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AMIA Jt Summits Transl Sci Proc. 2019 May 6;2019:789-798. eCollection 2019.

G-Computation and Hierarchical Models for Estimating Multiple Causal Effects From Observational Disease Registries With Irregular Visits.

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IBM T. J. Watson Research Center, Yorktown Heights, NY, USA.
Cure Huntington's Disease Institute, Princeton, NJ, USA.


Huntington's Disease (HD) is a neurodegenerative disorder with serious motor, cognitive, and behavioral symptoms. Chorea, a motor symptom of HD characterized by abrupt involuntary movements, is typically treated with tetrabenazine or certain off-label antipsychotics. Clinical trial evidence about the impact of these drugs in the HD population is scant. However, multiple observational HD registries have recently been used with success to model HD progression1,2 and provide an opportunity to obtain effect estimates in the absence of clinical trials. We use a dataset integrated from four large-scale HD registries to generate evidence on the efficacy of chorea treatments for chorea as well as their impact on other aspects of HD progression. Clinical conclusions are meant only to illustrate our methodological approach. We employ parametric G-computation for causal inference to adjust for confounding and accommodate irregular visits and treatment patterns. We fit Bayesian hierarchical models to the results of multiple related analyses to share strength across studies and handle multiple comparisons concerns.


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