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Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study.
Sci Rep 2018; 8(1):10898SR

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.

Authors+Show Affiliations

Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Radiology department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain. Centro de Investigación en Red en Enfermedades Raras (CIBERER), Madrid, Spain.Rehabilitation and physiotherapy department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Rehabilitation and physiotherapy department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Rehabilitation and physiotherapy department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Philips Healthcare Iberia, Madrid, Spain.Philips Healthcare Iberia, Madrid, Spain.Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain. Centro de Investigación en Red en Enfermedades Raras (CIBERER), Madrid, Spain.Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain. Centro de Investigación en Red en Enfermedades Raras (CIBERER), Madrid, Spain.No affiliation info availableNeuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain. jdiazm@santpau.cat. Centro de Investigación en Red en Enfermedades Raras (CIBERER), Madrid, Spain. jdiazm@santpau.cat.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

30022036

Citation

Figueroa-Bonaparte, Sebastian, et al. "Quantitative Muscle MRI to Follow Up Late Onset Pompe Patients: a Prospective Study." Scientific Reports, vol. 8, no. 1, 2018, p. 10898.
Figueroa-Bonaparte S, Llauger J, Segovia S, et al. Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study. Sci Rep. 2018;8(1):10898.
Figueroa-Bonaparte, S., Llauger, J., Segovia, S., Belmonte, I., Pedrosa, I., Montiel, E., ... Díaz-Manera, J. (2018). Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study. Scientific Reports, 8(1), p. 10898. doi:10.1038/s41598-018-29170-7.
Figueroa-Bonaparte S, et al. Quantitative Muscle MRI to Follow Up Late Onset Pompe Patients: a Prospective Study. Sci Rep. 2018 Jul 18;8(1):10898. PubMed PMID: 30022036.
* Article titles in AMA citation format should be in sentence-case
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 - ,, AU - Díaz-Manera,Jordi, Y1 - 2018/07/18/ PY - 2018/02/06/received PY - 2018/07/03/accepted PY - 2018/7/20/entrez PY - 2018/7/20/pubmed PY - 2019/11/15/medline SP - 10898 EP - 10898 JF - Scientific reports JO - Sci Rep VL - 8 IS - 1 N2 - 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. SN - 2045-2322 UR - https://www.unboundmedicine.com/medline/citation/30022036/Quantitative_muscle_MRI_to_follow_up_late_onset_Pompe_patients:_a_prospective_study_ L2 - http://dx.doi.org/10.1038/s41598-018-29170-7 DB - PRIME DP - Unbound Medicine ER -