Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy.

X-linked adrenoleukodystrophy balance body sway myelopathy spinal cord surrogate outcome

Journal

Frontiers in physiology
ISSN: 1664-042X
Titre abrégé: Front Physiol
Pays: Switzerland
ID NLM: 101549006

Informations de publication

Date de publication:
2020
Historique:
received: 10 03 2020
accepted: 15 06 2020
entrez: 9 8 2020
pubmed: 9 8 2020
medline: 9 8 2020
Statut: epublish

Résumé

Myelopathy is the core clinical manifestation of adrenoleukodystrophy (ALD), which is the most common peroxisomal disorder. Development of therapies requires sensitive and clinically relevant outcome measures. Together with spastic paraparesis, balance disturbance is the main cause of disability from myelopathy in ALD. In this cross-sectional study, we evaluated whether postural body sway - a measure of balance - could serve as a surrogate outcome in clinical trials. Forty-eight male ALD patients and 49 age-matched healthy male controls were included in this study. We compared sway amplitude and sway path of ALD patients to controls. We then correlated the body sway parameters showing the largest between-group differences with clinical measures of severity of myelopathy. To correct for age, we performed multiple linear regression analysis with age and severity of myelopathy as independent variables. All body sway parameters were significantly higher in patients than in controls, with medium to large effect sizes ( These results indicate that postural body sway may serve as a surrogate outcome for myelopathy in ALD. Such outcomes are important to evaluate new therapies in clinical trials. Further longitudinal studies are needed and ongoing in this cohort.

Sections du résumé

BACKGROUND BACKGROUND
Myelopathy is the core clinical manifestation of adrenoleukodystrophy (ALD), which is the most common peroxisomal disorder. Development of therapies requires sensitive and clinically relevant outcome measures. Together with spastic paraparesis, balance disturbance is the main cause of disability from myelopathy in ALD. In this cross-sectional study, we evaluated whether postural body sway - a measure of balance - could serve as a surrogate outcome in clinical trials.
METHODS METHODS
Forty-eight male ALD patients and 49 age-matched healthy male controls were included in this study. We compared sway amplitude and sway path of ALD patients to controls. We then correlated the body sway parameters showing the largest between-group differences with clinical measures of severity of myelopathy. To correct for age, we performed multiple linear regression analysis with age and severity of myelopathy as independent variables.
RESULTS RESULTS
All body sway parameters were significantly higher in patients than in controls, with medium to large effect sizes (
CONCLUSIONS CONCLUSIONS
These results indicate that postural body sway may serve as a surrogate outcome for myelopathy in ALD. Such outcomes are important to evaluate new therapies in clinical trials. Further longitudinal studies are needed and ongoing in this cohort.

Identifiants

DOI: 10.3389/fphys.2020.00786 PMID: 32765293 PMC: PMC7379508
pubmed: 32765293
doi: 10.3389/fphys.2020.00786
pmc: PMC7379508
doi:

Types de publication

Journal Article

Langues

eng

Pagination

786

Informations de copyright

Copyright © 2020 van Ballegoij, van de Stadt, Huffnagel, Kemp, van der Knaap and Engelen.

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Auteurs

Wouter J C van Ballegoij (WJC)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.
Department of Neurology, OLVG Hospital, Amsterdam, Netherlands.

Stephanie I W van de Stadt (SIW)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.

Irene C Huffnagel (IC)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.

Stephan Kemp (S)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.
Laboratory Genetic Metabolic Diseases, Department of Clinical Chemistry, Amsterdam University Medical Centers, Amsterdam Gastroenterology & Metabolism, University of Amsterdam, Amsterdam, Netherlands.

Marjo S van der Knaap (MS)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.
Department of Functional Genomics, Vrije Universiteit Amsterdam, Amsterdam, Netherlands.

Marc Engelen (M)

Department of Pediatric Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.

Classifications MeSH