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Clinical Outcomes In Duchenne Muscular Dystrophy: A Study Of 5345 Patients From The Treat-Nmd Dmd Global Database

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Author
Koeks, Zaïda
Bladen, Catherine L.
Salgado, David
van Zwet, Erik
Pogoryelova, Oksana
McMacken, Grace
Monges, Soledad
Foncuberta, Maria E.
Kekou, Kyriaki
Kosma, Konstantina
Dawkins, Hugh
Lamont, Leanne
Bellgard, Matthew I.
Roy, Anna J.
Chamova, Teodora
Guergueltcheva, Velina
Chan, Sophelia
Korngut, Lawrence
Campbell, Craig
Dai, Yi
Wang, Jen
Barišić, Nina
Brabec, Petr
Lähdetie, Jaana
Walter, Maggie C.
Schreiber-Katz, Olivia
Karcagi, Veronika
Garami, Marta
Herczegfalvi, Agnes
Viswanathan, Venkatarman
Bayat, Farhad
Buccella, Filippo
Ferlini, Alessandra
Kimura, En
van den Bergen, Janneke C.
Rodrigues, Miriam
Roxburgh, Richard
Lusakowska, Anna
Kostera-Pruszczyk, Anna
Santos, Rosário
Neagu, Elena
Artemieva, Svetlana
Rasic, Vedrana Milic
Vojinovic, Dina
Posada, Manuel
Bloetzer, Clemens
Klein, Andrea
Díaz-Manera, Jordi
Gallardo, Eduard
Karaduman, A. Ayşe
Oznur, Tunca
Topaloğlu, Haluk
El Sherif, Rasha
Stringer, Angela
Shatillo, Andriy V.
Martin, Ann S.
Peay, Holly L.
Kirschner, Jan
Flanigan, Kevin M.
Straub, Volker
Bushby, Kate
Béroud, Christophe
Verschuuren, Jan J.
Lochmüller, Hanns
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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.
URI
https://doi.org/10.3233/JND-170280
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5701764/
http://hdl.handle.net/11655/20890
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