Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks

Abstract Background Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child’s disabilit...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Carolyn E. Schwartz, Roland B. Stark, David Cella, Katrina Borowiec, Katherine L. Gooch, Ivana F. Audhya
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	Duchenne muscular dystrophy Disability Neuromuscular Proxy measurement Validation Item response theory Medicine R
Acceso en línea:	https://doaj.org/article/5adabc4ef23b4480bcb66f6b4683aadc
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:5adabc4ef23b4480bcb66f6b4683aadc
record_format	dspace
spelling	oai:doaj.org-article:5adabc4ef23b4480bcb66f6b4683aadc2021-11-28T12:22:46ZMeasuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks10.1186/s13023-021-02114-71750-1172https://doaj.org/article/5adabc4ef23b4480bcb66f6b4683aadc2021-11-01T00:00:00Zhttps://doi.org/10.1186/s13023-021-02114-7https://doaj.org/toc/1750-1172Abstract Background Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child’s disability accurately and comprehensively because it affects caregiver burden. We aimed to create a condition-specific caregiver proxy-report measure for Duchenne Muscular Dystrophy (DMD) in order to understand the impact of DMD on the caregiver. Drawing on relevant item banks from the Patient-Reported Outcome Measurement Information System (PROMIS), we sought to confirm their reliability and validity in the target sample of DMD caregivers. Methods This web-based study recruited DMD caregivers via Rare Patient Voice, patient-advocacy groups, and word of mouth. Recruitment was stratified by age of the caregiver’s child with DMD, which broadly represents stages of DMD progression: 2–7, 8–12, 13–17, and > 18. Telephone interviews with DMD parent-caregivers pretested possible measures for content validity. The web-based study utilized an algorithm to categorize respondents’ ambulatory status for tailored administration of PROMIS Parent-Proxy items as well as some new items developed based on caregiver interviews. Item response theory analyses were implemented. Results The study sample included 521 DMD caregivers representing equally the four age strata. The proxy-report measure included the following domains: fatigue impact, strength impact, cognitive function, upper extremity function, positive affect, negative affect, sleep-device symptoms, and mobility. The first five domains had strong psychometric characteristics (unidimensionality; acceptable model fit; strong standardized factor loadings; high marginal reliability). Negative Affect, covering anger, anxiety, depressive symptoms, and psychological stress, fit a bifactor model with good model fit, high marginal reliability, and strong factor loadings. The Sleep-device symptoms domain was not unidimensional, and the mobility domain did not have a simple structure due to residual correlations among items at opposite end of the mobility-disability continuum. These two domain scores were retained as clinimetric indices (i.e., uncalibrated scales), to achieve the overall goal of having a content-valid DMD-specific measure across all stages of disease severity. Conclusions The present study derived a DMD-specific proxy-report measure from PROMIS item banks and supplemental items that could potentially be utilized in caregiver research across all stages of the care recipient’s DMD. Future research will focus on assessing the responsiveness and validity of the measure over time and its comparison to DMD patient self-report.Carolyn E. SchwartzRoland B. StarkDavid CellaKatrina BorowiecKatherine L. GoochIvana F. AudhyaBMCarticleDuchenne muscular dystrophyDisabilityNeuromuscularProxy measurementValidationItem response theoryMedicineRENOrphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-14 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Duchenne muscular dystrophy Disability Neuromuscular Proxy measurement Validation Item response theory Medicine R
spellingShingle	Duchenne muscular dystrophy Disability Neuromuscular Proxy measurement Validation Item response theory Medicine R Carolyn E. Schwartz Roland B. Stark David Cella Katrina Borowiec Katherine L. Gooch Ivana F. Audhya Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
description	Abstract Background Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child’s disability accurately and comprehensively because it affects caregiver burden. We aimed to create a condition-specific caregiver proxy-report measure for Duchenne Muscular Dystrophy (DMD) in order to understand the impact of DMD on the caregiver. Drawing on relevant item banks from the Patient-Reported Outcome Measurement Information System (PROMIS), we sought to confirm their reliability and validity in the target sample of DMD caregivers. Methods This web-based study recruited DMD caregivers via Rare Patient Voice, patient-advocacy groups, and word of mouth. Recruitment was stratified by age of the caregiver’s child with DMD, which broadly represents stages of DMD progression: 2–7, 8–12, 13–17, and > 18. Telephone interviews with DMD parent-caregivers pretested possible measures for content validity. The web-based study utilized an algorithm to categorize respondents’ ambulatory status for tailored administration of PROMIS Parent-Proxy items as well as some new items developed based on caregiver interviews. Item response theory analyses were implemented. Results The study sample included 521 DMD caregivers representing equally the four age strata. The proxy-report measure included the following domains: fatigue impact, strength impact, cognitive function, upper extremity function, positive affect, negative affect, sleep-device symptoms, and mobility. The first five domains had strong psychometric characteristics (unidimensionality; acceptable model fit; strong standardized factor loadings; high marginal reliability). Negative Affect, covering anger, anxiety, depressive symptoms, and psychological stress, fit a bifactor model with good model fit, high marginal reliability, and strong factor loadings. The Sleep-device symptoms domain was not unidimensional, and the mobility domain did not have a simple structure due to residual correlations among items at opposite end of the mobility-disability continuum. These two domain scores were retained as clinimetric indices (i.e., uncalibrated scales), to achieve the overall goal of having a content-valid DMD-specific measure across all stages of disease severity. Conclusions The present study derived a DMD-specific proxy-report measure from PROMIS item banks and supplemental items that could potentially be utilized in caregiver research across all stages of the care recipient’s DMD. Future research will focus on assessing the responsiveness and validity of the measure over time and its comparison to DMD patient self-report.
format	article
author	Carolyn E. Schwartz Roland B. Stark David Cella Katrina Borowiec Katherine L. Gooch Ivana F. Audhya
author_facet	Carolyn E. Schwartz Roland B. Stark David Cella Katrina Borowiec Katherine L. Gooch Ivana F. Audhya
author_sort	Carolyn E. Schwartz
title	Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
title_short	Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
title_full	Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
title_fullStr	Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
title_full_unstemmed	Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks
title_sort	measuring duchenne muscular dystrophy impact: development of a proxy-reported measure derived from promis item banks
publisher	BMC
publishDate	2021
url	https://doaj.org/article/5adabc4ef23b4480bcb66f6b4683aadc
work_keys_str_mv	AT carolyneschwartz measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks AT rolandbstark measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks AT davidcella measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks AT katrinaborowiec measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks AT katherinelgooch measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks AT ivanafaudhya measuringduchennemusculardystrophyimpactdevelopmentofaproxyreportedmeasurederivedfrompromisitembanks
_version_	1718408015713927168

Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks

Ejemplares similares