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Clinical Outcomes in Duchenne Muscular Dystrophy: a Study of 5345 Patients from the TREAT-NMD DMD Global Database

Zaïda KoeksJanbernd KirschnerHanns Lochmüller

出版	Universität, 2017
URL	http://books.google.com.hk/books?id=kvqfzwEACAAJ&hl=&source=gbs_api

註釋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.br