease duration (RR per 5-year increase, 0.97; 95% CI: 0.96, 0.99), autoimmune myopathies (RR, 0.87; 95% CI: 0.79, 0.95), Charcot-Marie-Tooth disease or genetic neuropathies (RR, 0.72; 95% CI: 0.60, 0.86), spinal and bulbar muscular atrophy (RR, 0.81; 95% CI: 0.67, 0.98), inability to work (RR, 0.49; 95% CI: 0.37, 0.65), retirement (RR, 0.81; 95% CI: 0.74, 0.89) and early retirement (RR, 0.66; 95% CI: 0.53, 0.83). Among minors, PedsQL-NMM scores were higher at ages 10–14 versus 0–4 years (RR = 1.47, 95% CI: 1.12, 1.93). NMDs are associated with marked reductions in HRQoL. Disease heterogeneity, financial strain, and social factors should be considered in economic evaluations and health policy decision-making for NMDs. • Neuromuscular diseases (NMDs) are chronic, often progressive conditions associated with weakness, functional limitations, and reduced participation in work and daily activities. However, population-based evidence describing health related quality of life (HRQoL) among NMD populations remains limited. • This national study of 850 adults and 94 minors with NMDs demonstrated substantial reductions in HRQoL across age groups (mean HUI3 0.27; PedsQL-NMM 45.9) and identified financial toxicity, employment disruption, disease duration and selected clinical characteristics as major determinants of HRQoL. • Findings highlight the need to incorporate socioeconomic burden and disease heterogeneity into economic evaluations and policy decisions for NMD populations.
Institutions: Ottawa Hospital Research Institute, Centre National de la Recherche Scientifique, Inserm, Hospices Civils de Lyon, Institut NeuroMyoGène, Laboratoire Physiopathologie et Génétique du Neurone et du Muscle, Muscular Dystrophy Canada, University of Ottawa, Children's Hospital of Eastern Ontario, Ottawa Hospital, University of Calgary, Hotchkiss Brain Institute, Université de Sherbrooke, BC Children's Hospital
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: gold
Estimated APC paid: $3,190
OpenAlex ID: W7225815713
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