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
AU - Henricson, Erik K.
AU - Abresch, Richard T.
AU - Joyce, Nanette C.
AU - McDonald, Craig M.
N1 - 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
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 -