Understanding North Star Ambulatory Assessment total scores and their implications for standards of care using observational data

Georgia Stimpson; Meredith K James; Michela Guglieri; Amy Wolfe; Adnan Manzur; Anna Sarkozy; Giovanni Baranello; Francesco Muntoni; Anna Mayhew; UK NorthStar Clinical Network

doi:10.1016/j.ejpn.2024.09.004

Understanding North Star Ambulatory Assessment total scores and their implications for standards of care using observational data

Eur J Paediatr Neurol. 2024 Nov:53:123-130. doi: 10.1016/j.ejpn.2024.09.004. Epub 2024 Sep 27.

Authors

Georgia Stimpson¹, Meredith K James², Michela Guglieri², Amy Wolfe³, Adnan Manzur⁴, Anna Sarkozy⁴, Giovanni Baranello⁴, Francesco Muntoni⁴, Anna Mayhew⁵; UK NorthStar Clinical Network

Affiliations

¹ Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
² Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
³ Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.
⁴ Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.
⁵ Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Electronic address: [email protected].

PMID: 39500128
DOI: 10.1016/j.ejpn.2024.09.004

Abstract

NorthStar Ambulatory Assessment (NSAA) total score (TS) is an ordinal scale to evaluate disease progression and treatment response in ambulatory Duchenne Muscular Dystrophy individuals. Clinical management according to standard of care could be enhanced by understanding how changes in the TS could inform standards of care. Here we describe the associated item performance patterns in the NorthStar Database for ranges of NSAA TS and its timed tests (10 m walk/run and rise from floor). We then compare these patterns depending on whether a participant is on an improving/stable (≤2-point loss in the prior year) or declining (>2-point loss in the prior year) trend. These TS and trends are subsequently linked and referenced to therapy standards of care. We included 761 participants from the UK NorthStar observational clinical database between 5 and 16 years, who were on steroids. Differences and trends in item ability, compensations, and times can suggest specific disease complications and lead towards anticipatory therapy recommendations. Families and therapists can benefit from using the TS and trend to guide therapy management.

Keywords: Duchenne muscular dystrophy; Motor function; Standards of care.

Publication types

Observational Study

MeSH terms

Adolescent
Child
Child, Preschool
Databases, Factual
Disease Progression
Female
Humans
Male
Muscular Dystrophy, Duchenne* / physiopathology
Muscular Dystrophy, Duchenne* / therapy
Standard of Care*
Walking / physiology