Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

Cooperative International Neuromuscular Research Group Investigators

doi:10.1016/j.jval.2021.05.016

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

Cooperative International Neuromuscular Research Group Investigators

Neurology

Research output: Contribution to journal › Article › peer-review

6 Scopus citations

Abstract

Objectives: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately fatal neuromuscular disease. Methods: Patients with DMD were recruited from 20 centers across 9 countries as part of the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (NCT00468832). The psychometric properties of the PedsQL 4.0 GCS were examined using Rasch analysis. Results: In total, 329 patients with DMD (mean age 9 years, range 3–18 years, 75% ambulatory) completed the PedsQL 4.0 GCS. The most difficult instrument items, expressing the greatest loss in health-related quality of life, were those associated with emotional well-being (eg, being teased by other children, feeling sad, and not making friends), as opposed to somatic disability (eg, lifting heavy objects, participating in sports, and running). The mean item and person fit residuals were estimated at 0.301 (SD: 1.385) and −0.255 (1.504), respectively. In total, 87% (20 of 23) of items displayed disordered thresholds, and many exhibited nontrivial dependency. The overall item-trait interaction χ² value was 178 (115 degrees of freedom, P<.001). Our analysis also revealed significant issues with differential item functioning, and by investigating residual principal component loadings, the PedsQL 4.0 GCS total score was found to be multidimensional. Conclusions: The PedsQL 4.0 GCS records information clinically relevant to patients with DMD, but the total scale score may not be fit for purpose as a measure health-related quality of life in this disease population.

Original language	English (US)
Pages (from-to)	1490-1498
Number of pages	9
Journal	Value in Health
Volume	24
Issue number	10
DOIs	https://doi.org/10.1016/j.jval.2021.05.016
State	Published - Oct 2021

Bibliographical note

Funding Information:
Duchenne Natural History Study was funded through grants from the US Department of Education / National Institute on Disability and Rehabilitation Research ( H133B031118 and H133B090001 ), US Department of Defense ( W81XWH-09-1-0592 ), the National Institutes of Health ( UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875 , and RO1AR062380 ), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study.

Funding Information:
Funding/Support: This study was funded by Sarepta Therapeutics Inc.Cooperative International Neuromuscular Research Group (CINRG) Investigators: V. Vishwanathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); S. Chidambaranathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); W. Douglas Biggar (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Laura C. McAdam (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Jean K. Mah (Alberta Children's Hospital, Calgary, AB, Canada); Mar Tulinius (Queen Silvia Children's Hospital, G?teborg, Sweden); Avital Cnaan (Children's National Medical Center, Washington DC, USA); Lauren P. Morgenroth (Children's National Medical Center, Washington DC, USA); Robert Leshner (Children's National Medical Center, Washington DC, USA); Carolina TesiRocha (Children's National Medical Center, Washington DC, USA); Mathula Thangarajh (Children's National Medical Center, Washington DC, USA); Tina Duong (Children's National Medical Center, Washington DC, USA); Andrew Kornberg (Royal Children's Hospital, Melbourne, Australia); Monique Ryan (Royal Children's Hospital, Melbourne, Australia); Yoram Nevo (Hadassah Hebrew University Hospital, Jerusalem, Israel); Alberto Dubrovsky (Instituto de Neurosciencias Fundacion Favaloro, Buenos Aires, Argentina); Paula R. Clemens (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Hoda Abdel-Hamid (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Anne M. Connolly (Washington University in St Louis, St Louis, MO, USA); Alan Pestronk (Washington University in St Louis, St Louis, MO, USA); Jean Teasley (Children's Hospital of Virginia, Richmond, VA, USA); Tulio E. Bertorini (University of Tennessee, Memphis, TN, USA); Richard Webster (Children's Hospital at Westmead, Sydney, Australia); Hanna Kolski (University of Alberta, Edmonton, AB, Canada); Nancy Kuntz (Mayo Clinic, Rochester, MN, USA); Sherilyn Driscoll (Mayo Clinic, Rochester, MN, USA); John B. Bodensteiner (Mayo Clinic, Rochester, MN, USA); Jose Carlo (University of Puerto Rico, San Juan, Puerto, Rico); Ksenija Gorni (University of Pavia and Niguarda Ca' Granda Hospital, Milan, Italy); Timothy Lotze (Texas Children's Hospital, Houston, TX, USA); John W. Day (University of Minnesota, Minneapolis, MN, USA); Peter Karachunski (University of Minnesota, Minneapolis, MN, USA); Erik K. Henricson (University of California, Davis, CA, USA); Richard T. Abresch (University of California, Davis, CA, USA); Nanette C. Joyce (University of California, Davis, CA, USA); Craig M. McDonald (University of California, Davis, CA, USA). Duchenne Natural History Study was funded through grants from the US Department of Education/National Institute on Disability and Rehabilitation Research (H133B031118 and H133B090001), US Department of Defense (W81XWH-09-1-0592), the National Institutes of Health (UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875, and RO1AR062380), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study.

Funding Information:
Funding/Support: This study was funded by Sarepta Therapeutics Inc .

Publisher Copyright:
© 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research

Keywords

Cooperative International Neuromuscular Research Group
disability
patient-reported outcome
psychometric analysis
quality of life

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10.1016/j.jval.2021.05.016

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@article{484d6ca3ee624f97a65bb8dbff1daeac,

title = "Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy",

abstract = "Objectives: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately fatal neuromuscular disease. Methods: Patients with DMD were recruited from 20 centers across 9 countries as part of the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (NCT00468832). The psychometric properties of the PedsQL 4.0 GCS were examined using Rasch analysis. Results: In total, 329 patients with DMD (mean age 9 years, range 3–18 years, 75% ambulatory) completed the PedsQL 4.0 GCS. The most difficult instrument items, expressing the greatest loss in health-related quality of life, were those associated with emotional well-being (eg, being teased by other children, feeling sad, and not making friends), as opposed to somatic disability (eg, lifting heavy objects, participating in sports, and running). The mean item and person fit residuals were estimated at 0.301 (SD: 1.385) and −0.255 (1.504), respectively. In total, 87% (20 of 23) of items displayed disordered thresholds, and many exhibited nontrivial dependency. The overall item-trait interaction χ2 value was 178 (115 degrees of freedom, P<.001). Our analysis also revealed significant issues with differential item functioning, and by investigating residual principal component loadings, the PedsQL 4.0 GCS total score was found to be multidimensional. Conclusions: The PedsQL 4.0 GCS records information clinically relevant to patients with DMD, but the total scale score may not be fit for purpose as a measure health-related quality of life in this disease population.",

keywords = "Cooperative International Neuromuscular Research Group, disability, patient-reported outcome, psychometric analysis, quality of life",

author = "{Cooperative International Neuromuscular Research Group Investigators} and Erik Landfeldt and Joel Iff and Erik Henricson and V. Vishwanathan and S. Chidambaranathan and Biggar, {W. Douglas} and McAdam, {Laura C.} and Mah, {Jean K.} and Mar Tulinius and Avital Cnaan and Morgenroth, {Lauren P.} and Robert Leshner and Carolina TesiRocha and Mathula Thangarajh and Tina Duong and Andrew Kornberg and Monique Ryan and Yoram Nevo and Alberto Dubrovsky and Clemens, {Paula R.} and Hoda Abdel-Hamid and Connolly, {Anne M.} and Alan Pestronk and Jean Teasley and Bertorini, {Tulio E.} and Richard Webster and Hanna Kolski and Nancy Kuntz and Sherilyn Driscoll and Bodensteiner, {John B.} and Jose Carlo and Ksenija Gorni and Timothy Lotze and Day, {John W.} and Karachunski, {Peter I} and Henricson, {Erik K.} and Abresch, {Richard T.} and Joyce, {Nanette C.} and McDonald, {Craig M.}",

note = "Funding Information: Duchenne Natural History Study was funded through grants from the US Department of Education / National Institute on Disability and Rehabilitation Research ( H133B031118 and H133B090001 ), US Department of Defense ( W81XWH-09-1-0592 ), the National Institutes of Health ( UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875 , and RO1AR062380 ), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study. Funding Information: Funding/Support: This study was funded by Sarepta Therapeutics Inc.Cooperative International Neuromuscular Research Group (CINRG) Investigators: V. Vishwanathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); S. Chidambaranathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); W. Douglas Biggar (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Laura C. McAdam (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Jean K. Mah (Alberta Children's Hospital, Calgary, AB, Canada); Mar Tulinius (Queen Silvia Children's Hospital, G?teborg, Sweden); Avital Cnaan (Children's National Medical Center, Washington DC, USA); Lauren P. Morgenroth (Children's National Medical Center, Washington DC, USA); Robert Leshner (Children's National Medical Center, Washington DC, USA); Carolina TesiRocha (Children's National Medical Center, Washington DC, USA); Mathula Thangarajh (Children's National Medical Center, Washington DC, USA); Tina Duong (Children's National Medical Center, Washington DC, USA); Andrew Kornberg (Royal Children's Hospital, Melbourne, Australia); Monique Ryan (Royal Children's Hospital, Melbourne, Australia); Yoram Nevo (Hadassah Hebrew University Hospital, Jerusalem, Israel); Alberto Dubrovsky (Instituto de Neurosciencias Fundacion Favaloro, Buenos Aires, Argentina); Paula R. Clemens (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Hoda Abdel-Hamid (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Anne M. Connolly (Washington University in St Louis, St Louis, MO, USA); Alan Pestronk (Washington University in St Louis, St Louis, MO, USA); Jean Teasley (Children's Hospital of Virginia, Richmond, VA, USA); Tulio E. Bertorini (University of Tennessee, Memphis, TN, USA); Richard Webster (Children's Hospital at Westmead, Sydney, Australia); Hanna Kolski (University of Alberta, Edmonton, AB, Canada); Nancy Kuntz (Mayo Clinic, Rochester, MN, USA); Sherilyn Driscoll (Mayo Clinic, Rochester, MN, USA); John B. Bodensteiner (Mayo Clinic, Rochester, MN, USA); Jose Carlo (University of Puerto Rico, San Juan, Puerto, Rico); Ksenija Gorni (University of Pavia and Niguarda Ca' Granda Hospital, Milan, Italy); Timothy Lotze (Texas Children's Hospital, Houston, TX, USA); John W. Day (University of Minnesota, Minneapolis, MN, USA); Peter Karachunski (University of Minnesota, Minneapolis, MN, USA); Erik K. Henricson (University of California, Davis, CA, USA); Richard T. Abresch (University of California, Davis, CA, USA); Nanette C. Joyce (University of California, Davis, CA, USA); Craig M. McDonald (University of California, Davis, CA, USA). Duchenne Natural History Study was funded through grants from the US Department of Education/National Institute on Disability and Rehabilitation Research (H133B031118 and H133B090001), US Department of Defense (W81XWH-09-1-0592), the National Institutes of Health (UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875, and RO1AR062380), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study. Funding Information: Funding/Support: This study was funded by Sarepta Therapeutics Inc . Publisher Copyright: {\textcopyright} 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research",

year = "2021",

month = oct,

doi = "10.1016/j.jval.2021.05.016",

language = "English (US)",

volume = "24",

pages = "1490--1498",

journal = "Value in Health",

issn = "1098-3015",

publisher = "Elsevier Limited",

number = "10",

}

TY - JOUR

T1 - Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

AU - Cooperative International Neuromuscular Research Group Investigators

AU - Landfeldt, Erik

AU - Iff, Joel

AU - Henricson, Erik

AU - Vishwanathan, V.

AU - Chidambaranathan, S.

AU - Biggar, W. Douglas

AU - McAdam, Laura C.

AU - Mah, Jean K.

AU - Tulinius, Mar

AU - Cnaan, Avital

AU - Morgenroth, Lauren P.

AU - Leshner, Robert

AU - TesiRocha, Carolina

AU - Thangarajh, Mathula

AU - Duong, Tina

AU - Kornberg, Andrew

AU - Ryan, Monique

AU - Nevo, Yoram

AU - Dubrovsky, Alberto

AU - Clemens, Paula R.

AU - Abdel-Hamid, Hoda

AU - Connolly, Anne M.

AU - Pestronk, Alan

AU - Teasley, Jean

AU - Bertorini, Tulio E.

AU - Webster, Richard

AU - Kolski, Hanna

AU - Kuntz, Nancy

AU - Driscoll, Sherilyn

AU - Bodensteiner, John B.

AU - Carlo, Jose

AU - Gorni, Ksenija

AU - Lotze, Timothy

AU - Day, John W.

AU - Karachunski, Peter I

AU - Henricson, Erik K.

AU - Abresch, Richard T.

AU - Joyce, Nanette C.

AU - McDonald, Craig M.

N1 - Funding Information: Duchenne Natural History Study was funded through grants from the US Department of Education / National Institute on Disability and Rehabilitation Research ( H133B031118 and H133B090001 ), US Department of Defense ( W81XWH-09-1-0592 ), the National Institutes of Health ( UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875 , and RO1AR062380 ), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study. Funding Information: Funding/Support: This study was funded by Sarepta Therapeutics Inc.Cooperative International Neuromuscular Research Group (CINRG) Investigators: V. Vishwanathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); S. Chidambaranathan (Sundaram Medical Foundation and Apollo Children's Hospital, Chennai, India); W. Douglas Biggar (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Laura C. McAdam (Holland Bloorview Kids Rehab Hospital, Toronto, ON, Canada); Jean K. Mah (Alberta Children's Hospital, Calgary, AB, Canada); Mar Tulinius (Queen Silvia Children's Hospital, G?teborg, Sweden); Avital Cnaan (Children's National Medical Center, Washington DC, USA); Lauren P. Morgenroth (Children's National Medical Center, Washington DC, USA); Robert Leshner (Children's National Medical Center, Washington DC, USA); Carolina TesiRocha (Children's National Medical Center, Washington DC, USA); Mathula Thangarajh (Children's National Medical Center, Washington DC, USA); Tina Duong (Children's National Medical Center, Washington DC, USA); Andrew Kornberg (Royal Children's Hospital, Melbourne, Australia); Monique Ryan (Royal Children's Hospital, Melbourne, Australia); Yoram Nevo (Hadassah Hebrew University Hospital, Jerusalem, Israel); Alberto Dubrovsky (Instituto de Neurosciencias Fundacion Favaloro, Buenos Aires, Argentina); Paula R. Clemens (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Hoda Abdel-Hamid (University of Pittsburgh and Department of Veterans Affairs, Pittsburgh, PA, USA); Anne M. Connolly (Washington University in St Louis, St Louis, MO, USA); Alan Pestronk (Washington University in St Louis, St Louis, MO, USA); Jean Teasley (Children's Hospital of Virginia, Richmond, VA, USA); Tulio E. Bertorini (University of Tennessee, Memphis, TN, USA); Richard Webster (Children's Hospital at Westmead, Sydney, Australia); Hanna Kolski (University of Alberta, Edmonton, AB, Canada); Nancy Kuntz (Mayo Clinic, Rochester, MN, USA); Sherilyn Driscoll (Mayo Clinic, Rochester, MN, USA); John B. Bodensteiner (Mayo Clinic, Rochester, MN, USA); Jose Carlo (University of Puerto Rico, San Juan, Puerto, Rico); Ksenija Gorni (University of Pavia and Niguarda Ca' Granda Hospital, Milan, Italy); Timothy Lotze (Texas Children's Hospital, Houston, TX, USA); John W. Day (University of Minnesota, Minneapolis, MN, USA); Peter Karachunski (University of Minnesota, Minneapolis, MN, USA); Erik K. Henricson (University of California, Davis, CA, USA); Richard T. Abresch (University of California, Davis, CA, USA); Nanette C. Joyce (University of California, Davis, CA, USA); Craig M. McDonald (University of California, Davis, CA, USA). Duchenne Natural History Study was funded through grants from the US Department of Education/National Institute on Disability and Rehabilitation Research (H133B031118 and H133B090001), US Department of Defense (W81XWH-09-1-0592), the National Institutes of Health (UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875, and RO1AR062380), and Parent Project Muscular Dystrophy. The authors thank the participating patients and their families for taken part of this research and gratefully acknowledge the participation of all investigators, clinical coordinators, clinical evaluator trainers, clinical evaluators, coordinators, and supporting staff for their contributions to the study. Funding Information: Funding/Support: This study was funded by Sarepta Therapeutics Inc . Publisher Copyright: © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research

PY - 2021/10

Y1 - 2021/10

N2 - Objectives: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately fatal neuromuscular disease. Methods: Patients with DMD were recruited from 20 centers across 9 countries as part of the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (NCT00468832). The psychometric properties of the PedsQL 4.0 GCS were examined using Rasch analysis. Results: In total, 329 patients with DMD (mean age 9 years, range 3–18 years, 75% ambulatory) completed the PedsQL 4.0 GCS. The most difficult instrument items, expressing the greatest loss in health-related quality of life, were those associated with emotional well-being (eg, being teased by other children, feeling sad, and not making friends), as opposed to somatic disability (eg, lifting heavy objects, participating in sports, and running). The mean item and person fit residuals were estimated at 0.301 (SD: 1.385) and −0.255 (1.504), respectively. In total, 87% (20 of 23) of items displayed disordered thresholds, and many exhibited nontrivial dependency. The overall item-trait interaction χ2 value was 178 (115 degrees of freedom, P<.001). Our analysis also revealed significant issues with differential item functioning, and by investigating residual principal component loadings, the PedsQL 4.0 GCS total score was found to be multidimensional. Conclusions: The PedsQL 4.0 GCS records information clinically relevant to patients with DMD, but the total scale score may not be fit for purpose as a measure health-related quality of life in this disease population.

AB - Objectives: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately fatal neuromuscular disease. Methods: Patients with DMD were recruited from 20 centers across 9 countries as part of the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (NCT00468832). The psychometric properties of the PedsQL 4.0 GCS were examined using Rasch analysis. Results: In total, 329 patients with DMD (mean age 9 years, range 3–18 years, 75% ambulatory) completed the PedsQL 4.0 GCS. The most difficult instrument items, expressing the greatest loss in health-related quality of life, were those associated with emotional well-being (eg, being teased by other children, feeling sad, and not making friends), as opposed to somatic disability (eg, lifting heavy objects, participating in sports, and running). The mean item and person fit residuals were estimated at 0.301 (SD: 1.385) and −0.255 (1.504), respectively. In total, 87% (20 of 23) of items displayed disordered thresholds, and many exhibited nontrivial dependency. The overall item-trait interaction χ2 value was 178 (115 degrees of freedom, P<.001). Our analysis also revealed significant issues with differential item functioning, and by investigating residual principal component loadings, the PedsQL 4.0 GCS total score was found to be multidimensional. Conclusions: The PedsQL 4.0 GCS records information clinically relevant to patients with DMD, but the total scale score may not be fit for purpose as a measure health-related quality of life in this disease population.

KW - Cooperative International Neuromuscular Research Group

KW - disability

KW - patient-reported outcome

KW - psychometric analysis

KW - quality of life

UR - http://www.scopus.com/inward/record.url?scp=85115743599&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85115743599&partnerID=8YFLogxK

U2 - 10.1016/j.jval.2021.05.016

DO - 10.1016/j.jval.2021.05.016

M3 - Article

C2 - 34593173

AN - SCOPUS:85115743599

SN - 1098-3015

VL - 24

SP - 1490

EP - 1498

JO - Value in Health

JF - Value in Health

IS - 10

ER -

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

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