Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.
Pompe disease
Rasch analysis
daily life activities
patient‐reported outcome measure
Journal
European journal of neurology
ISSN: 1468-1331
Titre abrégé: Eur J Neurol
Pays: England
ID NLM: 9506311
Informations de publication
Date de publication:
28 Aug 2024
28 Aug 2024
Historique:
revised:
29
05
2024
received:
24
12
2023
accepted:
19
06
2024
medline:
31
8
2024
pubmed:
31
8
2024
entrez:
29
8
2024
Statut:
aheadofprint
Résumé
The Rasch-Built Pompe-Specific Activity (R-PAct) scale is a patient-reported outcome measure specifically designed to quantify the effects of Pompe disease on daily life activities, developed for use in Dutch- and English-speaking countries. This study aimed to validate the R-PAct for use in other countries. Four other language versions (German, French, Italian, and Spanish) of the R-PAct were created and distributed among Pompe patients (≥16 years old) in Germany, France, Spain, Italy, and Switzerland and pooled with data of newly diagnosed patients from Australia, Belgium, Canada, the Netherlands, New Zealand, the USA, and the UK and the original validation cohort (n = 186). The psychometric properties of the scale were assessed by exploratory factor analysis and Rasch analysis. Data for 520 patients were eligible for analysis. Exploratory factor analysis suggested that the items separated into two domains: Activities of Daily Living and Mobility. Both domains independently displayed adequate Rasch model measurement properties, following the removal of one item ("Are you able to practice a sport?") from the Mobility domain, and can be added together to form a "higher order" factor as well. Differential item functioning (DIF)-by-language assessment indicated DIF for several items; however, the impact of accounting for DIF was negligible. We recalibrated the nomogram (raw score interval-level transformation) for the updated 17-item R-PAct scale. The minimal detectable change value was 13.85 for the overall R-PAct. After removing one item, the modified-R-PAct scale is a valid disease-specific patient-reported outcome measure for patients with Pompe disease across multiple countries.
Sections du résumé
BACKGROUND AND PURPOSE
OBJECTIVE
The Rasch-Built Pompe-Specific Activity (R-PAct) scale is a patient-reported outcome measure specifically designed to quantify the effects of Pompe disease on daily life activities, developed for use in Dutch- and English-speaking countries. This study aimed to validate the R-PAct for use in other countries.
METHODS
METHODS
Four other language versions (German, French, Italian, and Spanish) of the R-PAct were created and distributed among Pompe patients (≥16 years old) in Germany, France, Spain, Italy, and Switzerland and pooled with data of newly diagnosed patients from Australia, Belgium, Canada, the Netherlands, New Zealand, the USA, and the UK and the original validation cohort (n = 186). The psychometric properties of the scale were assessed by exploratory factor analysis and Rasch analysis.
RESULTS
RESULTS
Data for 520 patients were eligible for analysis. Exploratory factor analysis suggested that the items separated into two domains: Activities of Daily Living and Mobility. Both domains independently displayed adequate Rasch model measurement properties, following the removal of one item ("Are you able to practice a sport?") from the Mobility domain, and can be added together to form a "higher order" factor as well. Differential item functioning (DIF)-by-language assessment indicated DIF for several items; however, the impact of accounting for DIF was negligible. We recalibrated the nomogram (raw score interval-level transformation) for the updated 17-item R-PAct scale. The minimal detectable change value was 13.85 for the overall R-PAct.
CONCLUSIONS
CONCLUSIONS
After removing one item, the modified-R-PAct scale is a valid disease-specific patient-reported outcome measure for patients with Pompe disease across multiple countries.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e16397Investigateurs
H A van Kooten
(HA)
E Brusse
(E)
P A van Doorn
(PA)
A T van der Ploeg
(AT)
N A M E van der Beek
(NAME)
S Wenninger
(S)
H Babačić
(H)
B Schoser
(B)
F Montagnese
(F)
N Gracia Angarita
(N)
C Lefeuvre
(C)
N Taouagh
(N)
P Laforêt
(P)
A Béhin
(A)
C Tard
(C)
E Campana-Salort
(E)
S Sacconi
(S)
G Solé
(G)
M Spinazzi
(M)
F Bouhour
(F)
F Bouibede
(F)
D Hamroun
(D)
J Y Hogrel
(JY)
S Segovia
(S)
J Díaz-Manera
(J)
K G Claeys
(KG)
T Mongini
(T)
O Musumeci
(O)
A Toscano
(A)
T Hundsberger
(T)
M C Horton
(MC)
Informations de copyright
© 2024 The Author(s). European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.
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