Blinded continuous monitoring in clinical trials with recurrent event endpoints

In studies with recurrent event endpoints, misspecified assumptions of event rates or dispersion can lead to underpowered trials or overexposure of patients. Specification of overdispersion is often a particular problem as it is usually not reported in clinical trial publications. Changing event rates over the years have been described for some diseases, adding to the uncertainty in planning. To mitigate the risks of inadequate sample sizes, internal pilot study designs have been proposed with a preference for blinded sample size reestimation procedures, as they generally do not affect the type I error rate and maintain trial integrity. Blinded sample size reestimation procedures are available for trials with recurrent events as endpoints. However, the variance in the reestimated sample size can be considerable in particular with early sample size reviews. Motivated by a randomized controlled trial in paediatric multiple sclerosis, a rare neurological condition in children, we apply the concept of blinded continuous monitoring of information, which is known to reduce the variance in the resulting sample size. Assuming negative binomial distributions for the counts of recurrent relapses, we derive information criteria and propose blinded continuous monitoring procedures. The operating characteristics of these are assessed in Monte Carlo trial simulations demonstrating favourable properties with regard to type I error rate, power, and stopping time, ie, sample size.     

Estimating household structure in ancient China by using historical data: a latent class analysis of partially missing patterns

Summary.  Social data often contain missing information. The problem is inevitably severe when analysing historical data. Conventionally, researchers analyse complete records only. Listwise deletion not only reduces the effective sample size but also may result in biased estimation, depending on the missingness mechanism. We analyse household types by using population registers from ancient China (618–907 AD) by comparing a simple classification, a latent class model of the complete data and a latent class model of the complete and partially missing data assuming four types of ignorable and non-ignorable missingness mechanisms. The findings show that either a frequency classification or a latent class analysis using the complete records only yielded biased estimates and incorrect conclusions in the presence of partially missing data of a non-ignorable mechanism. Although simply assuming ignorable or non-ignorable missing data produced consistently similarly higher estimates of the proportion of complex households, a specification of the relationship between the latent variable and the degree of missingness by a row effect uniform association model helped to capture the missingness mechanism better and improved the model fit.     

Quasi-maximum likelihood estimation and inference in dynamic models with time-varying covariances

We study the properties of the quasi-maximum likelihood estimator (QMLE) and related test statistics in dynamic models that jointly parameterize conditional means and conditional covariances, when a normal log-likelihood os maximized but the assumption of normality is violated. Because the score of the normal log-likelihood has the martingale difference property when the forst two conditional moments are correctly specified, the QMLE is generally Consistent and has a limiting normal destribution. We provide easily computable formulas for asymptotic standard errors that are valid under nonnormality. Further, we show how robust LM tests for the adequacy of the jointly parameterized mean and variance can be computed from simple auxiliary regressions. An appealing feature of these robyst inference procedures is that only first derivatives of the conditional mean and variance functions are needed. A monte Carlo study indicates that the asymptotic results carry over to finite samples. Estimation of several AR and AR-GARCH time series models reveals that in most sotuations the robust test statistics compare favorably to the two standard (nonrobust) formulations of the Wald and IM tests. Also, for the GARCH models and the sample sizes analyzed here, the bias in the QMLE appears to be relatively small. An empirical application to stock return volatility illustrates the potential imprtance of computing robust statistics in practice.     

Rural/Urban Differences in Child Growth and Survival in Bolivia

Abstract Using data from a national survey in Bolivia and from a regional sample of poor communities in two regions of Bolivia, we model the effects of community conditions, household characteristics, and social relations on child health as measured by child mortality and stunted growth. Based on national data, children in rural areas are twice as likely to die before the age of two than are children in large cities, and about one‐third of the children in rural areas are stunted. Including measures of community, household, and maternal characteristics accounts for at least two‐thirds of the urban/rural difference in child health. We conclude that expansion of health services, improvements in household and community sanitation, and socioeco‐nomic development are all needed to overcome the rural disadvantage in child health.     

Bayesian analysis of directed graphs data with applications to social networks

Summary.  A fully Bayesian analysis of directed graphs, with particular emphasis on applica- tions in social networks, is explored. The model is capable of incorporating the effects of covariates, within and between block ties and multiple responses. Inference is straightforward by using software that is based on Markov chain Monte Carlo methods. Examples are provided which highlight the variety of data sets that can be entertained and the ease with which they can be analysed.     

The Access/Impact Problem and the Green and Gold Roads to Open Access

The research access/impact problem arises because journal articles are not accessible to all of their would-be users; hence, they are losing potential research impact. The solution is to make all articles Open Access (OA; i.e., accessible online, free for all). OA articles have significantly higher citation impact than non-OA articles. There are two roads to OA: the “golden” road (publish your article in an OA journal) and the “green” road (publish your article in a non-OA journal but also self-archive it in an OA archive). Only 5% of journals are gold, but over 90% are already green (i.e., they have given their authors the green light to self-archive); yet only about 10–20% of articles have been self-archived. To reach 100% OA, self-archiving needs to be mandated by researchers' employers and funders, as the United Kingdom and the United States have recently recommended, and universities need to implement that mandate.     

Ecocultural Attributes: Evaluating Ecological Degradation in Terms of Ecological Goods and Services Versus Subsistence and Tribal Values

It is becoming increasingly clear that scientists, managers, lawyers, public policymakers, and the public must decide how to value what is provided by, and is a consequence of, natural resources. While “Western” scientists have clear definitions for the goods and services that ecosystems provide, we contend that these categories do not encompass the full totality of the values provided by natural resources. Partly the confusion results from a limited view of natural resources derived from the need to monetize the value of ecosystems and their component parts. Partly it derives from the “Western” way of separating natural resources from cultural resources or values, and partly it derives from the false dichotomy of assuming that ecosystems are natural, and anything constructed by man is not natural. In this article, we explore the previous assumptions, and suggest that because cultural resources often derive from, and indeed require, intact and unspoiled natural ecosystems or settings, that these values are rightly part of natural resources. The distinction is not trivial because of the current emphasis on cleaning up chemically and radiologically contaminated sites, on restoration of damaged ecosystems, on natural resource damage assessments, and on long‐term stewardship goals. All of these processes depend upon defining natural resources appropriately. Several laws, regulations, and protocols depend upon natural resource trustees to protect natural resources on trust lands, which could lead to the circular definition that natural resources are those resources that the trustees feel they are responsible for. Where subsistence or tribal peoples are involved, the definition of natural resources should be broadened to include those ecocultural attributes that are dependent upon, and have incorporated, natural resources. For example, a traditional hunting and fishing ground is less valued by subsistence peoples if it is despoiled by contamination or physical ecosystem degradation; an Indian sacred ground is tarnished if the surrounding natural environment is degraded; a traditional homeland is less valued if the land itself is contaminated. Our argument is that intact natural resources are essential elements of many cultural resources, and this aspect requires and demands adequate consideration (and may therefore require compensation).     

A circular-cone test for testing homogeneity against a simple tree order

Several methods exist for the problem of testing the equality of several treatments against the one-sided alternative that the treatments are better than the control. These methods include Dunnett's test, Bartholomew's likelihood-ratio test, the Abelson-Tukey-Schaafsma-Smid optimal-contrast test, and the multiple-contrast test of Mukerjee, Robertson, and Wright. A new test is proposed based on an approximation of the likelihood-ratio test of Bartholomew. This test involves using a circular cone in place of the alternative-hypothesis cone. The circular-cone test has excellent power characteristics similar to those of Bartholomew's test. Moreover, it has the advantages of being simpler to compute and may be used with unequal sample sizes.     

A sea change on the island continent: frameworks for risk assessment,prevention and intervention in child health in Australia

This article overviews prevention and early intervention approaches focusing specifically on their relevance to the health of children and young people in Australia. Australian public health has a sound track record although concealed within the aggregate profile are a number of sub-populations with poorer health indicators. Recognition of this has increased efforts to improve the health of children and young people especially in exploring the impact of social environments within the communities where children are raised. This paper examines emerging research in this area drawing out key lessons and learning from Australian experience in the field of early intervention and prevention in community settings.