Search PubMed⌕ Search

SEARCH · Search PubMed

Results for “Statistical Power”

Search indexed PubMed citations on genomics, clinical trials, systematic reviews and public health. Explore titles, authors and supplied subject terms, then open the PubMed record.

Quote a phrase for an exact phrase match. Source license links do not imply unrestricted reuse.

At least 1,153 records · Page 64Linked to original sources

Statistical analysis of neural organization.

1. The responses of 32 taste neurons in the solitary nucleus of the rat to 12 stimuli were analyzed with multidimensional scaling (MDS) and cluster analysis (CA) procedures. These analyses of empirical taste data were compared with similar analyses of two model data sets of known configuration to help clarify the implications of these methods commonly used in forming conclusions about the organization of the taste system. 2. To relate to possible conclusions about groupings in taste, both model data sets were chosen as the best possible examples of ungrouped data, the first being completely regular (in the form of a checkerboard) across the taste space, the second randomly arranged. The analysis of the present empirical data appear to be similar to the present ungrouped models, more so the random than the regular model, in the sense that all are amenable to grouping. 3. Because of the similarity of these model MDS and CA solutions to the present empirical solutions and to most published analyses of this sort, the idea is suggested that the appearance of the plots per se for empirical data does not support the conclusion of grouping. And, technically, MDS and CA do not have the statistical power to provide conclusions about issues of neural organization. 4. MDS and CA analyses have two very powerful roles relating to their ability to disclose the hidden organization of complex data sets; they may lend support for or refute theories about the data sets developed from other considerations, and may help generate theories for further consideration. The question of groupings is only one of many such issues. 5. Because data in the present and other reports are quite adequately accounted by MDS solutions of low dimensionality, it is suggested that their organization is characterized as continuous (i.e., rather than belonging to several disjoint spaces). 6. The use of correlations as distance measures in MDS and CA procedures distorts the spatial solutions, making analysis by visual inspection misleading. For example, using correlations, the true or natural spatial arrangements of data sets are probably less circular or spherical than shown in published MDS solutions. Also they are probably more evenly distributed across the space in the sense that the points are actually more concentrated toward the centers of the spaces; this may have strong influences on interpretations of the general form of the solutions. CA solutions can be influenced in analogous fashion. These problems of distortion of the solutions can be avoided with use of direct, linear estimates of distances. (ABSTRACT TRUNCATED AT 400 WORDS)

Afferent Pathways↗

The effect of stannous fluoride on dentinal hypersensitivity.

Many agents have shown varying degrees of effectiveness on pain resulting from exposed dentine. One which has shown some promising results is stannous fluoride (SnF2). The purpose of the following paper is twofold: to review and summarise the clinical literature pertaining to the relative effectiveness of solutions or gels containing SnF2 in controlling pain associated with dentinal hypersensitivity; and to statistically re-evaluate these studies in combination, in order to develop recommendations for the optimal use of SnF2 for hypersensitivity. Seven blinded clinical studies were identified and reviewed. Five of these compared 0.4 per cent SnF2 gel solution to an identical placebo. One compared a 0.4 per cent SnF2 gel solution and a 0.717 per cent F solution to an aqueous placebo. The final study compared a 0.717 per cent F solution to an aqueous placebo. Statistical power analysis and a combined meta-analysis were used to ensure adequate internal consistency and to contribute to an overall consensus of the efficacy across time. It was concluded that the 0.717 per cent F solution provides a virtually immediate and definable effect, which seems to continue for several months. This effect was present in all subjects used in the study. This solution was applied directly to the sensitive area for one minute and allowed to remain for 3-5 minutes. An additional one minute application was applied if needed. The effect of the 0.4 per cent SnF2 gel appears to be more gradual, perhaps involving a different mechanism of action. This solution requires approximately two to four weeks of continuous treatment to be effective. It was concluded that an effective strategy involving the use of stannous fluoride gel includes the application of the 0.717 per cent F solution in the office, effectively providing immediate relief. The patient would then use the 0.4 per cent SnF2 gel at home in order to achieve the long-term effect. In order to control episodic pain while the gel is developing its effect, a small amount of the 0.717 per cent F solution could be given to the patient for occasional symptomatic application.

Dentin Sensitivity↗

Interleaved silent steady state (ISSS) imaging: a new sparse imaging method applied to auditory fMRI.

The acoustic scanner noise that is generated by rapid gradient switching in echo planar imaging (EPI) is an important confounding factor in auditory fMRI. "Sparse imaging" designs overcome the influence of scanner noise on stimulus presentation by acquiring single brain volumes following a silent stimulus presentation period. However, conventional sparse imaging requires assumptions about the time-to-peak of the evoked hemodynamic response and reduces the amount of EPI data which can be acquired and hence statistical power. In this article, we describe an "interleaved silent steady state" (ISSS) sampling scheme in which we rapidly acquire a set of EPI volumes following each silent stimulus presentation period. We avoid T1-related signal decay during the acquisition of the EPI volumes by maintaining the steady state longitudinal magnetization with a train of silent slice-selective excitation pulses during the silent period, ensuring that signal contrast is constant across successive scans. A validation study comparing ISSS to conventional sparse imaging demonstrates that ISSS imaging provides time course information that is absent in conventional sparse imaging data. The ISSS sequence has a temporal resolution like event-related (ER) imaging within a single trial (unlike conventional sparse imaging, where ER-like temporal resolution can only be achieved by compiling data across many jittered trials of the same stimulus type). This temporal resolution within trials makes ISSS particularly suitable for experiments in which a) scanner noise would interfere with the perception and processing of the stimulus; b) stimuli are several seconds in duration, and activation is expected to evolve and change as the stimulus unfolds; and c) it is impractical to present a single stimulus more than once (for example, repetition priming or familiarity effects would be expected).

Acoustic Stimulation↗

Methods of collecting and evaluating non-clinical cardiac electrophysiology data in the pharmaceutical industry: results of an international survey.

OBJECTIVE: To assess current practice in the pharmaceutical industry for assessing the potential for QT interval prolongation by non-cardiovascular medicinal products. METHODS: The survey was based on responses from the Toxicology and (Safety) Pharmacology laboratories (a total of 74 laboratories) of 54 companies based in Europe, Japan/Asia and the USA, received between January and March 1999. RESULTS: All 54 companies conducted preclinical in vivo electrocardiography (EGG) evaluation of new active substances (NASs). Thirty of these companies also conducted in vitro cardiac electrophysiology studies on their compounds. The majority of in vivo work was done in conscious beagle dogs. There was no consistency within the industry in defining the magnitude of change in QT interval that is considered biologically important. Most companies considered a change greater than 10% to be important, although the design of the studies suggested that group sizes used may not give sufficient statistical power to detect this size of change. Bazett's formula was used by 41% of laboratories to correct QT for changes in heart rate, despite the fact that this formula is generally deemed to be unsuitable for use in dogs. For studies in anaesthetised dogs, the majority of laboratories used barbiturate anaesthesia, but researchers should be aware of the effects of this and some other anaesthetic agents on QT interval. As for in vitro cardiac electrophysiology, there was wide diversity in the testing methodologies, particularly with regard to the test species and tissue type. As with QT prolongation, there was no consensus on the degree of action potential prolongation to cause concern. For both in vitro and in vivo testing, the majority of companies tested a minimum of three dose (or concentration) levels in order to ascertain any dose-response relationship. CONCLUSIONS: The survey provides a snapshot of the practice in the industry prior to any internationally-agreed consensus on the most effective and efficient approaches to minimising the risk of QT prolongation by new drugs in man. It must be stated that for any given methodology, the 'majority view' in the industry is not necessarily best practice.

Action Potentials↗

Design and analysis of admixture mapping studies.

Admixture between populations originating on different continents can be exploited to detect disease susceptibility loci at which risk alleles are distributed differentially between these populations. We first examine the statistical power and mapping resolution of this approach in the limiting situation in which gamete admixture and locus ancestry are measured without uncertainty. We show that, for a rare disease, the most efficient design is to study affected individuals only. In a typical African American population (two-way admixture proportions 0.8/0.2, ancestry crossover rate 2 per 100 cM), a study of 800 affected individuals has 90% power to detect at P values <10(-5) a locus that generates a risk ratio of 2 between populations, with an expected mapping resolution (size of 95% confidence region for the position of the locus) of 4 cM. In practice, to infer locus ancestry from marker data requires Bayesian computationally intensive methods, as implemented in the program ADMIXMAP. Affected-only study designs require strong prior information on the frequencies of each allele given locus ancestry. We show how data from unadmixed and admixed populations can be combined to estimate these ancestry-specific allele frequencies within the admixed population under study, allowing for variation between allele frequencies in unadmixed and admixed populations. Using simulated data based on the genetic structure of the African American population, we show that 60% of information can be extracted in a test for linkage using markers with an ancestry information content of 36% at 3-cM spacing. As in classic linkage studies, the most efficient strategy is to use markers at a moderate density for an initial genome search and then to saturate regions of putative linkage with additional markers, to extract nearly all information about locus ancestry.

Black People↗

Neuroimaging studies of word and pseudoword reading: consistencies, inconsistencies, and limitations.

Several functional neuroimaging studies have compared words and pseudowords to test different cognitive models of reading. There are difficulties with this approach, however, because cognitive models do not make clear-cut predictions at the neural level. Therefore, results can only be interpreted on the basis of prior knowledge of cognitive anatomy. Furthermore, studies comparing words and pseudowords have produced inconsistent results. The inconsistencies could reflect false-positive results due to the low statistical thresholds applied or confounds from nonlexical aspects of the stimuli. Alternatively, they may reflect true effects that are inconsistent across subjects; dependent on experimental parameters such as stimulus rate or duration; or not replicated across studies because of insufficient statistical power. In this fMRI study, we investigate consistent and inconsistent differences between word and pseudoword reading in 20 subjects, and distinguish between effects associated with increases and decreases in activity relative to fixation. In addition, the interaction of word type with stimulus duration is explored. We find that words and pseudowords activate the same set of regions relative to fixation, and within this system, there is greater activation for pseudowords than words in the left frontal operculum, left posterior inferior temporal gyrus, and the right cerebellum. The only effects of words relative to pseudowords consistent over subjects are due to decreases in activity for pseudowords relative to fixation; and there are no significant interactions between word type and stimulus duration. Finally, we observe inconsistent but highly significant effects of word type at the individual subject level. These results (i) illustrate that pseudowords place increased demands on areas that have previously been linked to lexical retrieval, and (ii) highlight the importance of including one or more baselines to qualify word type effects. Furthermore, (iii) they suggest that inconsistencies observed in the previous literature may result from effects arising from a small number of subjects only.

Adult↗

Does effect size in naltrexone trials for alcohol dependence differ for single-site vs. multi-center studies?

BACKGROUND: The opioid antagonist naltrexone was first shown in single-site trials to be efficacious in the treatment of alcohol dependence. Recent clinical trials of the medication have used multi-center designs, which permit greater generalization and increased statistical power. We compared effect sizes for these two kinds of trial design on the hypothesis that multi-center trials introduce sources of variation that reduce the observed effect size. METHODS: A meta-analysis of data from 19 placebo-controlled trials of the efficacy of naltrexone (7 multi-center and 12 single-site studies) was performed. Effect size estimates for these two study designs were compared using two outcomes: percentage of days drinking and percentage of subjects relapsing to heavy drinking. RESULTS: Compared with single-site studies, multi-center studies were estimated to yield a nonsignificantly smaller effect on the percentage of days drinking (Cohen's d = 0.20 vs. 0.33, respectively) and a significantly smaller effect on the percentage of subjects relapsing to heavy drinking (Cohen's d = 0.17 vs. 0.41, respectively; p = 0.014). Earlier studies showed a larger effect size than later studies. CONCLUSION: The smaller effect size seen with multi-center studies may reflect random error due to heterogeneity among the sites. However, because multi-center studies were, in general, conducted more recently than single-site studies, it was not possible conclusively to disentangle the moderating impact of study type and year of publication on effect size. Further research on factors that moderate effect size can contribute to the development of medications to treat alcohol dependence.

Alcohol Drinking↗

Estimating sample size for epidemiologic studies: the impact of ignoring exposure measurement uncertainty.

Sample size requirements for epidemiologic studies are usually determined on the basis of the desired level of statistical power. Suppose, however, that one is planning a study in which the participants' true exposure levels are unobservable. Instead, the analysis will be based on an imprecise surrogate measure that differs from true exposure by some non-negligible amount of measurement error. Sample size estimates for tests of association between the surrogate exposure measure and the outcome of interest may be misleading if they are based solely on the anticipated characteristics of the distribution of surrogate measures in the study population. We examine the accuracy of sample size estimates for cohort studies in which a continuous surrogate exposure measure is subject to either classical or Berkson measurement error. In particular, we evaluate the consequences of not adjusting the sample size estimation procedure for tests based on imprecise exposure measurements to account for anticipated differences between the distributions of the true exposure and the surrogate measure in the study population. As expected, failure to adjust for classical measurement error can lead to underestimation of the required sample size. Disregard of Berkson measurement error, however, can result in sample size estimates that exceed the actual number of participants required for tests of association between the outcome and the surrogate exposure measure. We illustrate this Berkson error effect by estimating sample size for a hypothetical cohort study that examines an association between childhood exposure to radioiodine and the development of thyroid neoplasms.

Adolescent↗

Individual trajectories in stress covary with immunity during recovery from cancer diagnosis and treatments.

Research connects stressful events with altered immune regulation, but the role of subjective stress is uncertain. Using a longitudinal design, we provide a statistically powerful test of the relationship between subjective stress (perceived stress, emotional distress) and immunity (T cell blastogenesis, natural killer cell cytotoxicity, [NKCC]) as individuals adjust to a severe stressor, a cancer diagnosis and its treatments. Women with regional breast cancer (N=113) were assessed at diagnosis/surgery and reassessed 4, 8, 12, and 18 months later. Latent growth curve analysis tested two hypotheses: (1) initial levels of subjective stress will correlate inversely with initial levels of immunity, and (2) rate of change in subjective stress will correlate inversely with rate of change in immunity. As predicted by Hypothesis 1, participants with high initial subjective stress showed poor initial blastogenesis. As predicted by Hypothesis 2, participants exhibiting an early, rapid decline in subjective stress also showed rapid improvement in NKCC. Follow-up analyses revealed perceived stress to be strongly related to immune function, while emotional distress was not. This is the first study to investigate trajectories in stress and immunity during recovery from a major stressor. Results imply that NK and T cells are sensitive to different aspects of the stress response. While T cell blastogenesis correlated with initial (peak) subjective stress, NKCC correlated with change (improvement) in subjective stress. These data highlight the importance of subjective stress, particularly stress appraisals, in the immune response to a major stressor.

Adaptation, Psychological↗

Number of days, number of subjects, and sources of variation in longitudinal intervention or crossover feeding trials with multiple days of measurement.

Dietary studies are often conducted as longitudinal intervention or crossover trials using multiple days of measurement on each subject during each of several measurement periods, and determining the required numbers of days and subjects is important in designing these studies. Linear mixed statistical models were used to derive equations for precision, statistical power and sample size (number of days and number of subjects) and to obtain estimates of between-subject, period-to-period, and day-to-day variation needed to apply the equations. Two cohorts of an on-going exercise intervention study, and a crossover study of Olestra, each with 14 d of measurement/subject per period, were used to obtain estimates of variability for energy and macronutrient intake. Numerical examples illustrate how the equations for calculating the number of days or number of subjects are applied in typical situations, and sample SAS code is given. It was found that between-subject, period-to-period, and day-to-day variation all contributed significantly to the variation in energy and macronutrient intake. The ratio of period-to-period and day-to-day standard deviations controls the trade-off between the number of days and the number of subjects, and this remained relatively stable across studies and energy and macronutrient intake variables. The greatest gains in precision were seen over the first few measurement days. Greater precision and fewer required days were noted in the study (Olestra) that exerted greater control over the subjects and diets during the feeding protocol.

Adult↗

Oral 5-aminosalicylic acid for maintenance of remission in ulcerative colitis.

BACKGROUND: The newer 5-ASA preparations were intended to avoid the adverse effects of SASP while maintaining its therapeutic benefits. The efficacy and safety of 5-ASA preparations have been evaluated in numerous clinical trials that have often lacked sufficient statistical power to arrive at definitive conclusions. Previously, it was found that newer 5-ASA drugs were more effective than placebo but no more effective than SASP in inducing remission in ulcerative colitis. This updated review includes more recent studies and evaluates the effectiveness, dose-responsiveness, and safety of 5-ASA preparations in terms of more precise outcome measures. OBJECTIVES: To assess the efficacy, dose-responsiveness and safety of the newer release formulations of 5-aminosalicylic acid (5-ASA) compared to placebo or sulfasalazine (SASP) in the maintenance of remission in ulcerative colitis. SEARCH STRATEGY: A computer-assisted literature search for relevant studies (1981-2005) was performed using MEDLINE, BIOS, the Cochrane Controlled Trials Register, the Cochrane IBD/FBD Group Specialized Trials Register, and the Science Citation Index, followed by a manual search of reference lists from previously retrieved articles, review articles, symposia proceedings, and abstracts from major gastrointestinal conferences. SELECTION CRITERIA: Studies were accepted for analysis if they were prospective, randomized, double-blinded, and placebo- or SASP-controlled clinical trials of parallel design with treatment duration of at least six months. DATA COLLECTION AND ANALYSIS: Based on an intention to treat principle, the primary outcome was the failure to maintain clinical or endoscopic remission. Secondary outcomes were the number of patients experiencing adverse events, the number of patients withdrawn due to adverse events, and exclusions or withdrawals after entry into the study (not due to relapse). All data were analyzed using the Peto odds ratio and corresponding 95% confidence intervals (CI). MAIN RESULTS: The Peto odds ratio for the failure to maintain clinical or endoscopic remission (withdrawals and relapses) for 5-ASA versus placebo was 0.47 (95% CI, 0.36 to 0.62) with an NNT of 6. These values were also calculated for the trials in which SASP and 5-ASA were compared, revealing an odds ratio of 1.29 (95% CI, 1.05 to 1.57), with a negative NNT value (-19), suggesting a higher degree of therapeutic effectiveness for SASP.SASP and 5-ASA had similar adverse event profiles, with odds ratios of 1.16(0.62 to 2.16), and 1.31(0.86 to 1.99), respectively. The NNH values were determined to be 171 and 78 respectively. AUTHORS' CONCLUSIONS: The newer 5-ASA preparations were superior to placebo in maintenance therapy. However, the newer preparations had a statistically significant therapeutic inferiority relative to SASP. This review updates the existing review of oral 5-aminosalicylic acid for maintenance of remission in ulcerative colitis which was published in the Cochrane Library (Issue 1, 2006).

Administration, Oral↗

Sample size for identifying differentially expressed genes in microarray experiments.

Microarray technology allows simultaneous comparison of expression levels of thousands of genes under each condition. This paper concerns sample size calculation in the identification of differentially expressed genes between a control and a treated sample. In a typical experiment, only a fraction of genes (altered genes) is expected to be differentially expressed between two samples. Sample size determination depends on a number of factors including the specified significance level (alpha), the desired statistical power (1-beta), the fraction (eta) of truly altered genes out of the total g genes studied, and the effect sizes (Delta) for the altered genes. This paper proposes a method to calculate the number of arrays required to detect at least 100lambda % (where 0 < lambda < or = 1) of the truly altered genes under the model of an equal effect size for all altered genes. The required numbers of arrays are tabulated for various values of alpha, beta, Delta, eta, and lambda for the one-sample and two-sample t-tests for g = 10,000. Based on the proposed approach, to identify up to 90% of truly altered genes among the unknown number of truly altered genes, the estimated numbers of arrays needed appear to be manageable. For instance, when the standardized effect size is at least 2.0, the number of arrays needed is less than or equal to 14 for the two-sample t-test and is less than or equal to 10 for the one-sample t-test. As the cost per array declines, such array numbers become practical. The proposed method offers a simple, intuitive, and practical way to determine the number of arrays needed in microarray experiments in which the true correlation structure among the genes under investigation cannot be reasonably assumed. An example dataset is used to illustrate the use of the proposed approach to plan microarray experiments.

Animals↗

Oral 5-aminosalicylic acid for maintaining remission in ulcerative colitis.

OBJECTIVES: To assess the efficacy, dose-responsiveness and safety of the newer release formulations of 5-aminosalicylic acid (5-ASA) compared to placebo or sulfasalazine (SASP) in the maintenance of remission in ulcerative colitis. SEARCH STRATEGY: A computer-assisted literature search for relevant studies (1981-1998) was performed using MEDLINE, BIOS, the Cochrane Controlled Trials Register, the Inflammatory Bowel Disease Trials Register, and Science Citation Index, followed by a manual search of reference lists from previously retrieved articles, review articles, symposia proceedings, and abstracts from major gastrointestinal conferences. SELECTION CRITERIA: Studies were accepted for analysis if they were prospective, randomized, double-blinded, and placebo- or SASP-controlled clinical trials of parallel design with treatment duration of at least six months. DATA COLLECTION AND ANALYSIS: Based on an intention to treat principle, the primary outcome was the failure to maintain clinical or endoscopic remission. Secondary outcomes were the number of patients experiencing adverse events, the number of patients withdrawn due to adverse events, and exclusions or withdrawals after entry into the study (not due to relapse). All data were analyzed using the Peto odds ratio and corresponding 95% confidence intervals (CI). MAIN RESULTS: The Peto odds ratio for the failure to maintain clinical or endoscopic remission (withdrawals and relapses) for 5-ASA versus placebo was 0. 47 (95% CI, 0.36 to 0.62) with an NNT of 6. These values were also calculated for the trials in which SASP and 5-ASA were compared, revealing an odds ratio of 1.29 (95% CI, 1.05 to 1.57), with a negative NNT value (-19), suggesting a higher degree of therapeutic effectiveness for SASP. SASP and 5-ASA had similar adverse event profiles, with odds ratios of 1.16(0.62 to 2.16), and 1.31(0.86 to 1.99), respectively. The NNH values were determined to be 171 and 78 respectively. REVIEWER'S CONCLUSIONS: The newer 5-ASA preparations were superior to placebo in maintenance therapy. However, the newer preparations had a statistically significant therapeutic inferiority relative to SASP.

Administration, Oral↗

Analysis of speech-related variance in rapid event-related fMRI using a time-aware acquisition system.

Speech production introduces signal changes in fMRI data that can mimic or mask the task-induced BOLD response. Rapid event-related designs with variable ISIs address these concerns by minimizing the correlation of task and speech-related signal changes without sacrificing efficiency; however, the increase in residual variance due to speech still decreases statistical power and must be explicitly addressed primarily through post-processing techniques. We investigated the timing, magnitude, and location of speech-related variance in an overt picture naming fMRI study with a rapid event-related design, using a data acquisition system that time-stamped image acquisitions, speech, and a pneumatic belt signal on the same clock. Using a spectral subtraction algorithm to remove scanner gradient noise from recorded speech, we related the timing of speech, stimulus presentation, chest wall movement, and image acquisition. We explored the relationship of an extended speech event time course and respiration on signal variance by performing a series of voxelwise regression analyses. Our results demonstrate that these effects are spatially heterogeneous, but their anatomic locations converge across subjects. Affected locations included basal areas (orbitofrontal, mesial temporal, brainstem), areas adjacent to CSF spaces, and lateral frontal areas. If left unmodeled, speech-related variance can result in regional detection bias that affects some areas critically implicated in language function. The results establish the feasibility of detecting and mitigating speech-related variance in rapid event-related fMRI experiments with single word utterances. They further demonstrate the utility of precise timing information about speech and respiration for this purpose.

Adult↗

AFLP utility for population assignment studies: analytical investigation and empirical comparison with microsatellites.

Individual-based population assignment tests have thus far mainly relied on the use of microsatellite loci. However, the logistic difficulty of screening large numbers of loci required to reach sufficient statistical power hampers the usefulness of microsatellites in situations of weak population structuring. Amplified fragment length polymorphisms (AFLP) represents an alternative for overcoming this logistical issue as the technique allows the user to characterize a much larger number of loci with a comparable analytical effort. In this study, an assignment test based on maximum likelihood for dominant markers was used to investigate the potential usefulness of AFLP for population assignment. We also compared assignment success achieved with AFLP with that obtained using microsatellites in a case study of low population differentiation involving whitefish (Coregonus clupeaformis) sympatric ecotypes. The analytical investigation showed that the minimum number of AFLP loci required to reach an assignment success of 95% stood within values that are easily achievable in many situations. This also showed how assignment success varied according to the number of AFLP loci used, their absolute frequency and their frequency differential and sampling errors, as well as the number of putative source populations. The case study showed that given a comparable analytical effort in the laboratory, AFLP were much more efficient than the microsatellite loci in discriminating the source of an individual among putative populations. AFLP resulted in higher assignment success at all levels of stringency and the log-likelihood differences between populations obtained with AFLP for each individual were much larger than those obtained with microsatellites. These results indicate that research involving individual-based population assignment methods should benefit importantly from the use of AFLP markers, especially in systems characterized by weak population structuring.

Data Interpretation, Statistical↗

Occupational exposure to solvents and gender-related risk of systemic sclerosis: a metaanalysis of case-control studies.

OBJECTIVE: In 2001 a metaanalysis reported an excess risk of systemic sclerosis (SSc) related to solvents exposure. The magnitude of risk varied among studies and sources of heterogeneity have not been investigated due to a lack of statistical power. We conducted a new metaanalysis to identify features associated with the magnitude of SSc risk in patients exposed to solvents. METHODS: We searched 4 databases (Medline, Pascal, Pascal Biomed, Francis). Inclusion criteria were: case-control study, occupational exposure to solvents (OES) assessed by questionnaire and summarized to "any solvent" or "any organic solvent," SSc defined by the American College of Rheumatology or the consultant's criteria. The quality of studies within this metaanalysis was scored according to the Newcastle-Ottawa scale. Odds ratios (OR) were adjusted for the "publication bias" and validated by a sensitivity analysis. Subgroup analyses investigated the effect of gender, quality of studies, and the type of controls. RESULTS: Among 11 studies (1291 patients and 3435 controls), 9 involved a majority of women (76.2 to 100%), while 2 involved men only. The risk of SSc associated with OES was variable among studies (p for heterogeneity = 0.01) and overrepresentation of higher OR values in smaller studies (p = 0.003) suggested "publication bias." SSc was associated with OES (OR 2.4; 95% CI 1.7-3.4; p < 0.0001), including after adjusting for bias (OR 1.8; 95% CI 1.2-2.5; p = 0.002). The relative risk was higher (p = 0.03) in men (OR 3.0; 95% CI 1.9-4.6; p < 0.0001) than in women (OR 1.8; 95% CI 1.5-2.1; p < 0.0001). CONCLUSION: Whereas SSc affects women predominantly, among subjects with occupational exposure to solvents, men are at higher risk than women for the disease.

Case-Control Studies↗

A new method for choosing sample size for confidence interval-based inferences.

Scientists often need to test hypotheses and construct corresponding confidence intervals. In designing a study to test a particular null hypothesis, traditional methods lead to a sample size large enough to provide sufficient statistical power. In contrast, traditional methods based on constructing a confidence interval lead to a sample size likely to control the width of the interval. With either approach, a sample size so large as to waste resources or introduce ethical concerns is undesirable. This work was motivated by the concern that existing sample size methods often make it difficult for scientists to achieve their actual goals. We focus on situations which involve a fixed, unknown scalar parameter representing the true state of nature. The width of the confidence interval is defined as the difference between the (random) upper and lower bounds. An event width is said to occur if the observed confidence interval width is less than a fixed constant chosen a priori. An event validity is said to occur if the parameter of interest is contained between the observed upper and lower confidence interval bounds. An event rejection is said to occur if the confidence interval excludes the null value of the parameter. In our opinion, scientists often implicitly seek to have all three occur: width, validity, and rejection. New results illustrate that neglecting rejection or width (and less so validity) often provides a sample size with a low probability of the simultaneous occurrence of all three events. We recommend considering all three events simultaneously when choosing a criterion for determining a sample size. We provide new theoretical results for any scalar (mean) parameter in a general linear model with Gaussian errors and fixed predictors. Convenient computational forms are included, as well as numerical examples to illustrate our methods.

Biometry↗

The early toxicology of physostigmine: a tale of beans, great men and egos.

Mid-19th century European visitors to Old Calabar, an eastern province of Nigeria, could not avoid becoming aware of native belief in the power of the seeds of a local plant to determine whether individuals were innocent or guilty of some serious misdemeanour. The seeds were those of a previously unknown legume and soon referred to as the ordeal bean of Old Calabar. Their administration was known locally as 'chop nut'. Missionaries who arrived in Calabar in 1846 estimated that chop nut caused some 120 deaths annually and documented the course of poisoning. The latter information and samples of the beans rapidly found their way to Scotland, the home of the missionaries' parent church, explaining why the early toxicology of physostigmine, quantitatively the most important of three active alkaloids in the beans, has such strong Scottish, predominantly Edinburgh, associations. However, it was 1855 before the first of many medical scientists, Robert Christison, a toxicologist of repute, investigated the effects of the beans to the extent of eating part of one himself and documenting the moderate, if not severe, consequences. A further 6 years were to pass before Balfour's comprehensive botanical description of the bean plant appeared. It was he who named it Physostigma venenosum. It was not so long until the next event, one that sparked more intensive and international interest in the beans. In 1863 a young Edinburgh ophthalmologist, Argyll Robertson, published a paper announcing the arrival of the first agent that constricted the pupil of the eye. The drug was an extract of Calabar beans and Argyll Robertson openly admitted that he had been alerted to its unusual property by his physician friend, Thomas Fraser. A minor flood of contributions on the ophthalmic uses of bean extracts followed in the medical press in the next few months; those on their systemic toxicity were fewer. Fraser's MD thesis, submitted to the University of Edinburgh in 1862 and clearly pre-dating Argyll Robertson's involvement with the beans, became generally available a few weeks after the appearance of Argyll Robertson's paper and was the first to address in detail the features of systemic administration of extracts of the beans. A major problem facing all early researchers of the beans was that of deciding how best to extract their active principle, a task made all the more difficult because bioassays were the only means of determining if the toxin was being tracked. The stability of extracts was an inevitable issue and the active principle finally became known as physostigma or physostigmine, after the botanical name of the parent plant. The features of physostigmine toxicity were soon exhaustively documented, both in animals and humans. How they were mediated was another matter altogether. Fraser maintained that muscular paralysis, the cardinal feature, was the result of depression of the spinal cord and was generally, but far from unanimously, supported. Of those who had reservations, Harley was the most prominent. He concluded that paralysis was secondary to effects on the motor nerve endings and, in so doing, came nearest to present-day knowledge at a time when acetylcholine, cholinesterases and cholinesterase inhibitors were not even imagined. Differences of opinion on the mode of action of the beans were to be expected and it is hardly surprising that they were not resolved. No standard formulation of physostigmine was available so the potency of those used would have varied from one investigator to another, the range of animals experimented upon was large while the number used by any researcher was commonly in single figures, more readily available cold-blooded creatures seemed less sensitive to physostigmine toxicity than warm-blooded ones and only Fraser determinedly pursued an answer; in general, the others made one foray into bean research then turned their attentions elsewhere. The same problems would beset other aspects of bean research. While Fraser did not get as close to the mode of action of physostigmine as Harley, he reigns supreme when it comes to antagonism between physostigmine and atropine. By this time, the 1870s had dawned and although the concept of antagonism between therapeutic agents was not new, it had little, if any, reliable scientific foundation. This was about to change; antagonism was becoming exciting and rational. Fraser's firm belief that physostigmine and atropine were mutually antagonistic at a physiological level was contrary to the conventional wisdom of his contemporaries. This alone would earn him a place in history but his contribution goes much, much further. Unlike any other at the time, he investigated it with scientific rigour, experimenting on only one species, ensuring as best he could the animals were the same weight, adjusting the doses of drugs he gave them for bodyweight, determining the minimum lethal dose of each drug before assessing their antagonistic effects, adopting a single, incontrovertible endpoint for efficacy and carrying out sufficient numbers of experiments to appear convincing in a later era where the statistical power of studies is all-important. To crown it all, he presented his results graphically. Fraser never claimed to have discovered the antagonism between physostigmine and atropine. Bartholow in 1873 did, based on work done in 1869. But his data hardly justify it. If anyone can reasonably claim this particular scientific crown it is an ophthalmologist, Niemetschek, working in Prague in 1864. His colleague in the same discipline, Kleinwächter, was faced with treating a young man with atropine intoxication. Knowing of the contrary actions of the two drugs on the pupil, Niemetschek suggested that Calabar bean extract might be useful. Kleinwächter had the courage to take the advice and his patient improved dramatically. Clearly, this evidence is nothing more than anecdotal, but the ophthalmologists were correct and, to the present day, physostigmine has had an intermittent role in the management of anticholinergic poisoning. The converse, giving atropine to treat poisoning with cholinesterase inhibitors, of which physostigmine was the first, has endured more consistently and remains standard practice today. It is salutary to realise that the doses and dosage frequency of atropine together with the endpoints that define they are adequate were formulated by Fraser and others a century and a half ago.

Animals↗