A new family of quantile regression models applied to nutritional data.

This paper introduces a new family of quantile regression models whose response variable follows a reparameterized Marshall-Olkin distribution indexed by quantile, scale, and asymmetry parameters. The family has arisen by applying the Marshall-Olkin approach to distributions belonging to the location-scale family. Models of higher flexibility and whose structure is similar to generalized linear models were generated by quantile reparameterization. The maximum likelihood (ML) method is presented for the estimation of the model parameters, and simulation studies evaluated the performance of the ML estimators. The advantages of the family are illustrated through an application to a set of nutritional data, whose results indicate it is a good alternative for modeling slightly asymmetric response variables with support on the real line.

Improved inference for MCP-Mod approach using time-to-event endpoints with small sample sizes.

The Multiple Comparison Procedures with Modeling Techniques (MCP-Mod) framework has been recently approved by the U.S. Food, Administration, and European Medicines Agency as fit-for-purpose for phase II studies. Nonetheless, this approach relies on the asymptotic properties of Maximum Likelihood (ML) estimators, which might not be reasonable for small sample sizes. In this paper, we derived improved ML estimators and correction for their covariance matrices in the censored Weibull regression model based on the corrective and preventive approaches. We performed two simulation studies to evaluate ML and improved ML estimators with their covariance matrices in (i) a regression framework (ii) the Multiple Comparison Procedures with Modeling Techniques framework. We have shown that improved ML estimators are less biased than ML estimators yielding Wald-type statistics that controls type I error without loss of power in both frameworks. Therefore, we recommend the use of improved ML estimators in the MCP-Mod approach to control type I error at nominal value for sample sizes ranging from 5 to 25 subjects per dose.

A general class of promotion time cure rate models with a new biological interpretation.

Over the last decades, the challenges in survival models have been changing considerably and full probabilistic modeling is crucial in many medical applications. Motivated from a new biological interpretation of cancer metastasis, we introduce a general method for obtaining more flexible cure rate models. The proposal model extended the promotion time cure rate model. Furthermore, it includes several well-known models as special cases and defines many new special models. We derive several properties of the hazard function for the proposed model and establish mathematical relationships with the promotion time cure rate model. We consider a frequentist approach to perform inferences, and the maximum likelihood method is employed to estimate the model parameters. Simulation studies are conducted to evaluate its performance with a discussion of the obtained results. A real dataset from population-based study of incident cases of melanoma diagnosed in the state of São Paulo, Brazil, is discussed in detail.

On a new piecewise regression model with cure rate: Diagnostics and application to medical data.

In this article, we discuss an extension of the classical negative binomial cure rate model with piecewise exponential distribution of the time to event for concurrent causes, which enables the modeling of monotonic and non-monotonic hazard functions (ie, the shape of the hazard function is not assumed as in traditional parametric models). This approach produces a flexible cure rate model, depending on the choice of time partition. We discuss local influence on this negative binomial power piecewise exponential model. We report on Monte Carlo simulation studies and application of the model to real melanoma and leukemia datasets.

Personalized Diet in Obesity: A Quasi-Experimental Study on Fat Mass and Fat-Free Mass Changes.

Considering that the prevalence of overweight and obesity in Southeast of Spain is high, the aim of this work was to analyze the relation between the adherence to a personalized diet and the effectiveness of changes in the body composition in overweight and obese adults in this region. This quasi-experimental study presents the following selection criteria: attendance at the consultation between 2006 and 2012, subjects ≥ 19 years of age with overweight or obesity. In total, 591 overweight or obese individuals were involved in this study, attending 4091 clinic consultations in total. Most of the sample consisted of subjects who attended >3 consultations (>1.5 months), and were females aged 19-64 years who obtained clinically significant changes in fat mass (FM, ≥5%) and recommended changes in fat-free mass (FFM, ≥0%). Based on the results obtained and the experience gained from this research, the following recommendations are established: (i) record fat mass and fat-free mass index as a complement to body mass index; (ii) use FM and FFM to evaluate effectiveness of interventions with the aim of obtaining body composition changes; (iii) use personalized diet to achieve significant changes in FM and avoid non-recommended changes in FFM.

A new cure rate model with flexible competing causes with applications to melanoma and transplantation data.

In this article, we introduce a long-term survival model in which the number of competing causes of the event of interest follows the zero-modified geometric (ZMG) distribution. Such distribution accommodates equidispersion, underdispersion, and overdispersion and captures deflation or inflation of zeros in the number of lesions or initiated cells after the treatment. The ZMG distribution is also an appropriate alternative for modeling clustered samples when the number of competing causes of the event of interest consists of two subpopulations, one containing only zeros (cure proportion), while in the other (noncure proportion) the number of competing causes of the event of interest follows a geometric distribution. The advantage of this assumption is that we can measure the cure proportion in the initiated cells. Furthermore, the proposed model can yield greater or lower cure proportion than that of the geometric distribution when modeling the number of competing causes. In this article, we present some statistical properties of the proposed model and use the maximum likelihood method to estimate the model parameters. We also conduct a Monte Carlo simulation study to evaluate the performance of the estimators. We present and discuss two applications using real-world medical data to assess the practical usefulness of the proposed model.

On the use of the modified power series family of distributions in a cure rate model context.

In this paper, we propose a generalization of the power series cure rate model for the number of competing causes related to the occurrence of the event of interest. The model includes distributions not yet used in the cure rate models context, such as the Borel, Haight and Restricted Generalized Poisson distributions. The model is conveniently parameterized in terms of the cure rate. Maximum likelihood estimation based on the Expectation Maximization algorithm is discussed. A simulation study designed to assess some properties of the estimators is carried out, showing the good performance of the proposed estimation procedure in finite samples. Finally, an application to a bone marrow transplant data set is presented.

Parametric modal regression with varying precision.

In this paper, we propose a simple parametric modal linear regression model where the response variable is gamma distributed using a new parameterization of this distribution that is indexed by mode and precision parameters, that is, in this new regression model, the modal and precision responses are related to a linear predictor through a link function and the linear predictor involves covariates and unknown regression parameters. The main advantage of our new parameterization is the straightforward interpretation of the regression coefficients in terms of the mode of the positive response variable, as is usual in the context of generalized linear models, and direct inference in parametric mode regression based on the likelihood paradigm. Furthermore, we discuss residuals and influence diagnostic tools. A Monte Carlo experiment is conducted to evaluate the performances of these estimators in finite samples with a discussion of the results. Finally, we illustrate the usefulness of the new model by two applications, to biology and demography.

Truncated Power-Normal Distribution with Application to Non-Negative Measurements.

This paper focuses on studying a truncated positive version of the power-normal (PN) model considered in Durrans (1992). The truncation point is considered to be zero so that the resulting model is an extension of the half normal distribution. Some probabilistic properties are studied for the proposed model along with maximum likelihood and moments estimation. The model is fitted to two real datasets and compared with alternative models for positive data. Results indicate good performance of the proposed model.

Bayesian bivariate survival analysis using the power variance function copula.

Copula models have become increasingly popular for modelling the dependence structure in multivariate survival data. The two-parameter Archimedean family of Power Variance Function (PVF) copulas includes the Clayton, Positive Stable (Gumbel) and Inverse Gaussian copulas as special or limiting cases, thus offers a unified approach to fitting these important copulas. Two-stage frequentist procedures for estimating the marginal distributions and the PVF copula have been suggested by Andersen (Lifetime Data Anal 11:333-350, 2005), Massonnet et al. (J Stat Plann Inference 139(11):3865-3877, 2009) and Prenen et al. (J R Stat Soc Ser B 79(2):483-505, 2017) which first estimate the marginal distributions and conditional on these in a second step to estimate the PVF copula parameters. Here we explore an one-stage Bayesian approach that simultaneously estimates the marginal and the PVF copula parameters. For the marginal distributions, we consider both parametric as well as semiparametric models. We propose a new method to simulate uniform pairs with PVF dependence structure based on conditional sampling for copulas and on numerical approximation to solve a target equation. In a simulation study, small sample properties of the Bayesian estimators are explored. We illustrate the usefulness of the methodology using data on times to appendectomy for adult twins in the Australian NH&MRC Twin registry. Parameters of the marginal distributions and the PVF copula are simultaneously estimated in a parametric as well as a semiparametric approach where the marginal distributions are modelled using Weibull and piecewise exponential distributions, respectively.