{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T20:58:39Z","timestamp":1790801919292,"version":"4.1.0"},"reference-count":41,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2022,9,4]],"date-time":"2022-09-04T00:00:00Z","timestamp":1662249600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J cachexia sarcopenia muscle"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Water T2 (T2<jats:sub>H2O<\/jats:sub>) mapping is increasingly being used in muscular dystrophies to assess active muscle damage. It has been suggested as a surrogate outcome measure for clinical trials. Here, we investigated the prognostic utility of T2<jats:sub>H2O<\/jats:sub> to identify changes in muscle function over time in limb girdle muscular dystrophies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients with genetically confirmed dysferlinopathy were assessed as part of the Jain Foundation Clinical Outcomes Study in dysferlinopathy. The cohort included 18 patients from two sites, both equipped with 3\u2010tesla magnetic resonance imaging (MRI) systems from the same vendor. T2<jats:sub>H2O<\/jats:sub> value was defined as higher or lower than the median in each muscle bilaterally. The degree of deterioration on four functional tests over 3\u00a0years was assessed in a linear model against covariates of high or low T2<jats:sub>H2O<\/jats:sub> at baseline, age, disease duration, and baseline function.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A higher T2<jats:sub>H2O<\/jats:sub> at baseline significantly correlated with a greater decline on functional tests in 21 out of 35 muscles and was never associated with slower decline. Higher baseline T2<jats:sub>H2O<\/jats:sub> in adductor magnus, vastus intermedius, vastus lateralis, and vastus medialis were the most sensitive, being associated bilaterally with greater decline in multiple timed tests. Patients with a higher than median baseline T2<jats:sub>H2O<\/jats:sub> (&gt;40.6\u00a0ms) in the right vastus medialis deteriorated 11 points more on the North Star Ambulatory Assessment for Dysferlinopathy and lost an additional 86\u00a0m on the 6\u2010min walk than those with a lower T2<jats:sub>H2O<\/jats:sub> (&lt;40.6\u00a0ms). Optimum sensitivity and specificity thresholds for predicting decline were 39.0\u00a0ms in adductor magnus and vastus intermedius, 40.0\u00a0ms in vastus medialis, and 40.5\u00a0ms in vastus lateralis from different sites equipped with different MRI systems.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In dysferlinopathy, T2<jats:sub>H2O<\/jats:sub> did not correlate with current functional ability. However, T2<jats:sub>H2O<\/jats:sub> at baseline was higher in patients who worsened more rapidly on functional tests. This suggests that inter\u2010patient differences in functional decline over time may be, in part, explained by different severities of the active muscle damage, assessed by T2<jats:sub>H2O<\/jats:sub> measure at baseline. Significant challenges remain in standardizing T2<jats:sub>H2O<\/jats:sub> values across sites to allow determining globally applicable thresholds. The results from the present work are encouraging and suggest that T2<jats:sub>H2O<\/jats:sub> could be used to improve prognostication, patient selection, and disease modelling for clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jcsm.13063","type":"journal-article","created":{"date-parts":[[2022,9,4]],"date-time":"2022-09-04T22:13:25Z","timestamp":1662329605000},"page":"2888-2897","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Water T2 could predict functional decline in patients with dysferlinopathy"],"prefix":"10.1002","volume":"13","author":[{"given":"Ursula","family":"Moore","sequence":"first","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute Newcastle University and Newcastle Hospitals NHS Foundation Trust  Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ericky","family":"Caldas de Almeida Ara\u00fajo","sequence":"additional","affiliation":[{"name":"NMR Laboratory, Neuromuscular Investigation Center Institute of Myology  Paris France"},{"name":"NMR Laboratory CEA\/DRF\/IBFJ\/MIRCen  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harmen","family":"Reyngoudt","sequence":"additional","affiliation":[{"name":"NMR Laboratory, Neuromuscular Investigation Center Institute of Myology  Paris France"},{"name":"NMR Laboratory CEA\/DRF\/IBFJ\/MIRCen  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gordish\u2010Dressman","sequence":"additional","affiliation":[{"name":"Center for Translational Science, Division of Biostatistics and Study Methodology Children's National Health System  Washington DC USA"},{"name":"Pediatrics, Epidemiology and Biostatistics George Washington University  Washington DC USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona E.","family":"Smith","sequence":"additional","affiliation":[{"name":"Magnetic Resonance Centre, Translational and Clinical Research Institute Newcastle University  Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian","family":"Wilson","sequence":"additional","affiliation":[{"name":"Magnetic Resonance Centre, Translational and Clinical Research Institute Newcastle University  Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith","family":"James","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute Newcastle University and Newcastle Hospitals NHS Foundation Trust  Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute Newcastle University and Newcastle Hospitals NHS Foundation Trust  Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Rufibach","sequence":"additional","affiliation":[{"name":"Jain Foundation  Seattle WA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John W.","family":"Day","sequence":"additional","affiliation":[{"name":"Department of Neurology and Neurological Sciences Stanford University School of Medicine  Stanford CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristi J.","family":"Jones","sequence":"additional","affiliation":[{"name":"The Children's Hospital at Westmead and The University of Sydney  Sydney NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana X.","family":"Bharucha\u2010Goebel","sequence":"additional","affiliation":[{"name":"Department of Neurology Children's National Health System  Washington DC USA"},{"name":"National Institutes of Health (NINDS)  Bethesda MD USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuelle","family":"Salort\u2010Campana","sequence":"additional","affiliation":[{"name":"Service des maladies neuromusculaire et de la SLA H\u00f4pital de La Timone  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan","family":"Pestronk","sequence":"additional","affiliation":[{"name":"Department of Neurology Washington University School of Medicine  St. Louis MO USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maggie C.","family":"Walter","sequence":"additional","affiliation":[{"name":"Friedrich\u2010Baur\u2010Institute, Department of Neurology Ludwig\u2010Maximilians\u2010University of Munich  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen","family":"Paradas","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Department of Neurology Hospital U. 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Muscular Dystrophy Research Centre, Translational and Clinical Research Institute Newcastle University and Newcastle Hospitals NHS Foundation Trust  Newcastle upon Tyne UK"},{"name":"Neuromuscular Disorders Unit, Neurology Department Hospital de la Santa Creu i Sant Pau  Barcelona Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red en Enfermedades Raras (CIBERER)  Madrid 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of Cachexia, Sarcopenia and 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