Evidence map›Paper›PMID 24779731›Full record

Trial reportBiometrics2014

Evaluating marker-guided treatment selection strategies.

Roland A Matsouaka, Junlong Li, Tianxi Cai

Abstract readRandomized Controlled Trial
In one paragraph

Trial report in Biometrics, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
–field-weighted citation impact
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

15 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Machine learning and personalized nutrition: a promising liaison?European journal of clinical nutrition · 2024
    Article
  4. Article
  5. Article
  6. Article
  7. Quantile-Optimal Treatment Regimes.Journal of the American Statistical Association · 2018
    Article
  8. An Algorithm for Generating Individualized Treatment Decision Trees and Random Forests.Journal of computational and graphical statistics : a joint publication of American Statistical Association, Institute of Mathematical Statistics, Interface Foundation of North America · 2018
    Article
  9. Article
  10. Article
  11. Concordance-Assisted Learning for Estimating Optimal Individualized Treatment Regimes.Journal of the Royal Statistical Society. Series B, Statistical methodology · 2017
    Article
  12. On Estimation of Optimal Treatment Regimes For MaximizingJournal of the Royal Statistical Society. Series B, Statistical methodology · 2017
    Article
  13. Article
  14. Article
  15. 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.

Roland A Matsouaka *Department of Epidemiology, Harvard School of Public Health, Boston, Massachusetts 02115, USA.
Junlong Li *Department of Biostatistics, Harvard School of Public Health, Boston, Massachusetts 02115, USA.
Tianxi CaiDepartment of Biostatistics, Harvard School of Public Health, Boston, Massachusetts 02115, USA.

Funding

Informatics for Integrating Biology/ the Bedside (RMI)U54LM008748 · NLM · BRIGHAM AND WOMEN'S HOSPITAL · PI KOHANE, ISAAC S. · 2004 to 2013
$39.0M
Statistical Methods for Optimizing Personalized Treatment SelectionR01HL089778 · NHLBI · STANFORD UNIVERSITY · PI LU TIAN · 2008 to 2026
$4.8M
Training in Neurostatistics and NeuroepidemiologyT32NS048005 · NINDS · HARVARD UNIVERSITY (SCH OF PUBLIC HLTH) · PI ONNELA, JUKKA-PEKKA · 2004 to 2018
$3.2M
Statistical Methods for HIV/AIDS StudiesR01AI052817 · NIAID · HARVARD UNIVERSITY (SCH OF PUBLIC HLTH) · PI WEI, LEE-JEN · 2002 to 2011
$2.5M
Robust Approaches to the Development and Evaluation of Prognostic ClassifiersR01GM079330 · NIGMS · HARVARD SCHOOL OF PUBLIC HEALTH · PI CAI, TIANXI · 2007 to 2014
$1.0M
NHLBI NIH HHS R01 HL089778NIAID NIH HHS R01 AI052817NIGMS NIH HHS R01 GM079330NIGMS NIH HHS R01-GM079330NINDS NIH HHS T32 NS048005NLM NIH HHS U54 LM008748
6 · The paper itself

Abstract

A potential venue to improve healthcare efficiency is to effectively tailor individualized treatment strategies by incorporating patient level predictor information such as environmental exposure, biological, and genetic marker measurements. Many useful statistical methods for deriving individualized treatment rules (ITR) have become available in recent years. Prior to adopting any ITR in clinical practice, it is crucial to evaluate its value in improving patient outcomes. Existing methods for quantifying such values mainly consider either a single marker or semi-parametric methods that are subject to bias under model misspecification. In this article, we consider a general setting with multiple markers and propose a two-step robust method to derive ITRs and evaluate their values. We also propose procedures for comparing different ITRs, which can be used to quantify the incremental value of new markers in improving treatment selection. While working models are used in step I to approximate optimal ITRs, we add a layer of calibration to guard against model misspecification and further assess the value of the ITR non-parametrically, which ensures the validity of the inference. To account for the sampling variability of the estimated rules and their corresponding values, we propose a resampling procedure to provide valid confidence intervals for the value functions as well as for the incremental value of new markers for treatment selection. Our proposals are examined through extensive simulation studies and illustrated with the data from a clinical trial that studies the effects of two drug combinations on HIV-1 infected patients.

Indexed as

Data Interpretation, StatisticalAcquired Immunodeficiency SyndromeAnti-HIV AgentsBiomarkersCD4 AntigensDrug CombinationsHumansOutcome Assessment, Health CarePrevalencePrognosisReproducibility of ResultsSensitivity and SpecificityTreatment OutcomeAnti-HIV AgentsBiomarkersCD4 AntigensDrug CombinationsBiomarker‐analysis designCounterfactual outcomePersonalized medicinePerturbation‐resamplingPredictive biomarkersSubgroup analysis

Identifiers

PMID24779731
PMCPMC4213325

What OpenQuestion holds

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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.