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Assessing the relationship of patient reported outcome measures with functional status in dysferlinopathy: a Rasch analysis approach

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Item Type:Article
Title:Assessing the relationship of patient reported outcome measures with functional status in dysferlinopathy: a Rasch analysis approach
Creators Name:Mayhew, A.G. and James, M.K. and Moore, U. and Sutherland, H. and Jacobs, M. and Feng, J. and Lowes, L.P. and Alfano, L.N. and Muni Lofra, R. and Rufibach, L.E. and Rose, K. and Duong, T. and Bello, L. and Pedrosa-Hernández, I. and Holsten, S. and Sakamoto, C. and Canal, A. and Sánchez-Aguilera Práxedes, N. and Thiele, S. and Siener, C. and Vandevelde, B. and DeWolf, B. and Maron, E. and Gordish-Dressman, H. and Hilsden, H. and Guglieri, M. and Hogrel, J.Y. and Blamire, A.M. and Carlier, P.G. and Spuler, S. and Day, J.W. and Jones, K.J. and Bharucha-Goebel, D.X. and Salort-Campana, E. and Pestronk, A. and Walter, M.C. and Paradas, C. and Stojkovic, T. and Mori-Yoshimura, M. and Bravver, E. and Díaz-Manera, J. and Pegoraro, E. and Mendell, J.R. and Straub, V.
Abstract:Dysferlinopathy is a muscular dystrophy with a highly variable functional disease progression in which the relationship of function to some patient reported outcome measures (PROMs) has not been previously reported. This analysis aims to identify the suitability of PROMs and their association with motor performance.Two-hundred and four patients with dysferlinopathy were identified in the Jain Foundation's Clinical Outcome Study in Dysferlinopathy from 14 sites in 8 countries. All patients completed the following PROMs: Individualized Neuromuscular Quality of Life Questionnaire (INQoL), International Physical Activity Questionnaire (IPAQ), and activity limitations for patients with upper and/or lower limb impairments (ACTIVLIMs). In addition, nonambulant patients completed the Egen Klassifikation Scale (EK). Assessments were conducted annually at baseline, years 1, 2, 3, and 4. Data were also collected on the North Star Assessment for Limb Girdle Type Muscular Dystrophies (NSAD) and Performance of Upper Limb (PUL) at these time points from year 2. Data were analyzed using descriptive statistics and Rasch analysis was conducted on ACTIVLIM, EK, INQoL. For associations, graphs (NSAD with ACTIVLIM, IPAQ and INQoL and EK with PUL) were generated from generalized estimating equations (GEE). The ACTIVLIM appeared robust psychometrically and was strongly associated with the NSAD total score (Pseudo R(2) 0.68). The INQoL performed less well and was poorly associated with the NSAD total score (Pseudo R(2) 0.18). EK scores were strongly associated with PUL (Pseudo R(2) 0.69). IPAQ was poorly associated with NSAD scores (Pseudo R(2) 0.09). This study showed that several of the chosen PROMs demonstrated change over time and a good association with functional outcomes. An alternative quality of life measure and method of collecting data on physical activity may need to be selected for assessing dysferlinopathy.
Keywords:Limb Girdle Muscular Dystrophy, Dysferlinopathy, PROMs, Quality of Life, Clinical Outcome Assessments
Source:Frontiers in Neurology
ISSN:1664-2295
Publisher:Frontiers Media SA
Volume:13
Page Range:828525
Date:10 March 2022
Official Publication:https://doi.org/10.3389/fneur.2022.828525
PubMed:View item in PubMed

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