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Statistical Approaches to Longitudinal Data Analysis in Neurodegenerative Diseases: Huntington’s Disease as a Model

  • Dementia (KS Marder, Section Editor)
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Abstract

Understanding the overall progression of neurodegenerative diseases is critical to the timing of therapeutic interventions and design of effective clinical trials. Disease progression can be assessed with longitudinal study designs in which outcomes are measured repeatedly over time and are assessed with respect to risk factors, either measured repeatedly or at baseline. Longitudinal data allows researchers to assess temporal disease aspects, but the analysis is complicated by complex correlation structures, irregularly spaced visits, missing data, and mixtures of time-varying and static covariate effects. We review modern statistical methods designed for these challenges. Among all methods, the mixed effect model most flexibly accommodates the challenges and is preferred by the FDA for observational and clinical studies. Examples from Huntington’s disease studies are used for clarification, but the methods apply to neurodegenerative diseases in general, particularly as the identification of prodromal forms of neurodegenerative disease through sensitive biomarkers is increasing.

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Acknowledgements

The authors would like to give a special thank you to Dr. Susan Fox for taking the time to review this manuscript.

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Authors and Affiliations

  1. Department of Epidemiology and Biostatistics, Texas A&M University, TAMU 1266, College Station, TX, 77843-1266, USA

    Tanya P. Garcia

  2. Department of Neurology, Columbia University, 630 West 168th Street, New York, NY, 10032, USA

    Karen Marder

Authors
  1. Tanya P. Garcia
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  2. Karen Marder
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Corresponding author

Correspondence to Tanya P. Garcia.

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Conflict of Interest

Tanya P. Garcia declares that she has no conflict of interest. Karen Marder reports grants from the Huntington’s Disease Society of America, CHDI, TEVA, 1UL1 RR024156-01, and non-financial support from Raptor Pharmaceutical.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

Funding

This work is supported in part by the National Institute Of Neurological Disorders And Stroke of the National Institutes of Health under Award Number K01NS099343, the Huntington’s Disease Society of America Human Biology Project Fellowship, Texas A&M School of Public Health Research Enhancement and Development Initiative (REDI-23-202059-36000), and National Center for Advancing Translational Sciences (2UL1RR024156-06).

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This article is part of the Topical Collection on Dementia

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Garcia, T.P., Marder, K. Statistical Approaches to Longitudinal Data Analysis in Neurodegenerative Diseases: Huntington’s Disease as a Model. Curr Neurol Neurosci Rep 17, 14 (2017). https://doi.org/10.1007/s11910-017-0723-4

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  • DOI: https://doi.org/10.1007/s11910-017-0723-4

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