Details

Autor(en) / Beteiligte

• Mcdonald, Craig M.
• Henricson, Erik K.
• Abresch, R. Ted
• Florence, Julaine M.
• Eagle, Michelle
• Gappmaier, Eduard
• Glanzman, Allan M.
• Spiegel, Robert
• Barth, Jay
• Elfring, Gary
• Reha, Allen
• Peltz, Stuart

Titel

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

Ist Teil von

• Muscle & nerve, 2013-09, Vol.48 (3), p.343-356

Ort / Verlag

United States: Blackwell Publishing Ltd

Erscheinungsjahr

2013

Link zum Volltext

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• ABSTRACT Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double‐blind, placebo‐controlled trial of ataluren. Placebo arm data (N = 57) provided insight into the natural history of the 6‐minute walk test (6MWT) and other endpoints. Methods: Evaluations performed every 6 weeks included the 6‐minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand‐held myometry. Results: 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. Conclusion: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials. Muscle Nerve 48: 343–356, 2013