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24-08-2023 | Amyotrophic Lateral Sclerosis | Original Communication

Interaction of sex and onset site on the disease trajectory of amyotrophic lateral sclerosis

Authors: Juliette Ortholand, Pierre-François Pradat, Sophie Tezenas du Montcel, Stanley Durrleman

Published in: Journal of Neurology | Issue 12/2023

Abstract

Background

Studies showed the impact of sex and onset site (spinal or bulbar) on disease onset and survival in ALS. However, they mainly result from cross-sectional or survival analysis, and the interaction of sex and onset site on the different proxies of disease trajectory has not been fully investigated.

Methods

We selected all patients with repeated observations in the PRO-ACT database. We divided them into four groups depending on their sex and onset site. We estimated a multivariate disease progression model, named ALS Course Map, to investigate the combined temporal changes of the four sub-scores of the revised ALS functional rating scale (ALSFRSr), the forced vital capacity (FVC), and the body mass index (BMI). We then compared the progression rate, the estimated age at onset, and the relative progression of the outcomes across each group.

Results

We included 1438 patients from the PRO-ACT database. They were 51% men with spinal onset, 12% men with bulbar onset, 26% women with spinal onset, and 11% women with bulbar onset. We showed a significant influence of both sex and onset site on the ALSFRSr progression. The BMI decreased 8.9 months earlier (95% CI [3.9, 13.8]) in women than men, after correction for the onset site. Among patients with bulbar onset, FVC was impaired 2.6 months earlier (95% CI [0.6, 4.6]) in women.

Conclusion

Using a multivariable disease modelling approach, we showed that sex and onset site are important drivers of the progression of motor function, BMI, and FVC decline.

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Title: Interaction of sex and onset site on the disease trajectory of amyotrophic lateral sclerosis
Authors: Juliette Ortholand
Pierre-François Pradat
Sophie Tezenas du Montcel
Stanley Durrleman
Publication date: 24-08-2023
Publisher: Springer Berlin Heidelberg
Keyword: Amyotrophic Lateral Sclerosis
Published in: Journal of Neurology / Issue 12/2023
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI: https://doi.org/10.1007/s00415-023-11932-7

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Springer Medicine

Interaction of sex and onset site on the disease trajectory of amyotrophic lateral sclerosis

Abstract

Background

Methods

Results

Conclusion

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Results

Conclusion

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