scholarly journals Sample-size estimation is not reported in 24% of randomised controlled trials of inflammatory bowel disease: A systematic review

2020 ◽  
pp. 205064062096789
Author(s):  
Zipporah Iheozor-Ejiofor ◽  
Svetlana Lakunina ◽  
Morris Gordon ◽  
Daniel Akintelure ◽  
Vasiliki Sinopoulou ◽  
...  
Keyword(s):  
Inflammatory Bowel Disease ◽  
Sample Size ◽  
Systematic Reviews ◽  
Bowel Disease ◽  
Randomised Controlled Trials ◽  
Sample Size Estimation ◽  
Controlled Trials ◽  
Size Estimation ◽  
Inflammatory Bowel ◽  
Randomised Controlled

Background Sample-size estimation is an important factor in designing a clinical trial. A recent study found that 65% of Cochrane systematic reviews had imprecise results. Objective This study set out to review the whole body of inflammatory bowel disease (IBD) randomised controlled trials systematically in order to identify the reporting of sample-size estimation. Methods We conducted a comprehensive hand search of the Cochrane Library and Cochrane IBD Specialized Trials Register. We extracted information on relevant features and the results of the included studies. We produced descriptive statistics for our results. Results A total of 242 randomised controlled trials were included from 44 Cochrane systematic reviews. About 25% of the studies failed to report on sample-size estimation. Of those that did report on sample-size estimation, 33% failed to recruit their target sample size. Conclusions Around half of the randomised controlled trials in IBD either do not report sample-size estimation or reach their recruitment target with the level of detail in reporting being limited.

P062 A SYSTEMATIC REVIEW ON POWER ESTIMATION TO SUPPORT SAMPLE SIZE CALCULATION FOR RANDOMISED TRIALS IN INFLAMMATORY BOWEL DISEASE

2020 ◽  
Vol 26 (Supplement_1) ◽  
pp. S9-S9
Author(s):  
Svetlana Lakunina ◽  
Zipporah Iheozor-Ejiofor ◽  
Morris Gordon ◽  
Daniel Akintelure ◽  
Vassiliki Sinopoulou
Keyword(s):  
Inflammatory Bowel Disease ◽  
Sample Size ◽  
Bowel Disease ◽  
Sample Size Calculation ◽  
Sample Size Estimation ◽  
Power Calculation ◽  
Size Estimation ◽  
Target Sample ◽  
Inflammatory Bowel ◽  
Randomised Controlled

Abstract Inflammatory bowel disease is a collection of disorders of the gastrointestinal tract, characterised by relapsing and remitting inflammation. Studies have reported several pharmacological or non-pharmacological interventions being effective in the management of the disease. Sample size estimation with power calculation is necessary for a trial to detect the effect of an intervention. This project critically evaluates the sample size estimation and power calculation reported by randomised controlled studies of inflammatory bowel disease management to effectively conclude appropriateness of the studies results. We conducted a literature search in the Cochrane database to identify systematic literature reviews. Their reference lists were screened, and studies were selected if they met the inclusion criteria. The data was extracted based on power calculation parameters and outcomes, results were analysed and summarised in percentages, means and graphs. We screened almost all trials about the management of inflammatory bowel disease published in the past 25 years. 232 studies were analysed, of which 167 reported power calculation. Less than half (48%) of these studies achieved their target sample size, needed for them to accurately conclude that the interventions were effective. Moreover, the average minimal difference those studies were aimed to detect was 30%, which could be not enough to prove the effect of an intervention. To conclude inaccurate power calculations and failure to achieve the target sample sizes can lead to errors in the results on how effective an intervention is in the management of inflammatory bowel disease.

scholarly journals Correction to: Sample size estimation for randomised controlled trials with repeated assessment of patient-reported outcomes: what correlation between baseline and follow-up outcomes should we assume?

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Stephen J. Walters ◽  
Richard M. Jacques ◽  
Inês Bonacho dos Anjos Henriques-Cadby ◽  
Jane Candlish ◽  
Nikki Totton ◽  
...  
Keyword(s):  
Sample Size ◽  
Randomised Controlled Trials ◽  
Patient Reported Outcomes ◽  
Sample Size Estimation ◽  
Controlled Trials ◽  
Size Estimation ◽  
Patient Reported ◽  
Randomised Controlled ◽  
Repeated Assessment

Following publication of the original article [1], we have been notified that one of an error in the Conclusions section of the Abstract.

scholarly journals Sample size estimation for randomised controlled trials with repeated assessment of patient-reported outcomes: what correlation between baseline and follow-up outcomes should we assume?

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Stephen J. Walters ◽  
Richard M. Jacques ◽  
Inês Bonacho dos Anjos Henriques-Cadby ◽  
Jane Candlish ◽  
Nikki Totton ◽  
...  
Keyword(s):  
Sample Size ◽  
Randomised Controlled Trials ◽  
Sample Size Estimation ◽  
Controlled Trials ◽  
Size Estimation ◽  
Time Points ◽  
Patient Reported ◽  
Randomised Controlled ◽  
Post Randomisation

Abstract Background Patient-reported outcome measures (PROMs) are now frequently used in randomised controlled trials (RCTs) as primary endpoints. RCTs are longitudinal, and many have a baseline (PRE) assessment of the outcome and one or more post-randomisation assessments of outcome (POST). With such pre-test post-test RCT designs there are several ways of estimating the sample size and analysing the outcome data: analysis of post-randomisation treatment means (POST); analysis of mean changes from pre- to post-randomisation (CHANGE); analysis of covariance (ANCOVA). Sample size estimation using the CHANGE and ANCOVA methods requires specification of the correlation between the baseline and follow-up measurements. Other parameters in the sample size estimation method being unchanged, an assumed correlation of 0.70 (between baseline and follow-up outcomes) means that we can halve the required sample size at the study design stage if we used an ANCOVA method compared to a comparison of POST treatment means method. So what correlation (between baseline and follow-up outcomes) should be assumed and used in the sample size calculation? The aim of this paper is to estimate the correlations between baseline and follow-up PROMs in RCTs. Methods The Pearson correlation coefficients between the baseline and repeated PROM assessments from 20 RCTs (with 7173 participants at baseline) were calculated and summarised. Results The 20 reviewed RCTs had sample sizes, at baseline, ranging from 49 to 2659 participants. The time points for the post-randomisation follow-up assessments ranged from 7 days to 24 months; 464 correlations, between baseline and follow-up, were estimated; the mean correlation was 0.50 (median 0.51; standard deviation 0.15; range − 0.13 to 0.91). Conclusions There is a general consistency in the correlations between the repeated PROMs, with the majority being in the range of 0.4 to 0.6. The implications are that we can reduce the sample size in an RCT by 25% if we use an ANCOVA model, with a correlation of 0.50, for the design and analysis. There is a decline in correlation amongst more distant pairs of time points.

Application of Ultrasound Elastography for Assessing Intestinal Fibrosis in Inflammatory Bowel Disease: Fiction or Reality?

2020 ◽  
Vol 21 ◽  
Author(s):  
Roberto Gabbiadini ◽  
Eirini Zacharopoulou ◽  
Federica Furfaro ◽  
Vincenzo Craviotto ◽  
Alessandra Zilli ◽  
...  
Keyword(s):  
Inflammatory Bowel Disease ◽  
Sample Size ◽  
Bowel Disease ◽  
Small Sample Size ◽  
Shear Wave Elastography ◽  
Small Sample ◽  
Ultrasound Elastography ◽  
Invasive Technique ◽  
Intestinal Fibrosis ◽  
Inflammatory Bowel

Background: Intestinal fibrosis and subsequent strictures represent an important burden in inflammatory bowel disease (IBD). The detection and evaluation of the degree of fibrosis in stricturing Crohn’s disease (CD) is important to address the best therapeutic strategy (medical anti-inflammatory therapy, endoscopic dilation, surgery). Ultrasound elastography (USE) is a non-invasive technique that has been proposed in the field of IBD for evaluating intestinal stiffness as a biomarker of intestinal fibrosis. Objective: The aim of this review is to discuss the ability and current role of ultrasound elastography in the assessment of intestinal fibrosis. Results and Conclusion: Data on USE in IBD are provided by pilot and proof-of-concept studies with small sample size. The first type of USE investigated was strain elastography, while shear wave elastography has been introduced lately. Despite the heterogeneity of the methods of the studies, USE has been proven to be able to assess intestinal fibrosis in patients with stricturing CD. However, before introducing this technique in current practice, further studies with larger sample size and homogeneous parameters, testing reproducibility, and identification of validated cut-off values are needed.

scholarly journals Patient-Reported Outcomes in Inflammatory Bowel Disease: A Measurement of Effect in Research and Clinical Care

2021 ◽  
Vol 12 (2) ◽  
pp. 225-237
Author(s):  
Jane Fletcher ◽  
Sheldon C. Cooper ◽  
Amelia Swift
Keyword(s):  
Inflammatory Bowel Disease ◽  
Systematic Reviews ◽  
Bowel Disease ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Health Measurement ◽  
Outcome Set ◽  
Patient Reported ◽  
Inflammatory Bowel ◽  
Selection Of

The measurement of outcomes is key in evaluating healthcare or research interventions in inflammatory bowel disease (IBD). In patient-centred care, patient-reported outcome measures (PROMs) are central to this evaluation. In this review, we provide an overview of validated, adult disease-specific PROMs developed for use in IBD. Our aim is to assist clinicians and researchers in selection of PROMs to measure outcomes in their patient cohort. The Consensus-based Standards for the Selection of Health Measurement Instruments database of systematic reviews was the primary resource used to identify PROMs used in IBD. Search terms were ‘Crohn’s disease’, ‘ulcerative colitis’, and ‘IBD’. Seven systematic reviews were identified from this search. In addition, the publication by the IBD Core Outcome Set Working Group was used to identify further PROMs. Three systematic reviews were excluded as they did not meet the inclusion criteria. From the five included systematic reviews, we identified 21 PROMs and their shortened versions. In conclusion, it does not appear that any one PROM is entirely suitable for both research and clinical practice. Overall, the IBDQ-32 is most widely used in research but has the limitation of cost, whereas the IBD-Control has been recommended in the clinical core outcome set.

scholarly journals Head-to-head comparison of biological drugs for inflammatory bowel disease: from randomized controlled trials to real-world experience

2021 ◽  
Vol 14 ◽  
pp. 175628482110106
Author(s):  
Fabio Salvatore Macaluso ◽  
Marcello Maida ◽  
Mauro Grova ◽  
Federica Crispino ◽  
Giulia Teresi ◽  
...  
Keyword(s):  
Inflammatory Bowel Disease ◽  
Randomized Controlled Trials ◽  
Real World ◽  
Bowel Disease ◽  
Quality Data ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Therapeutic Algorithms ◽  
Inflammatory Bowel

During past years, the increasing knowledge of molecular mechanisms of inflammatory bowel disease (IBD) have led to the development of several targeted biological therapies. This great expansion of available medical options has prompted the need for comparative data between drugs. For years, given that most randomized controlled trials (RCTs) were performed only versus placebo, this demand has clashed with the absence of head-to-head trials comparing two or more treatments. The quality of evidence coming from real-world experience was low overall, so it was extremely difficult to clarify the correct positioning of the biologicals inside the therapeutic algorithms for IBD. Fortunately, times are changing: head-to-head comparative RCTs have been conducted or are ongoing, and the methodological quality of real-world studies is gradually increasing, mainly thanks to a higher rate of application of statistical methods capable of reducing the selection bias, such as the propensity score. In this evolving scenario, the increasing number of comparative RCTs is providing high-quality data for a correct drug positioning in IBD. In parallel, real-world observational studies are supporting the data coming from RCTs, and covering those comparisons not performed in the RCT setting. We believe that there is moderate evidence already available to support clinicians in the correct choice between different biologicals, and data will certainly be more robust in the near future.

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