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Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.

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Title: Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.
Authors: Szabo, Shelagh M. (AUTHOR), Audhya, Ivana F. (AUTHOR), Malone, Daniel C. (AUTHOR), Feeny, David (AUTHOR), Gooch, Katherine L. (AUTHOR)
Source: Quality of Life Research. Mar2020, Vol. 29 Issue 3, p593-605. 13p. 1 Diagram, 4 Charts, 2 Graphs.
Subjects: Duchenne muscular dystrophy, Meta-analysis, Arm
Abstract: Background: Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives.Methods: A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed.Results: From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient-proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified.Conclusion: Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted. [ABSTRACT FROM AUTHOR]
Copyright of Quality of Life Research is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Psychology and Behavioral Sciences Collection
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  Data: Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.
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  Data: <searchLink fieldCode="AR" term="%22Szabo%2C+Shelagh+M%2E%22">Szabo, Shelagh M.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Audhya%2C+Ivana+F%2E%22">Audhya, Ivana F.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Malone%2C+Daniel+C%2E%22">Malone, Daniel C.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Feeny%2C+David%22">Feeny, David</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Gooch%2C+Katherine+L%2E%22">Gooch, Katherine L.</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Quality+of+Life+Research%22">Quality of Life Research</searchLink>. Mar2020, Vol. 29 Issue 3, p593-605. 13p. 1 Diagram, 4 Charts, 2 Graphs.
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  Data: <searchLink fieldCode="DE" term="%22Duchenne+muscular+dystrophy%22">Duchenne muscular dystrophy</searchLink><br /><searchLink fieldCode="DE" term="%22Meta-analysis%22">Meta-analysis</searchLink><br /><searchLink fieldCode="DE" term="%22Arm%22">Arm</searchLink>
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  Label: Abstract
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  Data: <bold>Background: </bold>Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives.<bold>Methods: </bold>A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed.<bold>Results: </bold>From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient-proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified.<bold>Conclusion: </bold>Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Quality of Life Research is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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