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Prior Elicitation for Use in Clinical Trial Design and Analysis: A Literature Review.

ABSTRACT: Bayesian inference is increasingly popular in clinical trial design and analysis. The subjective knowledge derived from an expert elicitation procedure may be useful to define a prior probability distribution when no or limited data is available. This work aims to investigate the state-of-the-art Bayesian prior elicitation methods with a focus on clinical trial research. A literature search on the Current Index to Statistics (CIS), PubMed, and Web of Science (WOS) databases, considering "prior elicitation" as a search string, was run on 1 November 2020. Summary statistics and trend of publications over time were reported. Finally, a Latent Dirichlet Allocation (LDA) model was developed to recognise latent topics in the pertinent papers retrieved. A total of 460 documents pertinent to the Bayesian prior elicitation were identified. Of these, 213 (45.4%) were published in the "Probability and Statistics" area. A total of 42 articles pertain to clinical trial and the majority of them (81%) reports parametric techniques as elicitation method. The last decade has seen an increased interest in prior elicitation and the gap between theory and application getting narrower and narrower. Given the promising flexibility of non-parametric approaches to the experts' elicitation, more efforts are needed to ensure their diffusion also in applied settings.

SUBMITTER: Azzolina D

PROVIDER: S-EPMC7917693 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature

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Prior Elicitation for Use in Clinical Trial Design and Analysis: A Literature Review.

Azzolina Danila D Berchialla Paola P Gregori Dario D Baldi Ileana I

International journal of environmental research and public health 20210213 4

Bayesian inference is increasingly popular in clinical trial design and analysis. The subjective knowledge derived from an expert elicitation procedure may be useful to define a prior probability distribution when no or limited data is available. This work aims to investigate the state-of-the-art Bayesian prior elicitation methods with a focus on clinical trial research. A literature search on the Current Index to Statistics (CIS), PubMed, and Web of Science (WOS) databases, considering "prior e ...[more]

PMID: 33668623

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Project description:BackgroundChronic Achilles Tendinopathy is a common condition that can be challenging to treat. There are many modalities used as treatment ranging from physiotherapy, injections, shockwave therapy to surgical intervention. Platelet Rich Plasma (PRP) has increased in popularity recently as a treatment option for Achilles Tendinopathy. It contains growth factors that might accelerate healing and speed up the recovery of this condition. Many studies have been conducted in the last few years to assess the effectiveness of this treatment method. However, there was controversy as to whether PRP had a beneficial effect on chronic Achille tendinopathy.AimThis systematic review of the literature was conducted to ascertain the efficacy of Platelet Rich Plasma (PRP) as a treatment option in chronic Achilles tendinopathy.MethodsPRISMA reporting item for systematic review has been used to conduct the selection, Electronic databases included PubMed, EMBASE, Cochrane collaborate, Google scholar, the web of science and Cochrane Library were searched for all RCT, prospective and retrospective studies conducted between January 2010 to February 2019. The quality of each study was evaluated using the Oxford CEBM tool to assess the articles for validity, relevance, and applicability of the results. A total number of 288 were found, and only 11 met the inclusion criteria.Results5 Randomised control trials, 4 prospective and 2 retrospective cohort study were included in this systematic review. A total number of 406 patients were treated for non-insertional Achilles tendinopathy of which 230 patients had PRP local injection under Ultrasound guide.ConclusionAlthough many of the retrospective studies suggested an advantage of using PRP, the higher level of evidence studies do not support a significant efficacy. This systematic review showed very promising results from the use of Platelet Rich Plasma demonstrated by a significant improvement in the VAST-A score, but we certainly need decent size randomised control trials to show better and more accurate results.

Project description:BackgroundPatients with follicular lymphoma (FL) can have high response rates to early lines of treatment. However, among FL patients relapsed/refractory (r/r) after ≥2 prior lines of therapy (LOT), remission tends to be shorter and there is limited treatment guidance. This study sought to evaluate the clinical outcomes for r/r FL after ≥2 prior LOT identified through systematic literature review.MethodsEligible studies included comparative or non-comparative interventional or observational studies of systemic therapies among adults with FL r/r after ≥2 prior LOT published prior to 31st May 2021. Prior LOT must have included an anti-CD20 monoclonal antibody and an alkylating agent, in combination or separately. Overall response rate (ORR) and complete response (CR) were estimated using inverse-variance weighting with Freeman-Tukey double-arcsine transformations. Kaplan-Meier (KM) curves for progression-free survival (PFS) and overall survival (OS) estimated by reconstructing digitized curves using the Guyot algorithm, and survival analyses were conducted, stratified by ≥2 prior LOT and ≥ 3 prior LOT groups (as defined in the source material). Restricting the analyses to the observational cohorts was investigated as a sensitivity analysis.ResultsThe analysis-set included 20 studies published between 2014 and 2021. Studies were primarily US and/or European based, with the few exceptions using treatments approved in US/Europe. The estimated ORR was 58.47% (95% confidence interval [CI]: 51.13-65.62) and proportion of patients with CR was 19.63% (95% CI: 15.02-24.68). The median OS among those ≥2 prior LOT was 56.57 months (95% CI: 47.8-68.78) and median PFS was 9.78 months (95% CI: 9.01-10.63). The 24-month OS decreased from 66.50% in the ≥2 prior LOT group to 59.51% in the ≥3 prior LOT group, with a similar trend in PFS at 24-month (28.42% vs 24.13%).ConclusionsThis study found that few r/r FL patients with ≥2 prior LOT achieve CR, and despite some benefit, approximately 1/3 of treated patients die within 24 months. The shorter median PFS with increasing prior LOT suggest treatment durability is suboptimal in later LOT. These findings indicate that patients are underserved by treatments currently available in the US and Europe.

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Prior Elicitation for Use in Clinical Trial Design and Analysis: A Literature Review.

Publications

Prior Elicitation for Use in Clinical Trial Design and Analysis: A Literature Review.

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