Pediatric patients with spinal muscular atrophy (SMA) in China face significant challenges in their daily lives. A recent study delved into the psychometric properties of three widely used quality of life measures: the EQ-5D-Y-3L, PROMIS-25, and PedsQL 4.0. This research aims to understand which tool best captures the health-related quality of life for these young patients, providing insights that could influence both clinical assessments and policy decisions regarding treatment accessibility and market access for new therapies.
Methodology and Data Collection
Data for the study were collected through a web-based cross-sectional survey, involving parents of pediatric SMA patients aged 5 to 12. Parents completed proxy-reported versions of the EQ-5D-Y-3L, PROMIS-25, and PedsQL 4.0 questionnaires. Additionally, socio-economic and health status information was gathered to provide a comprehensive overview of the patients’ conditions. The study assessed various psychometric properties including ceiling and floor effects, factorial structure, convergent validity, and known-group validity of the three measures.
Key Findings and Analysis
The survey revealed significant floor effects in the physical function components of PROMIS-25 (41.3%) and PedsQL 4.0 (67.8%). For the EQ-5D-Y-3L, a notable 84.6% of respondents reported severe problems in “walking” and “looking after myself” dimensions. Despite these findings, minimal ceiling or floor effects were observed for the EQ-5D-Y-3L index value. Confirmatory factor analysis supported a six-factor structure for PROMIS-25 but not a four-factor structure for PedsQL 4.0. All hypothesized correlations among the dimensions of the three measures were confirmed, indicating a range of coefficients from 0.28 to 0.68. Analysis of variance showed that EQ-5D-Y-3L outperformed the other two measures in demonstrating known-group validity in 14 out of 16 comparisons.
Practical Inferences
The study offers several practical inferences for clinicians and policymakers:
- EQ-5D-Y-3L may be the most reliable measure for assessing quality of life in pediatric SMA patients.
- PROMIS-25 shows strong factorial validity but significant floor effects, limiting its usefulness in some contexts.
- Given the high floor effects, PedsQL 4.0 may be less effective for capturing physical health dimensions.
- Market access strategies should consider these findings to ensure the most accurate patient-reported outcomes are used in clinical trials and treatment efficacy studies.
The study concludes that the EQ-5D-Y-3L demonstrated superior discriminant power compared to PROMIS-25 and PedsQL 4.0, particularly in the context of known-group validity. The substantial floor effects observed in the physical health components across all three measures highlight the need for more refined tools to capture the nuanced experiences of pediatric SMA patients. These insights could be pivotal in enhancing the precision of health-related quality of life assessments, thereby facilitating better clinical decision-making and improved market access for innovative treatments.
Original Article:
Health Qual Life Outcomes. 2024 Jun 27;22(1):50. doi: 10.1186/s12955-024-02264-9.
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