Concept: Sample size
Figures in scientific publications are critically important because they often show the data supporting key findings. Our systematic review of research articles published in top physiology journals (n = 703) suggests that, as scientists, we urgently need to change our practices for presenting continuous data in small sample size studies. Papers rarely included scatterplots, box plots, and histograms that allow readers to critically evaluate continuous data. Most papers presented continuous data in bar and line graphs. This is problematic, as many different data distributions can lead to the same bar or line graph. The full data may suggest different conclusions from the summary statistics. We recommend training investigators in data presentation, encouraging a more complete presentation of data, and changing journal editorial policies. Investigators can quickly make univariate scatterplots for small sample size studies using our Excel templates.
- Proceedings of the National Academy of Sciences of the United States of America
- Published over 4 years ago
Both social isolation and loneliness are associated with increased mortality, but it is uncertain whether their effects are independent or whether loneliness represents the emotional pathway through which social isolation impairs health. We therefore assessed the extent to which the association between social isolation and mortality is mediated by loneliness. We assessed social isolation in terms of contact with family and friends and participation in civic organizations in 6,500 men and women aged 52 and older who took part in the English Longitudinal Study of Ageing in 2004-2005. A standard questionnaire measure of loneliness was administered also. We monitored all-cause mortality up to March 2012 (mean follow-up 7.25 y) and analyzed results using Cox proportional hazards regression. We found that mortality was higher among more socially isolated and more lonely participants. However, after adjusting statistically for demographic factors and baseline health, social isolation remained significantly associated with mortality (hazard ratio 1.26, 95% confidence interval, 1.08-1.48 for the top quintile of isolation), but loneliness did not (hazard ratio 0.92, 95% confidence interval, 0.78-1.09). The association of social isolation with mortality was unchanged when loneliness was included in the model. Both social isolation and loneliness were associated with increased mortality. However, the effect of loneliness was not independent of demographic characteristics or health problems and did not contribute to the risk associated with social isolation. Although both isolation and loneliness impair quality of life and well-being, efforts to reduce isolation are likely to be more relevant to mortality.
Objective To determine if a simple stimulation method increases the rate of infant voiding for clean catch urine within five minutes.Design Randomised controlled trial.Setting Emergency department of a tertiary paediatric hospital, Australia.Participants 354 infants (aged 1-12 months) requiring urine sample collection as determined by the treating clinician. 10 infants were subsequently excluded.Interventions Infants were randomised to either gentle suprapubic cutaneous stimulation (n=174) using gauze soaked in cold fluid (the Quick-Wee method) or standard clean catch urine with no additional stimulation (n=170), for five minutes.Main outcome measures The primary outcome was voiding of urine within five minutes. Secondary outcomes were successful collection of a urine sample, contamination rate, and parental and clinician satisfaction with the method.Results The Quick-Wee method resulted in a significantly higher rate of voiding within five minutes compared with standard clean catch urine (31% v 12%, P<0.001), difference in proportions 19% favouring Quick-Wee (95% confidence interval for difference 11% to 28%). Quick-Wee had a higher rate of successful urine sample collection (30% v 9%, P<0.001) and greater parental and clinician satisfaction (median 2 v 3 on a 5 point Likert scale, P<0.001). The difference in contamination between Quick-Wee and standard clean catch urine was not significant (27% v 45%, P=0.29). The number needed to treat was 4.7 (95% confidence interval 3.4 to 7.7) to successfully collect one additional urine sample within five minutes using Quick-Wee compared with standard clean catch urine.Conclusions Quick-Wee is a simple cutaneous stimulation method that significantly increases the five minute voiding and success rate of clean catch urine collection.Trial registration Australian New Zealand Clinical Trials Registry ACTRN12615000754549.
Background Previous trials suggesting that high-frequency oscillatory ventilation (HFOV) reduced mortality among adults with the acute respiratory distress syndrome (ARDS) were limited by the use of outdated comparator ventilation strategies and small sample sizes. Methods In a multicenter, randomized, controlled trial conducted at 39 intensive care units in five countries, we randomly assigned adults with new-onset, moderate-to-severe ARDS to HFOV targeting lung recruitment or to a control ventilation strategy targeting lung recruitment with the use of low tidal volumes and high positive end-expiratory pressure. The primary outcome was the rate of in-hospital death from any cause. Results On the recommendation of the data monitoring committee, we stopped the trial after 548 of a planned 1200 patients had undergone randomization. The two study groups were well matched at baseline. The HFOV group underwent HFOV for a median of 3 days (interquartile range, 2 to 8); in addition, 34 of 273 patients (12%) in the control group received HFOV for refractory hypoxemia. In-hospital mortality was 47% in the HFOV group, as compared with 35% in the control group (relative risk of death with HFOV, 1.33; 95% confidence interval, 1.09 to 1.64; P=0.005). This finding was independent of baseline abnormalities in oxygenation or respiratory compliance. Patients in the HFOV group received higher doses of midazolam than did patients in the control group (199 mg per day [interquartile range, 100 to 382] vs. 141 mg per day [interquartile range, 68 to 240], P<0.001), and more patients in the HFOV group than in the control group received neuromuscular blockers (83% vs. 68%, P<0.001). In addition, more patients in the HFOV group received vasoactive drugs (91% vs. 84%, P=0.01) and received them for a longer period than did patients in the control group (5 days vs. 3 days, P=0.01). Conclusions In adults with moderate-to-severe ARDS, early application of HFOV, as compared with a ventilation strategy of low tidal volume and high positive end-expiratory pressure, does not reduce, and may increase, in-hospital mortality. (Funded by the Canadian Institutes of Health Research; Current Controlled Trials numbers, ISRCTN42992782 and ISRCTN87124254 , and ClinicalTrials.gov numbers, NCT00474656 and NCT01506401 .).
Studies examining the relation of information processing speed, as measured by reaction time, with mortality are scarce. We explored these associations in a representative sample of the US population.
We wanted to examine tolerability and efficacy of NSI-189, a benzylpiperizine-aminiopyridine neurogenic compound for treating major depressive disorder (MDD). This was a Phase 1B, double blind, randomized, placebo controlled, multiple-dose study with three cohorts. The first cohort received 40 mg q.d. (n=6) or placebo (n=2), the second cohort 40 mg b.i.d. (n=6) or placebo (n=2), and the third cohort 40 mg t.i.d. (n=6) or placebo (n=2). Twenty-four patients with MDD were recruited, with the diagnosis and severity confirmed through remote interviews. Eligible patients received NSI-189 or placebo for 28 days in an inpatient setting with assessments for safety, pharmacokinetics (PK) and efficacy. Outpatient follow-up visits were conducted until day 84 (±3). NSI-189 was relatively well tolerated at all doses, with no serious adverse effects. NSI-189 area under the curve increased in a dose-related and nearly proportional manner across the three cohorts, with a half-life of 17.4-20.5 h. The exploratory efficacy measurements, including Symptoms Of Depression Questionnaire (SDQ), Montgomery-Asberg Depression Scale (MADRS), Clinical Global Impressions-Improvement (CGI-I), and The Massachusetts General Hospital (MGH) Cognitive and Physical Functioning Questionnaire (CPFQ) showed a promising reduction in depressive and cognitive symptoms across all measures for NSI-189, with significant improvement in the SDQ and CPFQ, and a medium to large effect size for all measures. These improvements persisted during the follow-up phase. In summary, NSI-189 shows potential as a treatment for MDD in an early phase study. The main limitation of this preliminary study was the small sample size of each cohort.Molecular Psychiatry advance online publication, 8 December 2015; doi:10.1038/mp.2015.178.
The largest cities, the most frequently used words, the income of the richest countries, and the most wealthy billionaires, can be all described in terms of Zipf’s Law, a rank-size rule capturing the relation between the frequency of a set of objects or events and their size. It is assumed to be one of many manifestations of an underlying power law like Pareto’s or Benford’s, but contrary to popular belief, from a distribution of, say, city sizes and a simple random sampling, one does not obtain Zipf’s law for the largest cities. This pathology is reflected in the fact that Zipf’s Law has a functional form depending on the number of events N. This requires a fundamental property of the sample distribution which we call ‘coherence’ and it corresponds to a ‘screening’ between various elements of the set. We show how it should be accounted for when fitting Zipf’s Law.
Numerous studies have looked at the potential benefits of various nootropic drugs such as Ginkgo biloba extract (EGb761®; Tanakan®) and piracetam (Nootropyl®) on age-related cognitive decline often leading to inconclusive results due to small sample sizes or insufficient follow-up duration. The present study assesses the association between intake of EGb761® and cognitive function of elderly adults over a 20-year period.
A barrier to preventative treatments for psychosis is the absence of accurate identification of persons at highest risk. A blood test that could substantially increase diagnostic accuracy would enhance development of psychosis prevention interventions.
BACKGROUND: Lidar height data collected by the Geosciences Laser Altimeter System (GLAS) from 2002 to 2008 has the potential to form the basis of a globally consistent sample-based inventory of forest biomass. GLAS lidar return data were collected globally in spatially discrete full waveform “shots,” which have been shown to be strongly correlated with aboveground forest biomass. Relationships observed at spatially coincident field plots may be used to model biomass at all GLAS shots, and well-established methods of model-based inference may then be used to estimate biomass and variance for specific spatial domains. However, the spatial pattern of GLAS acquisition is neither random across the surface of the earth nor is it identifiable with any particular systematic design. Undefined sample properties therefore hinder the use of GLAS in global forest sampling. RESULTS: We propose a method of identifying a subset of the GLAS data which can justifiably be treated as a simple random sample in model-based biomass estimation. The relatively uniform spatial distribution and locally arbitrary positioning of the resulting sample is similar to the design used by the US national forest inventory (NFI). We demonstrated model-based estimation using a sample of GLAS data in the US state of California, where our estimate of biomass (211 Mg/hectare) was within the 1.4% standard error of the design-based estimate supplied by the US NFI. The standard error of the GLAS-based estimate was significantly higher than the NFI estimate, although the cost of the GLAS estimate (excluding costs for the satellite itself) was almost nothing, compared to at least US$ 10.5 million for the NFI estimate. CONCLUSIONS: Global application of model-based estimation using GLAS, while demanding significant consolidation of training data, would improve inter-comparability of international biomass estimates by imposing consistent methods and a globally coherent sample frame. The methods presented here constitute a globally extensible approach for generating a simple random sample from the global GLAS dataset, enabling its use in forest inventory activities.