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

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; Miguel Angel Barba-Romero; Joseba Barcena; Pilar Carbonell; María Rosario Carzorla; Carlota Creus; Jaume Coll-Cantí; Manuel Díaz; Cristina Domínguez; Roberto Fernández-Torrón; María José García-Antelo; Josep Maria Grau; Adolfo López De Munáin; Francisco Antonio Martínez-García; Yolanda Morgado; Antonio Moreno; Germán Morís; Miguel Angel Muñoz-Blanco; Andres Nascimento; José Luis Parajua; Arturo Robledo-Strauss; Íñigo Rojas-Marcos; Jose António Salazar; Mercedes Usón; Jordi Díaz-Manera

doi:10.1038/s41598-018-29170-7

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

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, Miguel Angel Barba-Romero, Joseba Barcena, Pilar Carbonell, María Rosario Carzorla, Carlota Creus, Jaume Coll-Cantí, Manuel Díaz, Cristina Domínguez, Roberto Fernández-TorrónMaría José García-Antelo, Josep Maria Grau, Adolfo López De Munáin, Francisco Antonio Martínez-García, Yolanda Morgado, Antonio Moreno, Germán Morís, Miguel Angel Muñoz-Blanco, Andres Nascimento, José Luis Parajua, Arturo Robledo-Strauss, Íñigo Rojas-Marcos, Jose António Salazar, Mercedes Usón, Jordi Díaz-Manera

Department of Medicine

Research output: Contribution to journal › Article › Research

24 Citations (Scopus)

Abstract

© 2018 The Author(s). 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.

Original language	English
Article number	10898
Journal	Scientific Reports
Volume	8
DOIs	https://doi.org/10.1038/s41598-018-29170-7
Publication status	Published - 1 Dec 2018

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Figueroa-Bonaparte, S., Llauger, J., Segovia, S., Belmonte, I., Pedrosa, I., Montiel, E., Montesinos, P., Sánchez-González, J., Alonso-Jiménez, A., Gallardo, E., Illa, I., Barba-Romero, M. A., Barcena, J., Carbonell, P., Carzorla, M. R., Creus, C., Coll-Cantí, J., Díaz, M., Domínguez, C., ... Díaz-Manera, J. (2018). Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study. Scientific Reports, 8, [10898]. https://doi.org/10.1038/s41598-018-29170-7

Figueroa-Bonaparte, Sebastian ; Llauger, Jaume ; Segovia, Sonia ; Belmonte, Izaskun ; Pedrosa, Irene ; Montiel, Elena ; Montesinos, Paula ; Sánchez-González, Javier ; Alonso-Jiménez, Alicia ; Gallardo, Eduard ; Illa, Isabel ; Barba-Romero, Miguel Angel ; Barcena, Joseba ; Carbonell, Pilar ; Carzorla, María Rosario ; Creus, Carlota ; Coll-Cantí, Jaume ; Díaz, Manuel ; Domínguez, Cristina ; Fernández-Torrón, Roberto ; García-Antelo, María José ; Grau, Josep Maria ; De Munáin, Adolfo López ; Martínez-García, Francisco Antonio ; Morgado, Yolanda ; Moreno, Antonio ; Morís, Germán ; Muñoz-Blanco, Miguel Angel ; Nascimento, Andres ; Parajua, José Luis ; Robledo-Strauss, Arturo ; Rojas-Marcos, Íñigo ; Salazar, Jose António ; Usón, Mercedes ; Díaz-Manera, Jordi. / Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study. In: Scientific Reports. 2018 ; Vol. 8.

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title = "Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study",

abstract = "{\textcopyright} 2018 The Author(s). 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.",

author = "Sebastian Figueroa-Bonaparte and Jaume Llauger and Sonia Segovia and Izaskun Belmonte and Irene Pedrosa and Elena Montiel and Paula Montesinos and Javier S{\'a}nchez-Gonz{\'a}lez and Alicia Alonso-Jim{\'e}nez and Eduard Gallardo and Isabel Illa and Barba-Romero, {Miguel Angel} and Joseba Barcena and Pilar Carbonell and Carzorla, {Mar{\'i}a Rosario} and Carlota Creus and Jaume Coll-Cant{\'i} and Manuel D{\'i}az and Cristina Dom{\'i}nguez and Roberto Fern{\'a}ndez-Torr{\'o}n and Garc{\'i}a-Antelo, {Mar{\'i}a Jos{\'e}} and Grau, {Josep Maria} and {De Mun{\'a}in}, {Adolfo L{\'o}pez} and Mart{\'i}nez-Garc{\'i}a, {Francisco Antonio} and Yolanda Morgado and Antonio Moreno and Germ{\'a}n Mor{\'i}s and Mu{\~n}oz-Blanco, {Miguel Angel} and Andres Nascimento and Parajua, {Jos{\'e} Luis} and Arturo Robledo-Strauss and {\'I}{\~n}igo Rojas-Marcos and Salazar, {Jose Ant{\'o}nio} and Mercedes Us{\'o}n and Jordi D{\'i}az-Manera",

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doi = "10.1038/s41598-018-29170-7",

language = "English",

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Figueroa-Bonaparte, S, Llauger, J, Segovia, S, Belmonte, I, Pedrosa, I, Montiel, E, Montesinos, P, Sánchez-González, J, Alonso-Jiménez, A, Gallardo, E, Illa, I, Barba-Romero, MA, Barcena, J, Carbonell, P, Carzorla, MR, Creus, C, Coll-Cantí, J, Díaz, M, Domínguez, C, Fernández-Torrón, R, García-Antelo, MJ, Grau, JM, De Munáin, AL, Martínez-García, FA, Morgado, Y, Moreno, A, Morís, G, Muñoz-Blanco, MA, Nascimento, A, Parajua, JL, Robledo-Strauss, A, Rojas-Marcos, Í, Salazar, JA, Usón, M & Díaz-Manera, J 2018, 'Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study', Scientific Reports, vol. 8, 10898. https://doi.org/10.1038/s41598-018-29170-7

Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study. / Figueroa-Bonaparte, Sebastian; Llauger, Jaume; Segovia, Sonia; Belmonte, Izaskun; Pedrosa, Irene; Montiel, Elena; Montesinos, Paula; Sánchez-González, Javier; Alonso-Jiménez, Alicia; Gallardo, Eduard; Illa, Isabel; Barba-Romero, Miguel Angel; Barcena, Joseba; Carbonell, Pilar; Carzorla, María Rosario; Creus, Carlota; Coll-Cantí, Jaume; Díaz, Manuel; Domínguez, Cristina; Fernández-Torrón, Roberto; García-Antelo, María José; Grau, Josep Maria; De Munáin, Adolfo López; Martínez-García, Francisco Antonio; Morgado, Yolanda; Moreno, Antonio; Morís, Germán; Muñoz-Blanco, Miguel Angel; Nascimento, Andres; Parajua, José Luis; Robledo-Strauss, Arturo; Rojas-Marcos, Íñigo; Salazar, Jose António; Usón, Mercedes; Díaz-Manera, Jordi.

In: Scientific Reports, Vol. 8, 10898, 01.12.2018.

Research output: Contribution to journal › Article › Research

TY - JOUR

T1 - Quantitative muscle MRI to follow up late onset Pompe patients: A prospective study

AU - Figueroa-Bonaparte, Sebastian

AU - Llauger, Jaume

AU - Segovia, Sonia

AU - Belmonte, Izaskun

AU - Pedrosa, Irene

AU - Montiel, Elena

AU - Montesinos, Paula

AU - Sánchez-González, Javier

AU - Alonso-Jiménez, Alicia

AU - Gallardo, Eduard

AU - Illa, Isabel

AU - Barba-Romero, Miguel Angel

AU - Barcena, Joseba

AU - Carbonell, Pilar

AU - Carzorla, María Rosario

AU - Creus, Carlota

AU - Coll-Cantí, Jaume

AU - Díaz, Manuel

AU - Domínguez, Cristina

AU - Fernández-Torrón, Roberto

AU - García-Antelo, María José

AU - Grau, Josep Maria

AU - De Munáin, Adolfo López

AU - Martínez-García, Francisco Antonio

AU - Morgado, Yolanda

AU - Moreno, Antonio

AU - Morís, Germán

AU - Muñoz-Blanco, Miguel Angel

AU - Nascimento, Andres

AU - Parajua, José Luis

AU - Robledo-Strauss, Arturo

AU - Rojas-Marcos, Íñigo

AU - Salazar, Jose António

AU - Usón, Mercedes

AU - Díaz-Manera, Jordi

PY - 2018/12/1

Y1 - 2018/12/1

N2 - © 2018 The Author(s). 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.

AB - © 2018 The Author(s). 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.

U2 - 10.1038/s41598-018-29170-7

DO - 10.1038/s41598-018-29170-7

M3 - Article

C2 - 30022036

VL - 8

M1 - 10898

ER -