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Developing a Natural History Progression Model for Duchenne Muscular Dystrophy Using the Six-Minute Walk Test.


ABSTRACT: The 6-minute walk test (6MWT) is used as a clinical endpoint to evaluate drug efficacy in Duchenne Muscular Dystrophy (DMD) trials. A model was developed using digitized 6MWT data that estimated two slopes and two intercepts to characterize 6MWT improvement during development and 6MWT decline. Mean baseline 6MWT was 362 (±87) meters. The model predicted an improvement at a rate of 20 meters/year (95% confidence interval (CI) = 9.4-30) up until 10 years old (95% CI?=?6.78-13.1), and then a decline at a rate of 85 meters/year (95% CI?=?72-98). Interpatient slope variability for improvement and decline were similar at 21.9 percentage of coefficient of variation (%CV) and 23.3%CV, respectively. Model simulations using age demographics from a previous DMD natural history study could reasonably predict the trend in improvement and decline in the 6MWT. This model can be used to quantitate individual patient trajectories, identify prognostic factors for disease progression, and evaluate drug effect.

SUBMITTER: Hamuro L 

PROVIDER: S-EPMC5613187 | BioStudies | 2017-01-01

REPOSITORIES: biostudies

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