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

Figures

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  Figure 1. Digitized data from two separate publications showing individual 6‐minute walk test (6MWT) trajectories on the y‐axis and the subject age on the x‐axis. The red lines indicate data from the Goemans et al.6 2013 publication and the black lines indicate data from the McDonald et al.8 2013 publication. Details of data digitization can be found in the Methods section.

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  Figure 2. Population (a) and individual (b) fits of the data to model 4. The digitized data is indicated with red circles and the population prediction in blue circles on the left plot a and individual predictions in multicolors for each individual on the right plot b. 6MWT, 6‐minute walk test.

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  Figure 3. Goodness of fit plots are presented for model 4. Observed data vs. population predicted (PRED) (a) and individual predicted (IPRED) (b). The line of unity is indicated. Conditional weighted residuals (CWRES) are plotted vs. age (c) and predicted 6‐minute walk test (6MWT) (d). The blue curve is the loess curve for the residuals. The top red curve is the loess curve for the absolute values of the residuals and the bottom red is a reflection of the top curve; these show the trend in the magnitude of the residuals.

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  Figure 4. A visual prediction check using 2,000 replicates. The observed data are indicated with blue open circles. The observed quantiles are in red and the predicted quantiles are in black. The dotted line represents the 95th percentile, the dashed line the 50th percentile, and the solid line the 5th percentile. 6MWT, 6‐minute walk test.