ArticleHealth and quality of life outcomes2023
Measurement properties of the EQ-5D-5L and PROPr in patients with spinal muscular atrophy.
Article in Health and quality of life outcomes, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
5 citing papers in PubMed, 6 citations in OpenAlex.
- Mapping Health State Utility from Disease-Specific Measures in Spinal Muscular Atrophy and Paroxysmal Nocturnal Hemoglobinuria.PharmacoEconomics · 2026Article
- Remaining Burden of Spinal Muscular Atrophy Among Treated Patients: A Survey of Patients and Caregivers.Annals of clinical and translational neurology · 2025Article
- Comparing EQ-5D-5L, PROPr, SF-6D and TTO utilities in patients with chronic skin diseases.The European journal of health economics : HEPAC : health economics in prevention and care · 2025Article
- Healthcare Resource Utilization, Economic Burden, and Multi-Level Medical Security System for Individuals with Spinal Muscular Atrophy in Shaanxi Province, China.Healthcare (Basel, Switzerland) · 2025Article
- Health-related quality of life of adults with spinal muscular atrophy: insights from a nationwide patient registry in Germany.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2024Article
Corrections and comments
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Authors and funding
7 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesSpinal muscular atrophy (SMA) is a rare monogenic neuromuscular disorder caused by loss of function mutations. Measuring health-related quality of life to support economic evaluations in this population is encouraged. However, empirical evidence on the performance of preference-based measures (PBMs) in individuals with SMA is limited. This study aimed to assess the psychometric properties of the EQ-5D-5L and the Patient-Reported Outcomes Measure Information System Preference measure (PROPr) in individuals with SMA.
methodsThe data used in this study were obtained via a web-based, cross-sectional survey. All participants completed the self-reporting EQ-5D-5L and PROMIS-29 questionnaires. Information about their socioeconomic and health status was also obtained. Ceiling and floor effects, convergent and divergent validity, known-group validity, and the agreement between the two measures were assessed.
resultsStrong ceiling and floor effects were observed for four dimensions of the EQ-5D-5L and three subscales, including pain intensity, pain interference, and physical function, of the PROMIS-29. All hypothesized associations between EQ-5D-5L/PROMIS-29 and other neuromuscular questions were confirmed, supporting good convergent validity. Moreover, both EQ-5D-5L and PROPr scores differentiated between impaired functional groups, demonstrating good discriminative ability. Poor agreement between the EQ-5D-5L and PROPr utility scores was observed.
conclusionsThe EQ-5D-5L and PROPr both appear to be valid PBMs for individuals with SMA. However, PROPr yielded considerably lower utility scores than EQ-5D-5L and their agreement was poor. Therefore, these two PBMs may not be used interchangeably in economic evaluations of SMA-related interventions.
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