Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy.

ABSTRACT:

Background

Deficits in ambulatory function progress at heterogeneous rates among individuals with Duchenne muscular dystrophy (DMD). The resulting inherent variability in ambulatory outcomes has complicated the design of drug efficacy trials and clouded the interpretation of trial results. We developed a prediction model for 1-year change in the six minute walk distance (6MWD) among DMD patients, and compared its predictive value to that of commonly used prognostic factors (age, baseline 6MWD, and steroid use).

Methods

Natural history data were collected from DMD patients at routine follow up visits approximately every 6 months over the course of 2-5 years. Assessments included ambulatory function and steroid use. The annualized change in 6MWD (Δ6MWD) was studied between all

SUBMITTER: Goemans N 

PROVIDER: S-EPMC5063281 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature