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Article: Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database
Title | Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database |
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Authors | |
Keywords | DMD Duchenne muscular dystrophy Neuromuscular diseases TREAT-NMD |
Issue Date | 2017 |
Publisher | IOS Press. The Journal's web site is located at https://www.iospress.nl/journal/journal-of-neuromuscular-diseases/ |
Citation | Journal of Neuromuscular Diseases, 2017, v. 4 n. 4, p. 293-306 How to Cite? |
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 ofDMDacross 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. |
Persistent Identifier | http://hdl.handle.net/10722/260561 |
ISSN | 2023 Impact Factor: 3.2 2023 SCImago Journal Rankings: 0.949 |
PubMed Central ID |
DC Field | Value | Language |
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dc.contributor.author | Koeks, Z | - |
dc.contributor.author | Bladen, CL | - |
dc.contributor.author | Van Zwet, E | - |
dc.contributor.author | Pogoryelova, O | - |
dc.contributor.author | Salgado, D | - |
dc.contributor.author | Chan, HSS | - |
dc.date.accessioned | 2018-09-14T08:43:45Z | - |
dc.date.available | 2018-09-14T08:43:45Z | - |
dc.date.issued | 2017 | - |
dc.identifier.citation | Journal of Neuromuscular Diseases, 2017, v. 4 n. 4, p. 293-306 | - |
dc.identifier.issn | 2214-3599 | - |
dc.identifier.uri | http://hdl.handle.net/10722/260561 | - |
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 ofDMDacross 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.language | eng | - |
dc.publisher | IOS Press. The Journal's web site is located at https://www.iospress.nl/journal/journal-of-neuromuscular-diseases/ | - |
dc.relation.ispartof | Journal of Neuromuscular Diseases | - |
dc.subject | DMD | - |
dc.subject | Duchenne muscular dystrophy | - |
dc.subject | Neuromuscular diseases | - |
dc.subject | TREAT-NMD | - |
dc.title | Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database | - |
dc.type | Article | - |
dc.identifier.email | Chan, HSS: sophehs@hku.hk | - |
dc.identifier.authority | Chan, HSS=rp02210 | - |
dc.description.nature | link_to_OA_fulltext | - |
dc.identifier.doi | 10.3233/JND-170280 | - |
dc.identifier.pmcid | PMC5701764 | - |
dc.identifier.scopus | eid_2-s2.0-85035064593 | - |
dc.identifier.hkuros | 290970 | - |
dc.identifier.volume | 4 | - |
dc.identifier.issue | 4 | - |
dc.identifier.spage | 293 | - |
dc.identifier.epage | 306 | - |
dc.publisher.place | Netherlands | - |
dc.identifier.issnl | 2214-3599 | - |