Evidence map›Paper›PMID 35085831›Full record

ArticleContemporary clinical trials2022

A likely responder approach for the analysis of randomized controlled trials.

Eugene Laska, Carole Siegel, Ziqiang Lin

Open access · hybridAbstract read
In one paragraph

Article in Contemporary clinical trials, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.0field-weighted citation impact, top 25% 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

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Article
  3. 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

3 authors at 1 institution in 1 country.

Eugene LaskaDepartment of Psychiatry, New York University Grossman School of Medicine, One Park Avenue, New York, NY 10016, USA; Department of Population Health, Division of Biostatistics, NYU Grossman School of Medicine, 180 Madison Avenue, New York, NY 10016, USA. Electronic address: eugene.laska@nyumc.org.
Carole SiegelDepartment of Psychiatry, New York University Grossman School of Medicine, One Park Avenue, New York, NY 10016, USA; Department of Population Health, Division of Biostatistics, NYU Grossman School of Medicine, 180 Madison Avenue, New York, NY 10016, USA. Electronic address: carole.siegel@nyumc.org.
Ziqiang LinDepartment of Psychiatry, New York University Grossman School of Medicine, One Park Avenue, New York, NY 10016, USA. Electronic address: ziqiang.lin@nyumc.org.
New York University · US

Funding

Topiramate as a treatment for Co-occurring AUD and PTSDP01AA027057 · NIAAA · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · PI MARMAR, CHARLES R. · 2018 to 2024
$6.4M
NIAAA NIH HHS P01 AA027057
6 · The paper itself

Abstract

objectiveTo further the precision medicine goal of tailoring medical treatment to individual patient characteristics by providing a method of analysis of the effect of test treatment, T, compared to a reference treatment, R, in participants in a RCT who are likely responders to T.

methodsLikely responders to T are individuals whose expected response at baseline exceeds a prespecified minimum. A prognostic score, the expected response predicted as a function of baseline covariates, is obtained at trial completion. It is a balancing score that can be used to match likely responders randomized to T with those randomized to R; the result is comparable treatment groups that have a common covariance distribution. Treatments are compared based on observed outcomes in this enriched sample. The approach is illustrated in a RCT comparing two treatments for opioid use disorder.

resultsA standard statistical analysis of the opioid use disorder RCT found no treatment difference in the total sample. However, a subset of likely responders to T were identified and in this group, T was statistically superior to R.

conclusionThe causal treatment effect of T relative to R among likely responders may be more important than the effect in the whole target population. The prognostic score function provides quantitative information to support patient specific treatment decisions regarding T furthering the goal of precision medicine.

Indexed as

Precision MedicineResearch DesignHumansRandomized Controlled Trials as TopicCausal inferenceEnriched sampleLikely respondersPrecision medicinePrognostic balancing score

Identifiers

PMID35085831
PMCPMC8934276
OpenAlexW4207003135

What OpenQuestion holds

Textmetadata
LicenceTDM
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 OpenQuestion graph.