A simple two-stage design for quantitative responses with application to a study in diabetic neuropathic pain

Two-stage designs offer substantial advantages for early phase II studies. The interim analysis following the first stage allows the study to be stopped for futility, or more positively, it might lead to early progression to the trials needed for late phase II and phase III. If the study is to continue to its second stage, then there is an opportunity for a revision of the total sample size. Two-stage designs have been implemented widely in oncology studies in which there is a single treatment arm and patient responses are binary. In this paper the case of two-arm comparative studies in which responses are quantitative is considered. This setting is common in therapeutic areas other than oncology. It will be assumed that observations are normally distributed, but that there is some doubt concerning their standard deviation, motivating the need for sample size review. The work reported has been motivated by a study in diabetic neuropathic pain, and the development of the design for that trial is described in detail.     

The generalized log-gamma mixture model with covariates: local influence and residual analysis

In a sample of censored survival times, the presence of an immune proportion of individuals who are not subject to death, failure or relapse, may be indicated by a relatively high number of individuals with large censored survival times. In this paper the generalized log-gamma model is modified for the possibility that long-term survivors may be present in the data. The model attempts to separately estimate the effects of covariates on the surviving fraction, that is, the proportion of the population for which the event never occurs. The logistic function is used for the regression model of the surviving fraction. Inference for the model parameters is considered via maximum likelihood. Some influence methods, such as the local influence and total local influence of an individual are derived, analyzed and discussed. Finally, a data set from the medical area is analyzed under the log-gamma generalized mixture model. A residual analysis is performed in order to select an appropriate model. The authors would like to thank the editor and referees for their helpful comments. This work was supported by CNPq, Brazil.     

A copula-based Markov chain model for the analysis of binary longitudinal data

A fully parametric first-order autoregressive (AR(1)) model is proposed to analyse binary longitudinal data. By using a discretized version of a copula, the modelling approach allows one to construct separate models for the marginal response and for the dependence between adjacent responses. In particular, the transition model that is focused on discretizes the Gaussian copula in such a way that the marginal is a Bernoulli distribution. A probit link is used to take into account concomitant information in the behaviour of the underlying marginal distribution. Fixed and time-varying covariates can be included in the model. The method is simple and is a natural extension of the AR(1) model for Gaussian series. Since the approach put forward is likelihood-based, it allows interpretations and inferences to be made that are not possible with semi-parametric approaches such as those based on generalized estimating equations. Data from a study designed to reduce the exposure of children to the sun are used to illustrate the methods.     

Bayesian robustness of the compound Poisson distribution under bidimensional prior: an application to the collective risk model

The distribution of the aggregate claims in one year plays an important role in Actuarial Statistics for computing, for example, insurance premiums when both the number and size of the claims must be implemented into the model. When the number of claims follows a Poisson distribution the aggregated distribution is called the compound Poisson distribution. In this article we assume that the claim size follows an exponential distribution and later we make an extensive study of this model by assuming a bidimensional prior distribution for the parameters of the Poisson and exponential distribution with marginal gamma. This study carries us to obtain expressions for net premiums, marginal and posterior distributions in terms of some well-known special functions used in statistics. Later, a Bayesian robustness study of this model is made. Bayesian robustness on bidimensional models was deeply treated in the 1990s, producing numerous results, but few applications dealing with this problem can be found in the literature.     

Activism among older women in Quebec, Canada: changing the world after age 65

The aim of this article is to examine senior women's involvement experience in Quebec, Canada. Study results are based on a qualitative methodology, and shed light on family history and continuity in the involvement trajectory, diversity in terms of group type and involvement practices, and gender differences in involvement. The meaning that older women attribute to their involvement is also addressed, including attitudes toward feminism. The discussion highlights the extent to which older women's involvement has been marked by their life trajectories, above and beyond their age. In sum, their involvement in the private and public spheres is quite impressive.     

Evaluating school and parent reports of the national student achievement testing system (SIMCE) in Chile: Access,comprehension, and use

This evaluation examined school and parent reports of the national student achievement testing system (SIMCE) in Chile regarding three dimensions: access, comprehension, and use. We conducted phone surveys with a representative sample of directors (N = 375), teachers (N = 1145) and parents (N = 625), and we collected more in-depth data through interviews and focus groups in 16 of these schools. The results indicate that access to the reports is not an obstacle to use for school actors, but it is for parents. While summative ratings of the reports in terms of their clarity and utility were generally very positive, the actual recall and interpretation of even basic information (assessed through case scenarios) was incorrect for a majority of teachers and parents, and reported uses of the information were both intended and unintended in nature. We also found some statistically significant differences regarding comprehension and use among subgroups of our sample. Our findings are especially relevant given the expectations attached to the use of the reports for school improvement on the one hand, and parents’ behavior as critical consumers of education on the other hand.     

Children’s Strategies for Agency in Preschool

This article is based on an ethnographic study of children’s everyday life in Swedish preschools. The ethnography is used to explore children’s strategies for influencing, defending and constructing the social order of a preschool institution. The focus of our concern is on how the children, in their interactions with each other and with the preschool teachers, manage the collective regulation and how they negotiate their participation in collective activities. There is an inherent tension between free play and the high degree of routinised and collective activities within the preschool institution. The study shows that children are active in playing at the border, acting as if the institution is the children’s place. It also shows how they draw on different strategies as resources for managing the regulations, accounting for personal autonomy and negotiating the social order. In taking a child perspective and acknowledging children as active agents, it is possible to see how they influence and shape their everyday life in a preschool context. In addition, the article illustrates individual children’s strategic and pragmatic use of resources and in doing so contribute to their own childhood and thereby become part of a social and cultural construction process.     

Number of children and later-life mortality among Finns born 1938–50

We investigated the association between number of offspring and later-life mortality of Finnish men and women born 1938–50, and whether the association was explained by living conditions in own childhood and adulthood, chronic conditions, fertility timing, and unobserved characteristics common to siblings. We used a longitudinal 1950 census sample to estimate mortality at ages 50–72. Relative to parents of two children, all-cause mortality is highest among childless men and women, and elevated among those with one child, independently of observed confounders. Fixed-effect models, which control for unobserved characteristics shared by siblings, clearly support these findings among men. Cardiovascular mortality is higher among men with no, one, or at least four children than among those with two. Living conditions in adulthood contribute to the association between the number of children and mortality to a greater extent than childhood background, and chronic conditions contribute to the excess mortality of the childless.     

Measuring disparities in access to water based on the normative content of the human right

Measuring access to water in the Sustainable Development Goals era involves taking into account the human rights framework. Therefore, its content should be considered to conceptualize the level of service through adequate indicators and to follow-up inequities reduction at global, national and local level. This research develops and tests a methodology to measure intra-community disparities based on human right to water normative criteria through a stratified sampling, splitting households served by community based organizations and those self-provided. This approach implies considering much reduced populations, thus special care needs to be taken with sample sizes and uncertainty of estimators. The proposed methodology is practical to locate and accurately characterize minority sectors within rural communities and allows moving beyond central-tendency estimators. It implies higher costs for field data collection than traditional approaches, but this can be assumed given the relevance of the approach from a human rights perspective, which calls for adequate tools for equity-oriented policy making at local level. The research point out how results might be used to shape decision-making processes.