A score test for binary data with patient non-compliance

A score test is developed for binary clinical trial data, which incorporates patient non-compliance while respecting randomization. It is assumed in this paper that compliance is all-or-nothing, in the sense that a patient either accepts all of the treatment assigned as specified in the protocol, or none of it. Direct analytic comparisons of the adjusted test statistic for both the score test and the likelihood ratio test are made with the corresponding test statistics that adhere to the intention-to-treat principle. It is shown that no gain in power is possible over the intention-to-treat analysis, by adjusting for patient non-compliance. Sample size formulae are derived and simulation studies are used to demonstrate that the sample size approximation holds. Copyright © 2003 John Wiley & Sons, Ltd.

Sparse kernel density construction using orthogonal forward regression with leave-one-out test score and local regularization

This paper presents an efficient construction algorithm for obtaining sparse kernel density estimates based on a regression approach that directly optimizes model generalization capability. Computational efficiency of the density construction is ensured using an orthogonal forward regression, and the algorithm incrementally minimizes the leave-one-out test score. A local regularization method is incorporated naturally into the density construction process to further enforce sparsity. An additional advantage of the proposed algorithm is that it is fully automatic and the user is not required to specify any criterion to terminate the density construction procedure. This is in contrast to an existing state-of-art kernel density estimation method using the support vector machine (SVM), where the user is required to specify some critical algorithm parameter. Several examples are included to demonstrate the ability of the proposed algorithm to effectively construct a very sparse kernel density estimate with comparable accuracy to that of the full sample optimized Parzen window density estimate. Our experimental results also demonstrate that the proposed algorithm compares favorably with the SVM method, in terms of both test accuracy and sparsity, for constructing kernel density estimates.

A combined score test for binary and ordinal endpoints from clinical trials

There is growing interest, especially for trials in stroke, in combining multiple endpoints in a single clinical evaluation of an experimental treatment. The endpoints might be repeated evaluations of the same characteristic or alternative measures of progress on different scales. Often they will be binary or ordinal, and those are the cases studied here. In this paper we take a direct approach to combining the univariate score statistics for comparing treatments with respect to each endpoint. The correlations between the score statistics are derived and used to allow a valid combined score test to be applied. A sample size formula is deduced and application in sequential designs is discussed. The method is compared with an alternative approach based on generalized estimating equations in an illustrative analysis and replicated simulations, and the advantages and disadvantages of the two approaches are discussed.

Understanding of auditory discourse in older adults: the effects of syntax and working memory

Background: As people age, language-processing ability changes. While several factors modify discourse comprehension ability in older adults, syntactic complexity of auditory discourse has received scant attention. This is despite the widely researched domain of syntactic processing of single sentences in older adults. Aims: The aims of this study were to investigate the ability of healthy older adults to understand stories that differed in syntactic complexity, and its relation to working memory. Methods & Procedures: A total of 51 healthy adults (divided into three age groups) took part. They listened to brief stories (syntactically simple and syntactically complex) and had to respond to false/true comprehension probes following each story. Working memory capacity (digit span, forward and backward) was also measured. Outcomes & Results: Differences were found in the ability of healthy older adults to understand simple and complex discourse. The complex discourse in particular was more sensitive in discerning age-related language patterns. Only the complex discourse task correlated moderately with age. There was no correlation between age and simple discourse. As far as working memory is concerned, moderate correlations were found between working memory and complex discourse. Education did not correlate with discourse, neither simple, nor complex. Conclusions: Older adults may be less efficient in forming syntactically complex representations and this may be influenced by limitations in working memory.

Equitability revisited: why the “equitable threat score” is not equitable

In the forecasting of binary events, verification measures that are “equitable” were defined by Gandin and Murphy to satisfy two requirements: 1) they award all random forecasting systems, including those that always issue the same forecast, the same expected score (typically zero), and 2) they are expressible as the linear weighted sum of the elements of the contingency table, where the weights are independent of the entries in the table, apart from the base rate. The authors demonstrate that the widely used “equitable threat score” (ETS), as well as numerous others, satisfies neither of these requirements and only satisfies the first requirement in the limit of an infinite sample size. Such measures are referred to as “asymptotically equitable.” In the case of ETS, the expected score of a random forecasting system is always positive and only falls below 0.01 when the number of samples is greater than around 30. Two other asymptotically equitable measures are the odds ratio skill score and the symmetric extreme dependency score, which are more strongly inequitable than ETS, particularly for rare events; for example, when the base rate is 2% and the sample size is 1000, random but unbiased forecasting systems yield an expected score of around −0.5, reducing in magnitude to −0.01 or smaller only for sample sizes exceeding 25 000. This presents a problem since these nonlinear measures have other desirable properties, in particular being reliable indicators of skill for rare events (provided that the sample size is large enough). A potential way to reconcile these properties with equitability is to recognize that Gandin and Murphy’s two requirements are independent, and the second can be safely discarded without losing the key advantages of equitability that are embodied in the first. This enables inequitable and asymptotically equitable measures to be scaled to make them equitable, while retaining their nonlinearity and other properties such as being reliable indicators of skill for rare events. It also opens up the possibility of designing new equitable verification measures.

Sentence repetition in adolescents with specific language impairments and autism: an investigation of complex syntax

Background: Recent studies have indicated that many children with autism spectrum disorders present with language difficulties that are similar to those of children with specific language impairments, leading some to argue for similar structural deficits in these two disorders. Aims: Repetition of sentences involving long-distance dependencies was used to investigate complex syntax in these groups. Methods & Procedures: Adolescents with specific language impairments (mean age = 15;3, n = 14) and autism spectrum disorders plus language impairment (autism plus language impairment; mean age = 14;8, n = 16) were recruited alongside typically developing adolescents (mean age = 14;4, n = 17). They were required to repeat sentences containing relative clauses that varied in syntactic complexity. Outcomes & Results: The adolescents with specific language impairments presented with greater syntactic difficulties than the adolescents with autism plus language impairment, as manifested by higher error rates on the more complex object relative clauses, and a greater tendency to make syntactic changes during repetition. Conclusions & Implications: Adolescents with specific language impairments may have more severe syntactic difficulties than adolescents with autism plus language impairment, possibly due to their short-term memory limitations.

A method for the economic valuation of animal welfare benefits using a single welfare score

Unless the benefits to society of measures to protect and improve the welfare of animals are made transparent by means of their valuation they are likely to go unrecognised and cannot easily be weighed against the costs of such measures as required, for example, by policy-makers. A simple single measure scoring system, based on the Welfare Quality® index, is used, together with a choice experiment economic valuation method, to estimate the value that people place on improvements to the welfare of different farm animal species measured on a continuous (0-100) scale. Results from using the method on a survey sample of some 300 people show that it is able to elicit apparently credible values. The survey found that 96% of respondents thought that we have a moral obligation to safeguard the welfare of animals and that over 72% were concerned about the way farm animals are treated. Estimated mean annual willingness to pay for meat from animals with improved welfare of just one point on the scale was £5.24 for beef cattle, £4.57 for pigs and £5.10 for meat chickens. Further development of the method is required to capture the total economic value of animal welfare benefits. Despite this, the method is considered a practical means for obtaining economic values that can be used in the cost-benefit appraisal of policy measures intended to improve the welfare of animals.

Differential effects of internal and external factors on the development of vocabulary, tense morphology and morpho-syntax in successive bilingual children

The present study investigates the effects of child internal (age/time) and child external/environmental factors on the development of a wide range of language domains in successive bilingual (L2) Turkish-English children of homogeneously low SES. Forty-three L2 children were tested on standardized assessments examining the acquisition of vocabulary and morpho-syntax. The L2 children exhibited a differential acquisition of the various domains: they were better on the general comprehension of grammar and tense morphology and less accurate on the acquisition of vocabulary and (complex) morpho-syntax. Profile effects were confirmed by the differential effects of internal and external factors on the language domains. The development of vocabulary and complex syntax were affected by internal and external factors, whereas external factors had no contribution to the development of tense morphology. These results are discussed in light of previous studies on the impact of internal and external factors in child L2 acquisition.