Reliability of the North Star Ambulatory Assessment in a multicentric setting

E S Mazzone1, S Messina, G Vasco

  • 1Department of Paediatric Neurology, Catholic University, Rome, Italy.

Insights

The North Star Ambulatory Assessment (NSAA) is suitable for clinical trials in Duchenne Muscular Dystrophy. Adequate training is crucial for achieving reliable results in multicenter studies.

Area of Science:

  • Neurology
  • Clinical Trials
  • Musculoskeletal Disorders

Background:

  • Duchenne Muscular Dystrophy (DMD) requires reliable outcome measures for clinical trials.
  • The North Star Ambulatory Assessment (NSAA) is designed for ambulant children with DMD.

Purpose of the Study:

  • To assess the suitability of the NSAA as an outcome measure in multicenter clinical trials.
  • To determine the training required for good interobserver reliability with the NSAA in a multicenter setting.

Main Methods:

  • Thirteen Italian centers participated.
  • Examiners received initial training via videos and examples.
  • Interobserver reliability was assessed after the first training session.
  • A second training session involved video review and discussion.
  • Reliability was reassessed after the second training session and again after one month.

Main Results:

  • Initial assessment showed poor interobserver reliability (<.5).
  • Following a second training session, interobserver reliability improved significantly to .995.
  • High intra-observer reliability (.95) was maintained over one month.

Conclusions:

  • The NSAA is easily administered and takes approximately 10 minutes.
  • The NSAA is a viable outcome measure for multicenter trials.
  • Effective, targeted training is essential for achieving reliable NSAA scores in multicenter settings.