dc.contributor.author |
Koeks, Z |
|
dc.contributor.author |
Bladen, CL |
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dc.contributor.author |
Salgado, D |
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dc.contributor.author |
van Zwet, E |
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dc.contributor.author |
Pogoryelova, O |
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dc.contributor.author |
McMacken, G |
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dc.contributor.author |
Monges, S |
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dc.contributor.author |
Foncuberta, M |
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dc.contributor.author |
Kekou, K |
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dc.contributor.author |
Kosma, K |
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dc.contributor.author |
Dawkins, H |
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dc.contributor.author |
Lamont, L |
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dc.contributor.author |
Bellgard, MI |
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dc.contributor.author |
Roy, AJ |
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dc.contributor.author |
Chamova, T |
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dc.contributor.author |
Guergueltcheva, V |
|
dc.contributor.author |
Chan, S |
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dc.contributor.author |
Korngut, L |
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dc.contributor.author |
Campbell, C |
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dc.contributor.author |
Dai, Y |
|
dc.contributor.author |
Wang, J |
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dc.contributor.author |
Barisic, N |
|
dc.contributor.author |
Brabec, P |
|
dc.contributor.author |
Lahdetie, J |
|
dc.contributor.author |
Walter, MC |
|
dc.contributor.author |
Schreiber-Katz, O |
|
dc.contributor.author |
Karcagi, V |
|
dc.contributor.author |
Garami, M |
|
dc.contributor.author |
Herczegfalvi, Ágnes |
|
dc.contributor.author |
Viswanathan, V |
|
dc.contributor.author |
Bayat, F |
|
dc.contributor.author |
Buccella, F |
|
dc.contributor.author |
Ferlini, A |
|
dc.contributor.author |
Kimura, E |
|
dc.contributor.author |
van den Bergen, JC |
|
dc.contributor.author |
Rodrigues, M |
|
dc.contributor.author |
Roxburgh, R |
|
dc.contributor.author |
Lusakowska, A |
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dc.contributor.author |
Kostera-Pruszczyk, A |
|
dc.contributor.author |
Santos, R |
|
dc.contributor.author |
Neagu, E |
|
dc.contributor.author |
Artemieva, S |
|
dc.contributor.author |
Rasic, VM |
|
dc.contributor.author |
Vojinovic, D |
|
dc.contributor.author |
Posada, M |
|
dc.contributor.author |
Bloetzer, C |
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dc.contributor.author |
Klein, A |
|
dc.contributor.author |
Diaz-Manera, J |
|
dc.contributor.author |
Gallardo, E |
|
dc.contributor.author |
Karaduman, AA |
|
dc.contributor.author |
Oznur, T |
|
dc.contributor.author |
Topaloglu, H |
|
dc.contributor.author |
El, Sherif R |
|
dc.contributor.author |
Stringer, A |
|
dc.contributor.author |
Shatillo, AV |
|
dc.contributor.author |
Martin, AS |
|
dc.contributor.author |
Peay, HL |
|
dc.contributor.author |
Kirschner, J |
|
dc.contributor.author |
Flanigan, KM |
|
dc.contributor.author |
Straub, V |
|
dc.contributor.author |
Bushby, K |
|
dc.contributor.author |
Beroud, C |
|
dc.contributor.author |
Verschuuren, JJ |
|
dc.contributor.author |
Lochmuller, H |
|
dc.date.accessioned |
2021-12-10T08:23:57Z |
|
dc.date.available |
2021-12-10T08:23:57Z |
|
dc.date.issued |
2017 |
|
dc.identifier |
85035064593 |
|
dc.identifier.citation |
pagination=293-306;
journalVolume=4;
journalIssueNumber=4;
journalTitle=JOURNAL OF NEUROMUSCULAR DISEASES; |
|
dc.identifier.uri |
http://repo.lib.semmelweis.hu//handle/123456789/4699 |
|
dc.identifier.uri |
doi:10.3233/JND-170280 |
|
dc.description.abstract |
BACKGROUND: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population. OBJECTIVE: To determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients. METHODS: In this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age. RESULTS: Loss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old (p = 0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery (p < 0.001) or ventilatory support (p < 0.001) and there was a mild cardioprotective effect of corticosteroids in the patient population aged 20 years and older (p = 0.0035). Patients with a single deletion of exon 45 showed an increased survival in contrast to other single exon deletions. CONCLUSIONS: This study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field. |
|
dc.relation.ispartof |
urn:issn:2214-3599 |
|
dc.title |
Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database |
|
dc.type |
Journal Article |
|
dc.date.updated |
2018-02-06T07:47:21Z |
|
dc.language.rfc3066 |
en |
|
dc.identifier.mtmt |
3291033 |
|
dc.identifier.pubmed |
29125504 |
|
dc.contributor.department |
SE/AOK/K/II. Sz. Gyermekgyógyászati Klinika |
|
dc.contributor.institution |
Semmelweis Egyetem |
|