Abstract
Many clinical trials collect information on multiple longitudinal outcomes. Depending on the nature of the disease and its symptoms, the longitudinal outcomes can be of mixed types, e.g., binary, ordinal and continuous. Clinical studies on Parkinson’s disease (PD) are good examples of this case. Due to the multidimensional nature of PD, it is difficult to identify a single outcome to represent the overall disease status and severity. Thus, clinical studies that search for treatments for PD usually collect multiple outcomes at different visits. In this chapter, we will introduce the multilevel item response theory (MLIRT) models that account for all the information from multiple longitudinal outcomes and provide valid inference for the overall treatment effects. We will also introduce the normal/independent (NI) distributions, which can be easily implemented into the MLIRT model hierarchically, to handle the outlier and heavy tails problems to produce robust inference. Other data features such as dependent censoring and skewness will also be discussed under the MLIRT framework.
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References
Baghfalaki, T., Ganjali, M., & Berridge, D. (2013). Robust joint modeling of longitudinal measurements and time to event data using normal/independent distributions: A Bayesian approach. Biometrical Journal, 55(6), 844–865.
Bushnell, D. M., & Martin, M. L. (1999). Quality of life and Parkinson’s disease: Translation and validation of the US Parkinson’s disease questionnaire (PDQ-39). Quality of Life Research, 8(4), 345–350.
Carlin, B. P., & Louis, T. A. (2011). Bayesian methods for data analysis. Boca Raton, FL: Chapman & Hall.
Chen, G., & Luo, S. (2016). Robust Bayesian hierarchical model using normal/independent distributions. Biometrical Journal, 58(4), 831–851.
Chen, M. H., Shao, Q. M., & Ibrahim, J. G. (2000). Monte Carlo methods in Bayesian computation. New York: Springer Series in Statistics.
Cummings, J. L. (1992). Depression and Parkinson’s disease: A review. The American Journal of Psychiatry, 149(4), 443–454.
David, D. (2007). Dunson. Bayesian methods for latent trait modelling of longitudinal data. Statistical Methods in Medical Research, 16(5), 399–415.
Elm, J. J., & The NINDS NET-PD Investigators. (2012). Design innovations and baseline findings in a long-term Parkinson’s trial: The National Institute of Neurological Disorders and Stroke exploratory trials in Parkinson’s Disease Long-Term study-1. Movement Disorders, 27(12), 1513–1521.
Fahn, S., Oakes, D., Shoulson, I., Kieburtz, K., Rudolph, A., Lang, A., et al. (2004). Levodopa and the progression of Parkinson’s disease. The New England Journal of Medicine, 351(24), 2498–2508.
Fox, J. P. (2010). Bayesian item response modeling: Theory and applications. New York: Springer.
Geisser, S. (1993). Predictive inference: An introduction (Vol. 55). Boca Raton, FL: CRC Press.
Gelman, A., Carlin, J. B., Stern, H. S., Dunson, D. B., Vehtari, A., & Rubin, D. B. (2013). Bayesian data analysis. CRC Press.
He, B., & Luo, S. (2013). Joint modeling of multivariate longitudinal measurements and survival data with applications to Parkinson’s disease. Statistical Methods in Medical Research.
Henderson, R., Diggle, P., & Dobson, A. (2000). Joint modelling of longitudinal measurements and event time data. Biostatistics, 1(4), 465–480.
Huang, P., Tilley, B. C., Woolson, R. F., & Lipsitz, S. (2005). Adjusting O’Brien’s test to control type I error for the generalized nonparametric Behrens-Fisher problem. Biometrics, 61(2), 532–539.
Jasra, A., Holmes, C. C., & Stephens, D. A. (2005). Markov chain Monte Carlo methods and the label switching problem in Bayesian mixture modeling. Statistical Science, 20(1), 50–67.
Kamata, A. (2001). Item analysis by the hierarchical generalized linear model. Journal of Educational Measurement, 38(1), 79–93.
Kass, R. E., & Raftery, A. E. (1995). Bayes factors. Journal of the American Statistical Association, 90(430), 773–795.
Lachos, V. H., Bandyopadhyay, D., & Dey, D. K. (2011). Linear and nonlinear mixed-effects models for censored HIV viral loads using normal/independent distributions. Biometrics, 67(4), 1594–1604.
Lachos, V. H., Castro, L. M., & Dey, D. K. (2013). Bayesian inference in nonlinear mixed-effects models using normal independent distributions. Computational Statistics & Data Analysis, 64, 237–252.
Lachos, V. H., Dey, D. K., & Cancho, V. G. (2009). Robust linear mixed models with skew-normal independent distributions from a Bayesian perspective. Journal of Statistical Planning and Inference, 139(12), 4098–4110.
Lange, K., & Sinsheimer, J. S. (1993). Normal/independent distributions and their applications in robust regression. Journal of Computational and Graphical Statistics, 2(2), 175–198.
Lavine, M., & Schervish, M. J. (1999). Bayes factors: What they are and what they are not. The American Statistician, 53(2), 119–122.
Lee, S.-Y., & Song, X.-Y. (2004). Evaluation of the bayesian and maximum likelihood approaches in analyzing structural equation models with small sample sizes. Multivariate Behavioral Research, 39(4), 653–686.
Lewis, S. M., & Raftery, A. E. (1997). Estimating Bayes factors via posterior simulation with the Laplace-Metropolis estimator. Journal of the American Statistical Association, 92(438), 648–655.
Liu, C. (1996). Bayesian robust multivariate linear regression with incomplete data. Journal of the American Statistical Association, 91(435), 1219–1227.
Lord, F. M., Novick, M. R., & Birnbaum, A. (1968). Statistical theories of mental test scores. Boston, MA: Addison-Wesley.
Luo, S. (2014). A Bayesian approach to joint analysis of multivariate longitudinal data and parametric accelerated failure time. Statistics in Medicine, 33(4), 580–594.
Luo, S., & Wang, J. (2014). Bayesian hierarchical model for multiple repeated measures and survival data: An application to parkinson’s disease. Statistics in Medicine, 33(24), 4279–4291.
Luo, S., Lawson, A. B., He, B., Elm, J. J., & Tilley, B. C. (2012). Bayesian multiple imputation for missing multivariate longitudinal data from a Parkinson’s disease clinical trial. Statistical Methods in Medical Research.
Luo, S., Ma, J., & Kieburtz, K. D. (2013). Robust Bayesian inference for multivariate longitudinal data by using normal/independent distributions. Statistics in Medicine, 32(22), 3812–3828.
Maier, K. S. (2001). A rasch hierarchical measurement model. Journal of Educational and Behavioral Statistics, 26(3), 307–330.
McRae, C., Diem, G., Vo, A., O’Brien, C., & Seeberger, Lauren. (2000). Schwab & England: Standardization of administration. Movement Disorders, 15(2), 335–336.
Miller, T. M., Balsis, S., Lowe, D. A., Benge, J. F., & Doody, R. S. (2012). Item response theory reveals variability of functional impairment within clinical dementia rating scale stages. Dementia and Geriatric Cognitive Disorders, 32(5), 362–366.
Müller, J., Wenning, G. K., Jellinger, K., McKee, A., Poewe, W., & Litvan, I. (2000). Progression of Hoehn and Yahr stages in Parkinsonian disorders: A clinicopathologic study. Neurology, 55(6), 888–891.
Parkinson Study Group. (1989). DATATOP: A multicenter controlled clinical trial in early Parkinson’s disease. Archives of Neurology, 46(10), 1052–1060.
Parkinson Study Group. (1993). Effects of tocopherol and deprenyl on the progression of disability in early Parkinson’s disease. The New England Journal of Medicine, 328(3), 176–183.
Parkinson Study Group. (2002). A controlled trial of rasagiline in early Parkinson disease: The TEMPO study. Archives of Neurology, 59(12), 1937.
Rijmen, F., Tuerlinckx, F., De Boeck, P., & Kuppens, P. (2003). A nonlinear mixed model framework for item response theory. Psychological methods, 8(2), 185.
Rosa, G. J. M., Padovani, C. R., & Gianola, D. (2003). Robust linear mixed models with normal/independent distributions and Bayesian MCMC implementation. Biometrical Journal, 45(5), 573–590.
Sahu, S. K., Dey, D. K., & Branco, M. D. (2003). A new class of multivariate skew distributions with applications to Bayesian regression models. Canadian Journal of Statistics, 31(2), 129–150.
Samejima, F. (1997). Graded response model. New York: Springer.
Snitz, B. E., Yu, L., Crane, P. K., Chang, C.-C. H., Hughes, T. F., & Ganguli, M.(2012). Subjective cognitive complaints of older adults at the population level: An item response theory analysis. Alzheimer Disease & Associated Disorders, 26(4), 344–351.
Spiegelhalter, D. J., Best, N. G., Carlin, B. P., & Van Der Linde, A. (2002). Bayesian measures of model complexity and fit. Journal of the Royal Statistical Society: Series B (Statistical Methodology), 64(4), 583–639.
Vaccarino, A. L., Anderson, K., Borowsky, B., Duff, K., Joseph, G., Mark, G., et al. (2011). An item response analysis of the motor and behavioral subscales of the unified Huntington’s disease rating scale in Huntington disease gene expansion carriers. Movement Disorders, 26(5), 877–884.
Wang, C., Douglas, J., & Anderson, S. (2002). Item response models for joint analysis of quality of life and survival. Statistics in Medicine, 21(1), 129–142.
Weisscher, N., Glas, C. A., Vermeulen, M., & De Haan, R. J. (2010). The use of an item response theory-based disability item bank across diseases: accounting for differential item functioning. Journal of Clinical Epidemiology, 63(5), 543–549.
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Chen, G., Luo, S. (2017). Robust Bayesian Hierarchical Model Using Monte-Carlo Simulation. In: Chen, DG., Chen, J. (eds) Monte-Carlo Simulation-Based Statistical Modeling . ICSA Book Series in Statistics. Springer, Singapore. https://doi.org/10.1007/978-981-10-3307-0_16
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