Bayesian model of disease progression in mucopolysaccaridosis IIIA.
Bayesian
MPS IIIA
clinical trial
disease progression model
rare disease
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
15 08 2022
15 08 2022
Historique:
revised:
25
03
2022
received:
13
10
2021
accepted:
22
04
2022
pubmed:
15
5
2022
medline:
20
7
2022
entrez:
14
5
2022
Statut:
ppublish
Résumé
Mucopolysaccaridosis IIIA (MPS IIIA) is a rare genetic disease that afflicts children and leads to neurocognitive degeneration. We develop a Bayesian disease progression model (DPM) of MPS IIIA that characterizes the pattern of cognitive growth and decline in this disease. The DPM is a repeated measures model that incorporates a nonlinear developmental trajectory and shape-invariant random effects. This approach quantifies the pattern of cognitive development in MPS IIIA and addresses differences in biological age, length of follow-up, and clinical outcomes across natural history subjects. The DPM can be used in clinical trials to estimate the percent slowing in disease progression for treatment relative to natural history. Simulations demonstrate that the DPM provides substantial improvements in power relative to alternative analyses.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
3579-3595Informations de copyright
© 2022 John Wiley & Sons Ltd.
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