Measurement properties of the EQ-5D-5L and PROPr in patients with spinal muscular atrophy.

ABSTRACT:

Objectives

Spinal 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.

Methods

The 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. Ce

SUBMITTER: Xu RH 

PROVIDER: S-EPMC10647137 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature