Sample size determination is often an important step in planning a statistical study—and it is usually a difficult one. Among the important hurdles to be surpassed, one must obtain an estimate of one or more error variances and specify an effect size of importance. There is the temptation to take some shortcuts. This article offers some suggestions for successful and meaningful sample size determination. Also discussed is the possibility that sample size may not be the main issue, that the real goal is to design a high-quality study. Finally, criticism is made of some ill-advised shortcuts relating to power and sample size.

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Some Practical Guidelines for Effective Sample Size Determination

Cross-sectional positron emission tomography (PET) studies find that cognitively normal carriers of the apolipoprotein E (APOE) epsilon4 allele, a common Alzheimer's susceptibility gene, have abnormally low measurements of the cerebral metabolic rate for glucose (CMRgl) in the same regions as patients with Alzheimer's dementia. In this article, we characterize longitudinal CMRgl declines in cognitively normal epsilon4 heterozygotes, estimate the power of PET to test the efficacy of treatments to attenuate these declines in 2 years, and consider how this paradigm could be used to efficiently test the potential of candidate therapies for the prevention of Alzheimer's disease. We studied 10 cognitively normal epsilon4 heterozygotes and 15 epsilon4 noncarriers 50-63 years of age with a reported family history of Alzheimer's dementia before and after an interval of approximately 2 years. The epsilon4 heterozygotes had significant CMRgl declines in the vicinity of temporal, posterior cingulate, and prefrontal cortex, basal forebrain, parahippocampal gyrus, and thalamus, and these declines were significantly greater than those in the epsilon4 noncarriers. In testing candidate primary prevention therapies, we estimate that between 50 and 115 cognitively normal epsilon4 heterozygotes are needed per active and placebo treatment group to detect a 25% attenuation in these CMRgl declines with 80% power and P = 0.005 in 2 years. Assuming these CMRgl declines are related to the predisposition to Alzheimer's dementia, this study provides a paradigm for testing the potential of treatments to prevent the disorder without having to study thousands of research subjects or wait many years to determine whether or when treated individuals develop symptoms.

Declining brain activity in cognitively normal apolipoprotein E epsilon 4 heterozygotes: A foundation for using positron emission tomography to efficiently test treatments to prevent Alzheimer's disease.

The authors have prospectively examined the occurrence of postoperative wound infection following clean neurosurgery in 936 patients. Fewer than 1% received perioperative antibiotic prophylaxis. The overall rate of deep wound infection was 2.6%; no deaths were directly attributable to these infections. Deep wound infections occurred significantly more frequently following craniotomy (4.3%) than following spinal (0.9%) or other clean neurosurgery. Among craniotomies, the deep wound infection rate varied significantly from 11% following repeat operations for recurrent gliomas to 2.5% following non-tumor surgery. Risk of deep wound infection varied more than 11-fold depending on the type of clean neurosurgical operation. It is most feasible to demonstrate the potential efficacy of perioperative antibiotics in clean neurosurgical procedures with the greatest risk of postoperative wound infection. The potential benefit from such prophylaxis would be greatest for patients undergoing these high-risk operations.

Wide variation in risk of wound infection following clean neurosurgery. Implications for perioperative antibiotic prophylaxis.

The expression and report of pain is influenced by social environment and culture. Previous studies have suggested ethnically determined differences in report of pain threshold, intensity and affect. The influence of ethnic differences between White British and South Asians has remained unexplored. Twenty age-matched, male volunteers in each group underwent evaluation. Cold and warm perception and cold and heat threshold were assessed using an ascending method of limits. Magnitude estimation of pain unpleasantness and pain intensity were investigated with thermal stimuli of 46, 47, 48 and 49 degrees C. Subjects also completed a pain anxiety questionnaire. Data was analysed using t-test, Mann-Whitney and repeated measures analysis of variance as appropriate. There were no differences in cold and warm perception between the two groups. There was a statistically significant difference between the two groups for heat pain threshold (P=0.006) and heat pain intensity demonstrated a significant effect for ethnicity (F=13.84, P=0.001). Although no group differences emerged for cold pain threshold and heat unpleasantness, South Asians demonstrated lower cold pain threshold and reported more unpleasantness at all temperatures but this was not statistically significant. Our study shows that ethnicity plays an important role in heat pain threshold and pain report, South Asian males demonstrated lower pain thresholds and higher pain report when compared with matched White British males. There were no differences in pain anxiety between the two groups and no correlations were identified between pain and pain anxiety Haemodynamic measures and anthropometry did not explain group differences.

Ethnic differences in thermal pain responses: A comparison of South Asian and White British healthy males

Sample size determination is one of the central tenets of medical research. If the sample size is inadequate, then the study will fail to detect a real difference between the effects of two clinical approaches. On the contrary, if the sample size is larger than what is needed, the study will become cumbersome and ethically prohibitive. Apart from this, the study will become expensive, time consuming and will have no added advantages. A study which needs a large sample size to prove any significant difference in two treatments must ensure the appropriate sample size. It is better to terminate such a study when the required sample size cannot be attained so that the funds and manpower can be conserved. When dealing with multiple sub-groups in a population the sample size should be increased the adequate level for each sub-group. To ensure the reliability of final comparison of the result, the significant level and power must be fixed before the sample size determination. Sample size determination is very important and always a difficult process to handle. It requires the collaboration of a specialist who has good scientific knowledge in the art and practice of medical statistics. A few suggestions are made in this paper regarding the methods to determine an optimum sample size in descriptive and analytical studies.

Relevance of Sample Size Determination in Medical Research

Sample-size determination

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Sample-size determination