Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy : A Rasch Analysis Approach
Mayhew, Anna G. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
James, Meredith K. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Moore, Ursula (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Sutherland, Helen (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Jacobs, Marni (Pediatrics. Epidemiology and Biostatistics. George Washington University)
Feng, Jia (Center for Translational Science. Division of Biostatistics and Study Methodology. Children's National Health System)
Lowes, Linda Pax (The Abigail Wexner Research Institute at Nationwide Children's Hospital)
Alfano, Lindsay N. (The Abigail Wexner Research Institute at Nationwide Children's Hospital)
Muni Lofra, Robert (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Rufibach, Laura E. (The Jain Foundation)
Rose, Kristy (The Children's Hospital at Westmead. The University of Sydney)
Duong, Tina (Lucile Salter Packard Children's Hospital at Stanford. Neurology)
Bello, Luca (Department of Neuroscience. University of Padova)
Pedrosa-Hernández, Irene (Hospital de la Santa Creu i Sant Pau (Barcelona, Catalunya))
Holsten, S. (Neuroscience Institute. Carolinas Neuromuscular/ALS-MDA Center. Carolinas HealthCare System)
Sakamoto, C. (Department of Physical Rehabilitation. National Center Hospital. National Center of Neurology and Psychiatry Tokyo)
Canal, Aurélie (Institut de Myologie. AP-HP. GH Pitié-Salpêtrière)
Sánchez-Aguilera Práxedes, N. (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Thiele, S. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians-University of Munich)
Siener, C. (Department of Neurology. Washington University School of Medicine)
Vandevelde, Bruno (Service des Maladies Neuromusculaire et de la SLA. Hôpital de La Timone)
DeWolf, Brittney (Cooperative International Neuromuscular Research Group. Children's National Health System)
Maron, E. (ELAN-PHYSIO. Praxis für Physiotherapie Maron)
Gordish-Dressman, Heather (George Washington University)
Hilsden, H. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Guglieri, M. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)
Hogrel, J.Y. (Institut de Myologie. AP-HP. GH Pitié-Salpêtrière)
Blamire, Andrew (Magnetic Resonance Centre. Institute for Cellular Medicine. Newcastle University)
Carlier, Pierre G. (Pitié-Salpêtrière University Hospital)
Spuler, S. (Charite Muscle Research Unit. Experimental and Clinical Research Center. A Joint Cooperation of the Charité Medical Faculty and the Max Delbrück Center for Molecular Medicine)
Day, John W. (Stanford University School of Medicine)
Jones, K.J. (The Children's Hospital at Westmead. The University of Sydney)
Bharucha-Goebel, Diana (National Institutes of Health (Bethesda, Estats Units d'Amèrica))
Salort-Campana, E. (Service des Maladies Neuromusculaire et de la SLA. Hôpital de La Timone)
Pestronk, Alan (Washington University School of Medicine)
Walter, M.C. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians-University of Munich)
Paradas, C. (Instituto de Biomedicina de Sevilla)
Stojkovic, T. (Institut de Myologie. AP-HP. GH Pitié-Salpêtrière)
Mori-Yoshimura, Madoka (National Center of Neurology and Psychiatry Tokyo)
Bravver, Elena (Neuroscience Institute. Carolinas Neuromuscular/ALS-MDA Center. Carolinas HealthCare System)
Diaz-Manera, Jordi (Institut d'Investigació Biomèdica Sant Pau)
Pegoraro, Elena (University of Padova)
Mendell, J. R. (The Abigail Wexner Research Institute at Nationwide Children's Hospital)
Jain COS Consortium, None (The Abigail Wexner Research Institute at Nationwide Children's Hospital)
Straub, V. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust)

Data:	2022
Resum:	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 0. 68). The INQoL performed less well and was poorly associated with the NSAD total score (Pseudo R 0. 18). EK scores were strongly associated with PUL (Pseudo R 0. 69). IPAQ was poorly associated with NSAD scores (Pseudo R 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.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Limb girdle muscular dystrophy ; Dysferlinopathy ; PROMs ; Quality of life ; Clinical outcome assessments
Publicat a:	Frontiers in neurology, Vol. 13 (october 2022) , p. 828525, ISSN 1664-2295

DOI: 10.3389/fneur.2022.828525
PMID: 35359643

10 p, 1.7 MB

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