Chapter 10. Considerations for Statistical Analysis
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A Guide to Presenting Clinical Prediction Models for Use in the Clinical Setting Laura J Bonnett, Kym IE Snell, Gary S Collins, Richard D Riley
A guide to presenting clinical prediction models for use in the clinical setting Laura J Bonnett, Kym IE Snell, Gary S Collins, Richard D Riley Addresses & positions Laura J Bonnett – University of Liverpool (Tenure Track Fellow); [email protected]; Department of Biostatistics, Waterhouse Building, Block F, 1-5 Brownlow Street, University of Liverpool, Liverpool, L69 3GL; https://orcid.org/0000-0002-6981-9212 Kym IE Snell – Centre for Prognosis Research, Research Institute for Primary Care and Health Sciences, Keele University (Research Fellow in Biostatistics); [email protected]; https://orcid.org/0000-0001- 9373-6591 Gary S Collins – Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford (Professor of Medical Statistics); [email protected]; https://orcid.org/0000-0002-2772-2316 Richard D Riley – Centre for Prognosis Research, Research Institute for Primary Care and Health Sciences, Keele University (Professor of Biostatistics); [email protected]; https://orcid.org/0000-0001-8699-0735 Corresponding Author Correspondence to: [email protected] Standfirst Clinical prediction models estimate the risk of existing disease or future outcome for an individual, conditional on their values of multiple predictors such as age, sex and biomarkers. In this article, Bonnett and colleagues provide a guide to presenting clinical prediction models so that they can be implemented in practice, if appropriate. They describe how to create four presentation formats, and discuss the advantages and disadvantages of each. A key message is the need for stakeholder engagement to determine the best presentation option in relation to the clinical context of use and the intended user. -
Statistical Approaches for Highly Skewed Data 1
Running head: STATISTICAL APPROACHES FOR HIGHLY SKEWED DATA 1 (in press) Journal of Clinical Child and Adolescent Psychology Statistical Approaches for Highly Skewed Data: Evaluating Relations between Child Maltreatment and Young Adults’ Non-Suicidal Self-injury Ana Gonzalez-Blanks, PhD Jessie M. Bridgewater, MA *Tuppett M. Yates, PhD Department of Psychology University of California, Riverside Acknowledgments: We gratefully acknowledge Dr. Chandra Reynolds who provided generous instruction and consultation throughout the execution of the analyses reported herein. *Please address correspondence to: Tuppett M. Yates, Department of Psychology, University of California, Riverside CA, 92521. Email: [email protected]. STATISTICAL APPROACHES FOR HIGHLY SKEWED DATA 2 Abstract Objective: Clinical phenomena often feature skewed distributions with an overabundance of zeros. Unfortunately, empirical methods for dealing with this violation of distributional assumptions underlying regression are typically discussed in statistical journals with limited translation to applied researchers. Therefore, this investigation compared statistical approaches for addressing highly skewed data as applied to the evaluation of relations between child maltreatment and non-suicidal self-injury (NSSI). Method: College students (N = 2,651; 64.2% female; 85.2% non-White) completed the Child Abuse and Trauma Scale and the Functional Assessment of Self-Mutilation. Statistical models were applied to cross-sectional data to provide illustrative comparisons across predictions to a) raw, highly skewed NSSI outcomes, b) natural log, square-root, and inverse NSSI transformations to reduce skew, c) zero-inflated Poisson (ZIP) and negative-binomial zero- inflated (NBZI) regression models to account for both disproportionate zeros and skewness in the NSSI data, and d) the skew-t distribution to model NSSI skewness. -
Negative Binomial Regression Models and Estimation Methods
Appendix D: Negative Binomial Regression Models and Estimation Methods By Dominique Lord Texas A&M University Byung-Jung Park Korea Transport Institute This appendix presents the characteristics of Negative Binomial regression models and discusses their estimating methods. Probability Density and Likelihood Functions The properties of the negative binomial models with and without spatial intersection are described in the next two sections. Poisson-Gamma Model The Poisson-Gamma model has properties that are very similar to the Poisson model discussed in Appendix C, in which the dependent variable yi is modeled as a Poisson variable with a mean i where the model error is assumed to follow a Gamma distribution. As it names implies, the Poisson-Gamma is a mixture of two distributions and was first derived by Greenwood and Yule (1920). This mixture distribution was developed to account for over-dispersion that is commonly observed in discrete or count data (Lord et al., 2005). It became very popular because the conjugate distribution (same family of functions) has a closed form and leads to the negative binomial distribution. As discussed by Cook (2009), “the name of this distribution comes from applying the binomial theorem with a negative exponent.” There are two major parameterizations that have been proposed and they are known as the NB1 and NB2, the latter one being the most commonly known and utilized. NB2 is therefore described first. Other parameterizations exist, but are not discussed here (see Maher and Summersgill, 1996; Hilbe, 2007). NB2 Model Suppose that we have a series of random counts that follows the Poisson distribution: i e i gyii; (D-1) yi ! where yi is the observed number of counts for i 1, 2, n ; and i is the mean of the Poisson distribution. -
Measures of Fit for Logistic Regression Paul D
Paper 1485-2014 SAS Global Forum Measures of Fit for Logistic Regression Paul D. Allison, Statistical Horizons LLC and the University of Pennsylvania ABSTRACT One of the most common questions about logistic regression is “How do I know if my model fits the data?” There are many approaches to answering this question, but they generally fall into two categories: measures of predictive power (like R-square) and goodness of fit tests (like the Pearson chi-square). This presentation looks first at R-square measures, arguing that the optional R-squares reported by PROC LOGISTIC might not be optimal. Measures proposed by McFadden and Tjur appear to be more attractive. As for goodness of fit, the popular Hosmer and Lemeshow test is shown to have some serious problems. Several alternatives are considered. INTRODUCTION One of the most frequent questions I get about logistic regression is “How can I tell if my model fits the data?” Often the questioner is expressing a genuine interest in knowing whether a model is a good model or a not-so-good model. But a more common motivation is to convince someone else--a boss, an editor, or a regulator--that the model is OK. There are two very different approaches to answering this question. One is to get a statistic that measures how well you can predict the dependent variable based on the independent variables. I’ll refer to these kinds of statistics as measures of predictive power. Typically, they vary between 0 and 1, with 0 meaning no predictive power whatsoever and 1 meaning perfect predictions. -
Multiple Binomial Regression Models of Learning Style Preferences of Students of Sidhu School, Wilkes University
International Journal of Statistics and Probability; Vol. 7, No. 3; May 2018 ISSN 1927-7032 E-ISSN 1927-7040 Published by Canadian Center of Science and Education Multiple Binomial Regression Models of Learning Style Preferences of Students of Sidhu School, Wilkes University Adekanmbi, D. B1 1 Department of Statistics, Ladoke Akintola University of Technology, Nigeria Correspondence: Adekanmbi, D.B, Department of Statistics, Ladoke Akintola University of Technology, Ogbomoso, Nigeria. Received: November 6, 2017 Accepted: November 27, 2017 Online Published: March 13, 2018 doi:10.5539/ijsp.v7n3p9 URL: https://doi.org/10.5539/ijsp.v7n3p9 Abstract The interest of this study is to explore the relationship between a dichotomous response, learning style preferences by university students of Sidhu School, Wilkes University, as a function of the following predictors: Gender, Age, employment status, cumulative grade point assessment (GPA) and level of study, as in usual generalized linear model. The response variable is the students’ preference for either Behaviorist or Humanist learning style. Four different binomial regression models were fitted to the data. Model A is a logit regression model that fits all the predictors, Model B is a probit model that fits all the predictors, Model C is a logit model with an effect modifier, while Model D is a probit model also with an effect modifier. Models A and B appeared to have performed poorly in fitting the data. Models C and D fit the data well as confirmed by the non-significant chi-square lack of fit with p-values 0.1409 and 0.1408 respectively. Among the four models considered for fitting the data, Model D, the probit model with effect modifier fit best. -
Transforming Summary Statistics from Logistic Regression to the Liability Scale: Application To
bioRxiv preprint doi: https://doi.org/10.1101/385740; this version posted August 6, 2018. The copyright holder for this preprint (which was not certified by peer review) is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. It is made available under aCC-BY-NC-ND 4.0 International license. Transforming summary statistics from logistic regression to the liability scale: application to genetic and environmental risk scores Alexandra C. Gilletta, Evangelos Vassosa, Cathryn M. Lewisa, b* a. Social, Genetic & Developmental Psychiatry Centre, Institute of Psychiatry, Psychology & Neuroscience, King’s College London, London, UK b. Department of Medical and Molecular Genetics, Faculty of Life Sciences and Medicine, King’s College London, London, UK Short Title: Transforming the odds ratio to the liability scale *Corresponding Author Cathryn M. Lewis Institute of Psychiatry, Psychology & Neuroscience Social, Genetic & Developmental Psychiatry Centre King’s College London 16 De Crespigny Park Denmark Hill, London, SE5 8AF, UK Tel: +44(0)20 7848 0873 Fax: +44(0)20 7848 0866 E-mail: [email protected] Keywords Statistical genetics, risk, logistic regression, liability threshold model, schizophrenia Page 1 bioRxiv preprint doi: https://doi.org/10.1101/385740; this version posted August 6, 2018. The copyright holder for this preprint (which was not certified by peer review) is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. It is made available under aCC-BY-NC-ND 4.0 International license. 1. Abstract 1.1. Objective Stratified medicine requires models of disease risk incorporating genetic and environmental factors. -
Regression Models for Count Data Jason Brinkley, Abt Associates
SESUG Paper SD135-2018 Regression Models for Count Data Jason Brinkley, Abt Associates ABSTRACT Outcomes in the form of counts are becoming an increasingly popular metric in a wide variety of fields. For example, studying the number of hospital, emergency room, or in-patient doctor’s office visits has been a major focal point for many recent health studies. Many investigators want to know the impact of many different variables on these counts and help describe ways in which interventions or therapies might bring those numbers down. Traditional least squares regression was the primary mechanism for studying this type of data for decades. However, alternative methods were developed some time ago that are far superior for dealing with this type of data. The focus of this paper is to illustrate how count regression models can outperform traditional methods while utilizing the data in a more appropriate manner. Poisson Regression and Negative Binomial Regression are popular techniques when the data are overdispersed and using Zero-Inflated techniques for data with many more zeroes than is expected under traditional count regression models. These examples are applied to studies with real data. INTRODUCTION Outcomes in the form of counts are becoming an increasingly popular metric in a wide variety of fields. In the health sciences examples include number of hospitalizations, chronic conditions, medications, and so on. Many investigators want to know the impact of many different variables on these counts and help describe ways in which interventions or therapies might bring those numbers down. Standard methods (regression, t-tests, ANOVA) are useful for some count data studies. -
Generalized Linear Models and Point Count Data: Statistical Considerations for the Design and Analysis of Monitoring Studies
Generalized Linear Models and Point Count Data: Statistical Considerations for the Design and Analysis of Monitoring Studies Nathaniel E. Seavy,1,2,3 Suhel Quader,1,4 John D. Alexander,2 and C. John Ralph5 ________________________________________ Abstract The success of avian monitoring programs to effec- Key words: Generalized linear models, juniper remov- tively guide management decisions requires that stud- al, monitoring, overdispersion, point count, Poisson. ies be efficiently designed and data be properly ana- lyzed. A complicating factor is that point count surveys often generate data with non-normal distributional pro- perties. In this paper we review methods of dealing with deviations from normal assumptions, and we Introduction focus on the application of generalized linear models (GLMs). We also discuss problems associated with Measuring changes in bird abundance over time and in overdispersion (more variation than expected). In order response to habitat management is widely recognized to evaluate the statistical power of these models to as an important aspect of ecological monitoring detect differences in bird abundance, it is necessary for (Greenwood et al. 1993). The use of long-term avian biologists to identify the effect size they believe is monitoring programs (e.g., the Breeding Bird Survey) biologically significant in their system. We illustrate to identify population trends is a powerful tool for bird one solution to this challenge by discussing the design conservation (Sauer and Droege 1990, Sauer and Link of a monitoring program intended to detect changes in 2002). Short-term studies comparing bird abundance in bird abundance as a result of Western juniper (Juniper- treated and untreated areas are also important because us occidentalis) reduction projects in central Oregon. -
1. INTRODUCTION the Use of Clinical and Laboratory Data to Detect
1. INTRODUCTION The use of clinical and laboratory data to detect conditions and predict patient outcomes is a mainstay of medical practice. Classification and prediction are equally important in other fields of course (e.g., meteorology, economics, computer science) and have been subjects of statistical research for a long time. The field is currently receiving more attention in medicine, in part because of biotechnologic advancements that promise accurate non-invasive modes of testing. Technologies include gene expression arrays, protein mass spectrometry and new imaging modalities. These can be used for purposes such as detecting subclinical disease, evaluating the prognosis of patients with disease and predicting their responses to different choices of therapy. Statistical methods have been developed for assessing the accuracy of classifiers in medicine (Zhou, Obuchowski, and McClish 2002; Pepe 2003), although this is an area of statistics that is evolving rapidly. In practice there may be multiple sources of information available to assist in prediction. For example, clinical signs and symptoms of disease may be supplemented by results of laboratory tests. As another example, it is expected that multiple biomarkers will be needed for detecting subclinical cancer with adequate sensitivity and specificity (Pepe et al. 2001). The starting point for the research we describe in this paper is the need to combine multiple predictors together, somehow, in order to predict outcome. We note in Section 2 that for a binary outcome, D, the best classifiers using the predictors Y =(Y1,Y2,...,YP ) are based on the risk score function RS(Y )=P (D =1|Y ) (1) or equivalently any monotone increasing transformation of it. -
Support Vector Hazards Machine: a Counting Process Framework for Learning Risk Scores for Censored Outcomes
Journal of Machine Learning Research 17 (2016) 1-37 Submitted 1/16; Revised 7/16; Published 8/16 Support Vector Hazards Machine: A Counting Process Framework for Learning Risk Scores for Censored Outcomes Yuanjia Wang [email protected] Department of Biostatistics Mailman School of Public Health Columbia University New York, NY 10032, USA Tianle Chen [email protected] Biogen 300 Binney Street Cambridge, MA 02142, USA Donglin Zeng [email protected] Department of Biostatistics University of North Carolina at Chapel Hill Chapel Hill, NC 27599, USA Editor: Karsten Borgwardt Abstract Learning risk scores to predict dichotomous or continuous outcomes using machine learning approaches has been studied extensively. However, how to learn risk scores for time-to-event outcomes subject to right censoring has received little attention until recently. Existing approaches rely on inverse probability weighting or rank-based regression, which may be inefficient. In this paper, we develop a new support vector hazards machine (SVHM) approach to predict censored outcomes. Our method is based on predicting the counting process associated with the time-to-event outcomes among subjects at risk via a series of support vector machines. Introducing counting processes to represent time-to-event data leads to a connection between support vector machines in supervised learning and hazards regression in standard survival analysis. To account for different at risk populations at observed event times, a time-varying offset is used in estimating risk scores. The resulting optimization is a convex quadratic programming problem that can easily incorporate non- linearity using kernel trick. We demonstrate an interesting link from the profiled empirical risk function of SVHM to the Cox partial likelihood. -
Lecture 3 Residual Analysis + Generalized Linear Models
Lecture 3 Residual Analysis + Generalized Linear Models Colin Rundel 1/23/2017 1 Residual Analysis 2 Atmospheric CO2 (ppm) from Mauna Loa 360 co2 350 1988 1992 1996 date 3 360 co2 350 1988 1992 1996 date 2.5 0.0 resid −2.5 1988 1992 1996 date Where to start? Well, it looks like stuff is going up on average … 4 360 co2 350 1988 1992 1996 date 2.5 0.0 resid −2.5 1988 1992 1996 date Where to start? Well, it looks like stuff is going up on average … 4 2.5 0.0 resid −2.5 1988 1992 1996 date 1.0 0.5 0.0 resid2 −0.5 −1.0 1988 1992 1996 date and then? Well there is some periodicity lets add the month … 5 2.5 0.0 resid −2.5 1988 1992 1996 date 1.0 0.5 0.0 resid2 −0.5 −1.0 1988 1992 1996 date and then? Well there is some periodicity lets add the month … 5 1.0 0.5 0.0 resid2 −0.5 −1.0 1988 1992 1996 date 0.8 0.4 0.0 resid3 −0.4 −0.8 1988 1992 1996 date and then and then? Maybe there is some different effect by year … 6 1.0 0.5 0.0 resid2 −0.5 −1.0 1988 1992 1996 date 0.8 0.4 0.0 resid3 −0.4 −0.8 1988 1992 1996 date and then and then? Maybe there is some different effect by year … 6 Too much (lm = lm(co2~date + month + as.factor(year), data=co2_df)) ## ## Call: ## lm(formula = co2 ~ date + month + as.factor(year), data = co2_df) ## ## Coefficients: ## (Intercept) date monthAug ## -2.645e+03 1.508e+00 -4.177e+00 ## monthDec monthFeb monthJan ## -3.612e+00 -2.008e+00 -2.705e+00 ## monthJul monthJun monthMar ## -2.035e+00 -3.251e-01 -1.227e+00 ## monthMay monthNov monthOct ## 4.821e-01 -4.838e+00 -6.135e+00 ## monthSep as.factor(year)1986 as.factor(year)1987 ## -6.064e+00 -2.585e-01 9.722e-03 ## as.factor(year)1988 as.factor(year)1989 as.factor(year)1990 ## 1.065e+00 9.978e-01 7.726e-01 ## as.factor(year)1991 as.factor(year)1992 as.factor(year)1993 ## 7.067e-01 1.236e-02 -7.911e-01 ## as.factor(year)1994 as.factor(year)1995 as.factor(year)1996 ## -4.146e-01 1.119e-01 3.768e-01 ## as.factor(year)1997 7 ## NA 360 co2 350 1988 1992 1996 date 8 Generalized Linear Models 9 Background A generalized linear model has three key components: 1. -
The Overlooked Potential of Generalized Linear Models in Astronomy, I: Binomial Regression
Astronomy and Computing 12 (2015) 21–32 Contents lists available at ScienceDirect Astronomy and Computing journal homepage: www.elsevier.com/locate/ascom Full length article The overlooked potential of Generalized Linear Models in astronomy, I: Binomial regression R.S. de Souza a,∗, E. Cameron b, M. Killedar c, J. Hilbe d,e, R. Vilalta f, U. Maio g,h, V. Biffi i, B. Ciardi j, J.D. Riggs k, for the COIN collaboration a MTA Eötvös University, EIRSA ``Lendulet'' Astrophysics Research Group, Budapest 1117, Hungary b Department of Zoology, University of Oxford, Tinbergen Building, South Parks Road, Oxford, OX1 3PS, United Kingdom c Universitäts-Sternwarte München, Scheinerstrasse 1, D-81679, München, Germany d Arizona State University, 873701, Tempe, AZ 85287-3701, USA e Jet Propulsion Laboratory, 4800 Oak Grove Dr., Pasadena, CA 91109, USA f Department of Computer Science, University of Houston, 4800 Calhoun Rd., Houston TX 77204-3010, USA g INAF — Osservatorio Astronomico di Trieste, via G. Tiepolo 11, 34135 Trieste, Italy h Leibniz Institute for Astrophysics, An der Sternwarte 16, 14482 Potsdam, Germany i SISSA — Scuola Internazionale Superiore di Studi Avanzati, Via Bonomea 265, 34136 Trieste, Italy j Max-Planck-Institut für Astrophysik, Karl-Schwarzschild-Str. 1, D-85748 Garching, Germany k Northwestern University, Evanston, IL, 60208, USA article info a b s t r a c t Article history: Revealing hidden patterns in astronomical data is often the path to fundamental scientific breakthroughs; Received 26 September 2014 meanwhile the complexity of scientific enquiry increases as more subtle relationships are sought. Con- Accepted 2 April 2015 temporary data analysis problems often elude the capabilities of classical statistical techniques, suggest- Available online 29 April 2015 ing the use of cutting edge statistical methods.