Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study

Abstract Late onset Pompe disease (LOPD) is a slow, progressive disorder characterized by skeletal and respiratory muscle weakness. Enzyme replacement therapy (ERT) slows down the progression of muscle symptoms. Reliable biomarkers are needed to follow up ERT-treated and asymptomatic LOPD patients i...

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Autores principales:	Sebastian Figueroa-Bonaparte, Jaume Llauger, Sonia Segovia, Izaskun Belmonte, Irene Pedrosa, Elena Montiel, Paula Montesinos, Javier Sánchez-González, Alicia Alonso-Jiménez, Eduard Gallardo, Isabel Illa, Spanish Pompe group, Jordi Díaz-Manera
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2018
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/92acffe4ea3d495381a7f63dd8c21825
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spelling	oai:doaj.org-article:92acffe4ea3d495381a7f63dd8c218252021-12-02T15:08:37ZQuantitative muscle MRI to follow up late onset Pompe patients: a prospective study10.1038/s41598-018-29170-72045-2322https://doaj.org/article/92acffe4ea3d495381a7f63dd8c218252018-07-01T00:00:00Zhttps://doi.org/10.1038/s41598-018-29170-7https://doaj.org/toc/2045-2322Abstract Late onset Pompe disease (LOPD) is a slow, progressive disorder characterized by skeletal and respiratory muscle weakness. Enzyme replacement therapy (ERT) slows down the progression of muscle symptoms. Reliable biomarkers are needed to follow up ERT-treated and asymptomatic LOPD patients in clinical practice. In this study, 32 LOPD patients (22 symptomatic and 10 asymptomatic) underwent muscle MRI using 3-point Dixon and were evaluated at the time of the MRI with several motor function tests and patient-reported outcome measures, and again after one year. Muscle MRI showed a significant increase of 1.7% in the fat content of the thigh muscles in symptomatic LOPD patients. In contrast, there were no noteworthy differences between muscle function tests in the same period of time. We did not observe any significant changes either in muscle MRI or in muscle function tests in asymptomatic patients over the year. We conclude that 3-point Dixon muscle MRI is a useful tool for detecting changes in muscle structure in symptomatic LOPD patients and could become part of the current follow-up protocol in daily clinics.Sebastian Figueroa-BonaparteJaume LlaugerSonia SegoviaIzaskun BelmonteIrene PedrosaElena MontielPaula MontesinosJavier Sánchez-GonzálezAlicia Alonso-JiménezEduard GallardoIsabel IllaSpanish Pompe groupJordi Díaz-ManeraNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 8, Iss 1, Pp 1-11 (2018)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Sebastian Figueroa-Bonaparte Jaume Llauger Sonia Segovia Izaskun Belmonte Irene Pedrosa Elena Montiel Paula Montesinos Javier Sánchez-González Alicia Alonso-Jiménez Eduard Gallardo Isabel Illa Spanish Pompe group Jordi Díaz-Manera Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
description	Abstract Late onset Pompe disease (LOPD) is a slow, progressive disorder characterized by skeletal and respiratory muscle weakness. Enzyme replacement therapy (ERT) slows down the progression of muscle symptoms. Reliable biomarkers are needed to follow up ERT-treated and asymptomatic LOPD patients in clinical practice. In this study, 32 LOPD patients (22 symptomatic and 10 asymptomatic) underwent muscle MRI using 3-point Dixon and were evaluated at the time of the MRI with several motor function tests and patient-reported outcome measures, and again after one year. Muscle MRI showed a significant increase of 1.7% in the fat content of the thigh muscles in symptomatic LOPD patients. In contrast, there were no noteworthy differences between muscle function tests in the same period of time. We did not observe any significant changes either in muscle MRI or in muscle function tests in asymptomatic patients over the year. We conclude that 3-point Dixon muscle MRI is a useful tool for detecting changes in muscle structure in symptomatic LOPD patients and could become part of the current follow-up protocol in daily clinics.
format	article
author	Sebastian Figueroa-Bonaparte Jaume Llauger Sonia Segovia Izaskun Belmonte Irene Pedrosa Elena Montiel Paula Montesinos Javier Sánchez-González Alicia Alonso-Jiménez Eduard Gallardo Isabel Illa Spanish Pompe group Jordi Díaz-Manera
author_facet	Sebastian Figueroa-Bonaparte Jaume Llauger Sonia Segovia Izaskun Belmonte Irene Pedrosa Elena Montiel Paula Montesinos Javier Sánchez-González Alicia Alonso-Jiménez Eduard Gallardo Isabel Illa Spanish Pompe group Jordi Díaz-Manera
author_sort	Sebastian Figueroa-Bonaparte
title	Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
title_short	Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
title_full	Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
title_fullStr	Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
title_full_unstemmed	Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study
title_sort	quantitative muscle mri to follow up late onset pompe patients: a prospective study
publisher	Nature Portfolio
publishDate	2018
url	https://doaj.org/article/92acffe4ea3d495381a7f63dd8c21825
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Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study

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