Evidence map›Paper›PMID 37965484›Full record

ArticleContemporary clinical trials communications2023

Conducting a bayesian multi-armed trial with response adaptive randomization for comparative effectiveness of medications for CSPN.

Alexandra R Brown, Byron J Gajewski, Dinesh Pal Mudaranthakam, Mamatha Pasnoor, Mazen M Dimachkie, Omar Jawdat, Laura Herbelin, Matthew S Mayo, Richard J Barohn

Open access · goldAbstract read
In one paragraph

Article in Contemporary clinical trials communications, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 36% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 1 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 2 institutions in 1 country.

Alexandra R BrownDepartment of Biostatistics & Data Science, The University of Kansas Medical Center, Kansas City, KS, USA.
Byron J GajewskiDepartment of Biostatistics & Data Science, The University of Kansas Medical Center, Kansas City, KS, USA.
Dinesh Pal MudaranthakamDepartment of Biostatistics & Data Science, The University of Kansas Medical Center, Kansas City, KS, USA.
Mamatha PasnoorDepartment of Neurology, The University of Kansas Medical Center, Kansas City, KS, USA.
Mazen M DimachkieDepartment of Neurology, The University of Kansas Medical Center, Kansas City, KS, USA.
Omar JawdatDepartment of Neurology, The University of Kansas Medical Center, Kansas City, KS, USA.
Laura HerbelinDepartment of Neurology, The University of Kansas Medical Center, Kansas City, KS, USA.
Matthew S MayoDepartment of Biostatistics & Data Science, The University of Kansas Medical Center, Kansas City, KS, USA.
Richard J BarohnDepartment of Neurology, The University of Missouri School of Medicine, Columbia, MO, USA.
University of Kansas Medical Center · USUniversity of Missouri · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Response adaptive randomization is popular in adaptive trial designs, but the literature detailing its execution is lacking. These designs are desirable for patients/stakeholders, particularly in comparative effectiveness research, due to the potential benefits including improving participant buy-in by providing more participants with better treatment during the trial. Frequentist approaches have often been used, but adaptive designs naturally fit the Bayesian methodology; it was developed to deal with data as they come in by updating prior information. Methods: PAIN-CONTRoLS was a comparative-effectiveness trial utilizing Bayesian response adaptive randomization to four drugs, nortriptyline, duloxetine, pregabalin, or mexiline, for cryptogenic sensory polyneuropathy (CSPN) patients. The aim was to determine which treatment was most tolerable and effective in reducing pain. Quit and efficacy rates were combined into a utility function to develop a single outcome, which with treatment sample size, drove the adaptive randomization. Prespecified interim analyses allowed the study to stop for early success or update the randomization probabilities to the better-performing treatments. Results: Seven adaptations to the randomization occurred before the trial ended due to reaching the maximum sample size, with more participants receiving nortriptyline and duloxetine. At the end of the follow-up, nortriptyline and duloxetine had lower probabilities of participants that had stopped taking the study medication and higher probabilities were efficacious. Mexiletine had the highest quit rate, but had an efficacy rate higher than pregabalin. Conclusions: Response adaptive randomization has become a popular trial tool, especially for those utilizing Bayesian methods for analyses. By illustrating the execution of a Bayesian adaptive design, using the PAIN-CONTRoLS trial data, this paper continues the work to provide literature for conducting Bayesian response adaptive randomized trials.

Indexed as

Bayesian methodsConducting adaptive designInterim analysesOutcome driven

Identifiers

PMID37965484
PMCPMC10641102
OpenAlexW4387641133

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.