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The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM, PTC124-GD-007-DMD Study GroupSpiegel R, Barth J, Elfring G, Reha A, Peltz S - Muscle Nerve (2013)

Bottom Line: Baseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD.A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters.Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation.

View Article: PubMed Central - PubMed

Affiliation: Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Davis, California, 95817, USA.

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(a) Change in baseline time to stand from supine in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 32) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (b) Change in time to climb 4 stairs in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (c) Change in baseline time to run/walk 10 m in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation.
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fig02: (a) Change in baseline time to stand from supine in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 32) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (b) Change in time to climb 4 stairs in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (c) Change in baseline time to run/walk 10 m in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation.

Mentions: For the 40 patients on stable doses of glucocorticoids at the initiation of the study baseline the TFT results [mean (SD)] for 6 subjects <7 years and 34 subjects ≥7 years, respectively, were as follows: time to rise from supine = 4.0 s (1.13) and 12.9 s (12.33); time to climb 4 stairs = 2.5 s (0.67) and 6.6 s (6.30); time to run/walk 10 m = 4.8 s (0.86) and 7.1 s (2.80). Mean changes from baseline ± SEM over 48 weeks are plotted in Figure 2a–c. For all TFTs there was little change over 48 weeks in patients <7 years of age, whereas for patients ≥7 years there were greater changes in TFT values. In general, the SD and SEM of the change scores from baseline increase over time due to variability in disease progression.


The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM, PTC124-GD-007-DMD Study GroupSpiegel R, Barth J, Elfring G, Reha A, Peltz S - Muscle Nerve (2013)

(a) Change in baseline time to stand from supine in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 32) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (b) Change in time to climb 4 stairs in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (c) Change in baseline time to run/walk 10 m in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation.
© Copyright Policy - open-access
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC3824082&req=5

fig02: (a) Change in baseline time to stand from supine in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 32) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (b) Change in time to climb 4 stairs in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation. (c) Change in baseline time to run/walk 10 m in seconds (mean ± SEM) for steroid-treated patients ≥7 years old (n = 34) and <7 years old (n = 6) with DMD over 48 weeks of evaluation.
Mentions: For the 40 patients on stable doses of glucocorticoids at the initiation of the study baseline the TFT results [mean (SD)] for 6 subjects <7 years and 34 subjects ≥7 years, respectively, were as follows: time to rise from supine = 4.0 s (1.13) and 12.9 s (12.33); time to climb 4 stairs = 2.5 s (0.67) and 6.6 s (6.30); time to run/walk 10 m = 4.8 s (0.86) and 7.1 s (2.80). Mean changes from baseline ± SEM over 48 weeks are plotted in Figure 2a–c. For all TFTs there was little change over 48 weeks in patients <7 years of age, whereas for patients ≥7 years there were greater changes in TFT values. In general, the SD and SEM of the change scores from baseline increase over time due to variability in disease progression.

Bottom Line: Baseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD.A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters.Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation.

View Article: PubMed Central - PubMed

Affiliation: Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Davis, California, 95817, USA.

Show MeSH
Related in: MedlinePlus