Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy

PLoS One. 2016 Aug 5;11(8):e0160195. doi: 10.1371/journal.pone.0160195. eCollection 2016.

Abstract

The advent of therapeutic approaches for Duchenne muscular dystrophy (DMD) has highlighted the need to identify reliable outcome measures for young boys with DMD. The aim of this study was to develop a revised version of the North Star Ambulatory Assessment (NSAA) suitable for boys between the age of 3 and 5 years by identifying age appropriate items and revising the scoring system accordingly. Using the scale in 171 controls between the age of 2.9 and 4.8 years, we identified items that were appropriate at different age points. An item was defined as age appropriate if it was completed, achieving a full score, by at least 85% of the typically developing boys at that age. At 3 years (±3months) there were only 8 items that were age appropriate, at 3 years and 6 months there were 13 items while by the age of 4 years all 17 items were appropriate. A revised version of the scale was developed with items ordered according to the age when they could be reliably performed. The application of the revised version of the scale to data collected in young DMD boys showed that very few of the DMD boys were able to complete with a full score all the age appropriate items. In conclusion, our study suggests that a revised version of the NSAA can be used in boys from the age of 3 years to obtain information on how young DMD boys acquire new abilities and how this correlates with their peers.

Publication types

  • Multicenter Study

MeSH terms

  • Case-Control Studies
  • Child Development / physiology
  • Child, Preschool
  • Exercise Test / methods*
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne / physiopathology*
  • Prospective Studies

Grants and funding

This work was supported by Telethon-Uildm (Award number: GUP15011) http://www.telethon.it/. Elena Mazzone is a TREAT NMD research fellow funded by the Italian Telethon. Francesco Muntoni is supported by the Great Ormond Street Hospital (GOSH) Children’s Charity and the GOSH Biomedical Research Centre. The support of the Muscular Dystrophy Campaign to the UK North Star Network is also gratefully acknowledged. The authors have stated that they had no interests which might be perceived as posing a conflict or bias. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.