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http://doi.org/10.25358/openscience-490
Autoren: | Lollert, André Stihl, Clemens Hötker, Andreas Mengel, Eugen König, Jochem Laudemann, Katharina Gökce, Seyfullah Düber, Christoph Staatz, Gundula |
Titel: | Quantification of intramuscular fat in patients with late-onset Pompe disease by conventional magnetic resonance imaging for the long-term follow-up of enzyme replacement therapy |
Online-Publikationsdatum: | 11-Jan-2018 |
Erscheinungsdatum: | 2018 |
Sprache des Dokuments: | Englisch |
Zusammenfassung/Abstract: | OBJECTIVE: The objective of this study was to evaluate a quantitative method based on conventional T1-weighted magnetic resonance (MR) imaging to assess fatty muscular degeneration in patients with late-onset Pompe disease and to compare it with semi-quantitative visual evaluation (the Mercuri score). In addition, a long-term retrospective data analysis was performed to evaluate treatment response to enzyme replacement therapy with alglucosidase alfa. METHODS: MR images of the lumbar spine were acquired in 41 patients diagnosed with late-onset Pompe disease from 2006 through 2015. Two independent readers retrospectively evaluated fatty degeneration of the psoas and paraspinal muscles by applying the Mercuri score. Quantitative semi-automated muscle and fat tissue separation was performed, and inter-observer agreement and correlations with clinical parameters were assessed. Follow-up examinations were performed in 13 patients treated with alglucosidase alfa after a median of 39 months; in 7/13 patients, an additional follow-up examination was completed after a median of 63 months. RESULTS: Inter-observer agreement was high. Measurements derived from the quantitative method correlated well with Medical Research Council scores of muscle strength, with moderate correlations found for the 6-minute walk test, the 4-step stair climb test, and spirometry in the supine position. A significant increase in the MR-derived fat fraction of the psoas muscle was found between baseline and follow-up 1 (P = 0.016), as was a significant decrease in the performance on the 6-minute walk test (P = 0.006) and 4-step stair climb test (P = 0.034), as well as plasma creatine kinase (P = 0.016). No statistically significant difference in clinical or MR-derived parameters was found between follow-up 1 and follow-up 2. CONCLUSIONS: Quantification of fatty muscle degeneration using the semi-automated method can provide a more detailed overview of disease progression than semi-quantitative Mercuri scoring. MR-derived data correlated with clinical symptoms and patient exercise capacity. After an initial worsening, the fat fraction of the psoas muscle and performance on the 6-minute walk test stayed constant during long-term follow-up under enzyme replacement therapy. |
DDC-Sachgruppe: | 610 Medizin 610 Medical sciences |
Veröffentlichende Institution: | Johannes Gutenberg-Universität Mainz |
Organisationseinheit: | FB 04 Medizin |
Veröffentlichungsort: | Mainz |
ROR: | https://ror.org/023b0x485 |
DOI: | http://doi.org/10.25358/openscience-490 |
URN: | urn:nbn:de:hebis:77-publ-577604 |
Version: | Published version |
Publikationstyp: | Zeitschriftenaufsatz |
Nutzungsrechte: | CC BY |
Informationen zu den Nutzungsrechten: | https://creativecommons.org/licenses/by/4.0/ |
Zeitschrift: | PLoS one 13 1 |
Seitenzahl oder Artikelnummer: | e0190784 |
Verlag: | PLoS |
Verlagsort: | Lawrence, Kan. |
Erscheinungsdatum: | 2018 |
ISSN: | 1932-6203 |
URL der Originalveröffentlichung: | http://dx.doi.org/10.1371/journal.pone.0190784 |
DOI der Originalveröffentlichung: | 10.1371/journal.pone.0190784 |
Enthalten in den Sammlungen: | JGU-Publikationen |
Dateien zu dieser Ressource:
Datei | Beschreibung | Größe | Format | ||
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57760.pdf | 5.1 MB | Adobe PDF | Öffnen/Anzeigen |