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http://doi.org/10.25358/openscience-490Full metadata record
| DC Field | Value | Language |
|---|---|---|
| dc.contributor.author | Lollert, André | - |
| dc.contributor.author | Stihl, Clemens | - |
| dc.contributor.author | Hötker, Andreas | - |
| dc.contributor.author | Mengel, Eugen | - |
| dc.contributor.author | König, Jochem | - |
| dc.contributor.author | Laudemann, Katharina | - |
| dc.contributor.author | Gökce, Seyfullah | - |
| dc.contributor.author | Düber, Christoph | - |
| dc.contributor.author | Staatz, Gundula | - |
| dc.date.accessioned | 2018-01-11T11:40:04Z | - |
| dc.date.available | 2018-01-11T12:40:04Z | - |
| dc.date.issued | 2018 | - |
| dc.identifier.uri | https://openscience.ub.uni-mainz.de/handle/20.500.12030/492 | - |
| dc.description.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. | en_GB |
| dc.language.iso | eng | - |
| dc.rights | CC BY | de_DE |
| dc.rights.uri | https://creativecommons.org/licenses/by/4.0/ | - |
| dc.subject.ddc | 610 Medizin | de_DE |
| dc.subject.ddc | 610 Medical sciences | en_GB |
| dc.title | 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 | en_GB |
| dc.type | Zeitschriftenaufsatz | de_DE |
| dc.identifier.urn | urn:nbn:de:hebis:77-publ-577604 | - |
| dc.identifier.doi | http://doi.org/10.25358/openscience-490 | - |
| jgu.type.dinitype | article | - |
| jgu.type.version | Published version | en_GB |
| jgu.type.resource | Text | - |
| jgu.organisation.department | FB 04 Medizin | - |
| jgu.organisation.number | 2700 | - |
| jgu.organisation.name | Johannes Gutenberg-Universität Mainz | - |
| jgu.rights.accessrights | openAccess | - |
| jgu.journal.title | PLoS one | - |
| jgu.journal.volume | 13 | - |
| jgu.journal.issue | 1 | - |
| jgu.pages.alternative | e0190784 | - |
| jgu.publisher.year | 2018 | - |
| jgu.publisher.name | PLoS | - |
| jgu.publisher.place | Lawrence, Kan. | - |
| jgu.publisher.uri | http://dx.doi.org/10.1371/journal.pone.0190784 | - |
| jgu.publisher.issn | 1932-6203 | - |
| jgu.organisation.place | Mainz | - |
| jgu.subject.ddccode | 610 | - |
| opus.date.accessioned | 2018-01-11T11:40:04Z | - |
| opus.date.modified | 2018-04-03T09:02:58Z | - |
| opus.date.available | 2018-01-11T12:40:04 | - |
| opus.subject.dfgcode | 04-205 | - |
| opus.organisation.string | FB 04: Medizin: Klinik und Poliklinik für Diagnostische und Interventionelle Radiologie | de_DE |
| opus.organisation.string | FB 04: Medizin: Institut für Med. Biometrie, Epidemologie und Informatik | de_DE |
| opus.organisation.string | FB 04: Medizin: Kinderklinik und Kinderpoliklinik | de_DE |
| opus.identifier.opusid | 57760 | - |
| opus.institute.number | 0419 | - |
| opus.institute.number | 0424 | - |
| opus.institute.number | 0429 | - |
| opus.metadataonly | false | - |
| opus.type.contenttype | Keine | de_DE |
| opus.type.contenttype | None | en_GB |
| opus.affiliated | Lollert, André | - |
| opus.affiliated | Hötker, Andreas | - |
| opus.affiliated | Mengel, Eugen | - |
| opus.affiliated | König, Jochem | - |
| opus.affiliated | Laudemann, Katharina | - |
| opus.affiliated | Düber, Christoph | - |
| opus.affiliated | Staatz, Gundula | - |
| jgu.publisher.doi | 10.1371/journal.pone.0190784 | |
| jgu.organisation.ror | https://ror.org/023b0x485 | |
| Appears in collections: | JGU-Publikationen | |
