Bayesian model of disease progression in mucopolysaccaridosis IIIA

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 meas...

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Bibliographic Details
Published inStatistics in medicine Vol. 41; no. 18; pp. 3579 - 3595
Main Authors Marion, Joe, Ruiz, Juan, Saville, Benjamin R.
Format Journal Article
LanguageEnglish
Published England Wiley Subscription Services, Inc 15.08.2022
Subjects
Bayesian
clinical trial
disease progression model
MPS IIIA
rare disease
clinical trial
disease progression model
rare disease
MPS IIIA
Bayesian
Online AccessGet full text
ISSN0277-6715
1097-0258
1097-0258
DOI10.1002/sim.9435

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Summary: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.
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ISSN:0277-6715
1097-0258
1097-0258
DOI:10.1002/sim.9435