The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing

Erik Henricson1, Richard Abresch1, Jay J Han1

  • 1Department of Physical Medicine and Rehabilitation, University of California, Davis, Sacramento, California, USA.

Plos Currents
|January 27, 2016
PubMed

Insights

Minimal clinically important differences in Duchenne muscular dystrophy (DMD) vary by functional ability. Smaller 6-minute walk test (6MWT) gains yield meaningful quality of life improvements in lower-functioning individuals with DMD.

Area of Science:

  • Pediatric Orthopedics
  • Neuromuscular Disorders
  • Clinical Outcome Measurement

Background:

  • Establishing minimal clinically important differences (MCID) for the 6-minute walk test (6MWT) in Duchenne muscular dystrophy (DMD) is crucial for assessing treatment efficacy.
  • Current data lacks clear anchors for a 30-meter longitudinal change in 6MWT as an MCID, especially concerning patient-reported outcomes (PROs) across different ambulatory abilities.

Purpose of the Study:

  • To correlate measures of walking ability and patient-reported outcomes (PROs) in ambulatory Duchenne muscular dystrophy (DMD) patients.
  • To determine if MCIDs of PROs contribute to different estimates of 6-minute walk distance (6MWD) change at varying functional levels.
  • To establish clinically meaningful changes in PROs and their relationship to 6MWD in DMD.

Main Methods:

  • Correlated the 6-minute walk test (6MWT), 10-meter run/walk velocity, PedsQL, and POSNA Pediatric Outcomes Data Collection Instrument (PODCI) in 24 ambulatory DMD patients and 36 controls.
  • Analyzed 1-year changes in these measures and their correlations.
  • Assessed MCIDs for PROs and their impact on 6MWD change estimates.

Main Results:

  • Significant differences in PedsQL and PODCI scores were observed between DMD patients and controls (p<0.00001).
  • PODCI transfer/mobility scores showed the strongest correlation with 6MWD and 10-meter run/walk velocity in DMD (r=0.79 and r=0.76, respectively).
  • One-year changes in 6MWD, 10-meter run/walk velocity, and PODCI scores exceeded MCID. 6MWD change strongly correlated with PODCI changes (r=0.76-0.93).

Conclusions:

  • Smaller increases in 6-minute walk distance (6MWD) correlate with meaningful quality of life improvements at lower functional levels in Duchenne muscular dystrophy (DMD).
  • Larger 6MWD gains may be required to achieve similar quality of life improvements at higher functional levels.
  • PODCI scores effectively estimate 6MWT performance and clinical meaningfulness in DMD.
Abstract

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