Evidence of a disability paradox in patient‐reported outcomes in haemophilia

O’Hara, J; Martin, AP; Nugent, D; Witkop, M; Buckner, TW; Skinner, MW; O’Mahony, B; Mulhern, B; Morgan, G; Li, N; Sawyer, EK

Evidence of a disability paradox in patient‐reported outcomes in haemophilia

O’Hara, J Martin, AP Nugent, D Witkop, M Buckner, TW Skinner, MW O’Mahony, B Mulhern, B

Morgan, G Li, N Sawyer, EK

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Publisher:: Wiley
Publication Type:: Journal Article
Citation:: Haemophilia, 2021, 27, (2), pp. 245-252
Issue Date:: 2021-02-17

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Field	Value	Language
dc.contributor.author	O’Hara, J
dc.contributor.author	Martin, AP
dc.contributor.author	Nugent, D
dc.contributor.author	Witkop, M
dc.contributor.author	Buckner, TW
dc.contributor.author	Skinner, MW
dc.contributor.author	O’Mahony, B
dc.contributor.author	Mulhern, B https://orcid.org/0000-0003-3656-8063
dc.contributor.author	Morgan, G
dc.contributor.author	Li, N
dc.contributor.author	Sawyer, EK
dc.date.accessioned	2021-08-30T01:02:37Z
dc.date.available	2021-02-08
dc.date.available	2021-08-30T01:02:37Z
dc.date.issued	2021-02-17
dc.identifier.citation	Haemophilia, 2021, 27, (2), pp. 245-252
dc.identifier.issn	1351-8216
dc.identifier.issn	1365-2516
dc.identifier.uri	http://hdl.handle.net/10453/150259
dc.description.abstract	<h4>Introduction</h4>People with inherited and long-term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox).<h4>Aim</h4>To investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population.<h4>Methods</h4>We conducted a discrete choice experiment including duration to capture valuations of health states based on patient-reported preferences. Participants indicated their preferences for hypothetical health states using the EQ-5D-5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional-logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ-5D-5L health states.<h4>Results</h4>After removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ-5D-5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity.<h4>Conclusion</h4>Our findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	Wiley
dc.relation.ispartof	Haemophilia
dc.relation.isbasedon	10.1111/hae.14278
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	1103 Clinical Sciences
dc.subject.classification	Cardiovascular System & Hematology
dc.title	Evidence of a disability paradox in patient‐reported outcomes in haemophilia
dc.type	Journal Article
utslib.citation.volume	27
utslib.location.activity	England
utslib.for	1103 Clinical Sciences
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Business
pubs.organisational-group	/University of Technology Sydney/Strength - CHERE - Centre for Health Economics Research and Evaluation
utslib.copyright.status	open_access	*
dc.date.updated	2021-08-30T01:02:35Z
pubs.issue	2
pubs.publication-status	Published
pubs.volume	27
utslib.citation.issue	2

Abstract:

Introduction

People with inherited and long-term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox).

Aim

To investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population.

Methods

We conducted a discrete choice experiment including duration to capture valuations of health states based on patient-reported preferences. Participants indicated their preferences for hypothetical health states using the EQ-5D-5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional-logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ-5D-5L health states.

Results

After removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ-5D-5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity.

Conclusion

Our findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.

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http://hdl.handle.net/10453/150259