RT Journal Article
T1 Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.
A1 Mayhew, Anna G
A1 James, Meredith K
A1 Moore, Ursula
A1 Sutherland, Helen
A1 Jacobs, Marni
A1 Feng, Jia
A1 Lowes, Linda Pax
A1 Alfano, Lindsay N
A1 Muni Lofra, Robert
A1 Rufibach, Laura E
A1 Rose, Kristy
A1 Duong, Tina
A1 Bello, Luca
A1 Pedrosa-Hernández, Irene
A1 Holsten, Scott
A1 Sakamoto, Chikako
A1 Canal, Aurélie
A1 Sánchez-Aguilera Práxedes, Nieves
A1 Thiele, Simone
A1 Siener, Catherine
A1 Vandevelde, Bruno
A1 DeWolf, Brittney
A1 Maron, Elke
A1 Gordish-Dressman, Heather
A1 Hilsden, Heather
A1 Guglieri, Michela
A1 Hogrel, Jean-Yves
A1 Blamire, Andrew M
A1 Carlier, Pierre G
A1 Spuler, Simone
A1 Day, John W
A1 Jones, Kristi J
A1 Bharucha-Goebel, Diana X
A1 Salort-Campana, Emmanuelle
A1 Pestronk, Alan
A1 Walter, Maggie C
A1 Paradas, Carmen
A1 Stojkovic, Tanya
A1 Mori-Yoshimura, Madoka
A1 Bravver, Elena
A1 Díaz-Manera, Jordi
A1 Pegoraro, Elena
A1 Mendell, Jerry R
A1 Straub, Volker
K1 PROMs
K1 clinical outcome assessments
K1 dysferlinopathy
K1 limb girdle muscular dystrophy
K1 quality of life
AB 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 R2 0.68). The INQoL performed less well and was poorly associated with the NSAD total score (Pseudo R2 0.18). EK scores were strongly associated with PUL (Pseudo R2 0.69). IPAQ was poorly associated with NSAD scores (Pseudo R2 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.
SN 1664-2295
YR 2022
FD 2022-03-10
LK http://hdl.handle.net/10668/20643
UL http://hdl.handle.net/10668/20643
LA en
DS RISalud
RD Dec 22, 2024