Dataset Information

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. Ceiling and floor effects, convergent and divergent validity, known-group validity, and the agreement between the two measures were assessed.

Results

Strong 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.

Conclusions

The 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.

SUBMITTER: Xu RH

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

REPOSITORIES: biostudies-literature

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Publications

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

Xu Richard Huan RH Ma Bin B Xin Huanping H Zhang Huanyu H Zeng Yan Y Luo Nan N Dong Dong D

Health and quality of life outcomes 20231115 1

<h4>Objectives</h4>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 m ...[more]

PMID: 37968716

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Project description:ObjectivesThe EQ-5D-5L and its value sets are widely used internationally. However, in the US and elsewhere, there is growing use of PROMIS, which has a value set (PROPr) based on the stated preferences of the US population. This paper aims to compare the characteristics of EQ-5D-5L and PROPr value sets and to highlight potential implications for users.MethodsUS, Australian and English value sets were used for EQ-5D-5L. PROPr utilities were calculated based on PROMIS-29 + 2. We examined, in each case, (i) the characteristics (e.g. range of values, number of unique values) and distribution of all possible 'theoretical' utilities; (ii) dimension/domain importance ranking by the utility of corner states (i.e. health states with the worst level in one domain and the best in all others); (iii) comparisons of utilities for health states hypothesised to be comparable in terms of severity across EQ-5D-5L descriptive systems and PROMIS-29 + 2 domain scores; (iv) the changes in values of adjacent states (i.e. a one-level change in one dimension for EQ-5D-5L and a four-point change in raw scores for PROMIS-29 + 2, with the other dimensions held constant) for dimensions hypothesised to overlap conceptually or be correlated between the two instruments.ResultsEQ-5D-5L and PROPr utilities differ systematically. First, the US EQ-5D-5L utilities range from - 0.573 to 1, whereas PROPr values for PROMIS-29 + 2 range from - 0.022 to 0.954. Second, in the US (and English) EQ-5D-5L value sets, pain is the most important dimension whereas in PROPr pain is one of the least important (apart from sleep disturbance). Third, classified based on severity across EQ-5D-5L descriptive systems and PROMIS-29 + 2 domain scores, PROPr has substantially lower values than EQ-5D-5L values for comparable 'mild' health states, but higher values for more 'severe' health states. Last, when one dimension is considered across its best to worst levels and all other dimensions are held constant at their best or moderate level, in EQ-5D-5L value sets, the greatest changes in utility occur between levels 3 and 4 (moderate and severe) problems; in PROPr that occurred between the most severe states and their descriptively adjacent health states.ConclusionThere are very considerable differences between US EQ-5D-5L and PROPr utilities, despite both in principle representing utility on the same scale anchored at 0 and 1 and both representing the preferences of the US general public. It is important for decision makers and clinical triallists to be aware of these differences. Further work is needed to assess the impact of these differences in value sets using population and patient data, and in longitudinal settings.

Project description:BackgroundThe EQ-5D-3L and EQ-5D-5L are two generic health-related quality of life measures, which may be used in clinical and health economic research. They measure impairment in 5 aspects of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The aim of this study was to assess the performance of the EQ-5D-3L and EQ-5D-5L in measuring the self-reported health status of older patients with substantial multimorbidity and associated polypharmacy.MethodsBetween 2017 and 2019, we administered EQ-5D-3L and EQ-5D-5L to a subset of patients participating in the OPERAM trial at 6 months and 12 months after enrolment. The OPERAM trial is a two-arm multinational cluster randomised controlled trial of structured medication review assisted by a software-based decision support system versus usual pharmaceutical care, for older people (aged ≥ 70 years) with multimorbidity and polypharmacy. In the psychometric analyses, we only included participants who completed the measures in full at 6 and 12 months. We assessed whether responses to the measures were consistent by assessing the proportion of EQ-5D-5L responses, which were 2 or more levels away from that person's EQ-5D-3L response. We also compared the measures in terms of informativity, and discriminant validity and responsiveness relative to the Barthel Index, which measures independence in activities of daily living.Results224 patients (mean age of 77 years; 56% male) were included in the psychometric analyses. Ceiling effects reported with the EQ-5D-5L (22%) were lower than with the EQ-5D-3L (29%). For the mobility item, the EQ-5D-5L demonstrated better informativity (Shannon's evenness index score of 0.86) than the EQ-5D-3L (Shannon's evenness index score of 0.69). Both the 3L and 5L versions of EQ-5D demonstrated good performance in terms of discriminant validity, i.e. (out of all items of the EQ-5D-3L and EQ-5D-5L, the pain/discomfort and anxiety/depression items had the weakest correlation with the Barthel Index. Both the 3L and 5L versions of EQ-5D demonstrated good responsiveness to changes in the Barthel Index.ConclusionBoth EQ-5D-3L and EQ-5D-5L demonstrated validity and responsiveness when administered to older adults with substantial multimorbidity and polypharmacy who were able to complete the measures.

Project description:BackgroundFor many decision makers in Health Technology Assessment the EQ-5D-5L is the standard measure of health-related quality of life (HRQL). However, evidence has shown the limitations of the measure in certain disease areas, including dermatology. Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss.ObjectivesThis study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C).MethodsData from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed.ResultsData were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3-61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO.ConclusionsThe EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. Data from other measures could be considered if they are shown to be more relevant.

Dataset Information

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

Objectives

Methods

Results

Conclusions

Publications

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

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