Journal Article DKFZ-2026-01578

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Tree-based exploratory identification of predictive biomarkers in non-randomized data.

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2026
BioMed Central London

BMC medical research methodology 26(1), 144 () [10.1186/s12874-026-02928-8]
 GO

Abstract: The idea of 'stratified medicine' is an important driver of methodological research on the identification of predictive biomarkers. Most methods proposed so far for this purpose have been developed for the use on randomized data only. However, often data from clinical registries or non-randomized studies are the only available data source. For such data, methods for estimation of the average treatment effect are well established. Research on confounder adjustment when investigating the heterogeneity of treatment effects and the variables responsible for this is usually restricted to regression modelling. In this paper, we demonstrate how the predMOB, a tree-based method that specifically searches for predictive factors, can be combined with common strategies for confounder adjustment. In an extensive simulation study, we show that the proposed strategies allow for the correct identification of predictive factors in the presence of confounding. It is demonstrated that the most robust method is a combination of both covariate adjustment and Inverse Probability of Treatment Weighting (IPTW). Applications to the German Breast Cancer Study Group (GBSG) trial 2 and the Acute Myeloid Leukemia Study Group (AMLSG) 16-10 study illustrate these conclusions.

Keyword(s): Confounder adjustment ; Non-randomized data ; PredMOB ; Predictive factors ; Treatment effect modification

Classification:

Note: #EA:C060#LA:C060#

Contributing Institute(s):
  1. Biostatistik (C060)
Research Program(s):
  1. 313 - Krebsrisikofaktoren und Prävention (POF4-313) (POF4-313)

Appears in the scientific report 2026
Database coverage:
Medline ; DOAJ ; Article Processing Charges ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; DOAJ Seal ; Ebsco Academic Search ; Essential Science Indicators ; Fees ; IF < 5 ; JCR ; PubMed Central ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection
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 Record created 2026-06-29, last modified 2026-06-30



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