Evidence map›Paper›PMID 41398053›Full record

ArticleNPJ precision oncology2025

Benchmarking progression-free survival ratio as primary endpoint in precision oncology clinical trials.

Federico Nichetti, Jennifer Hüllein, Pauline du Rusquec, Erin Pleasance, Li Chen, Andreas Mock, Peter Horak, Paolo Ambrosini, Simone Rota, Monica Niger and 11 more

Abstract read
In one paragraph

Article in NPJ precision oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. Review
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

21 authors.

Federico Nichetti *Oncology Unit 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy. federico.nichetti@iov.veneto.it.
Jennifer Hüllein *Computational Oncology, Molecular Precision Oncology Program, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Pauline du RusquecDepartment of Drug Development and Innovation (D3i), Institut Curie, Paris, France.
Erin PleasanceCanada's Michael Smith Genome Sciences Centre, BC Cancer, Vancouver, BC, Canada.
Li ChenDivision of Cancer Biostatistics, Department of Internal Medicine, University of Kentucky, Lexington, KY, USA.
Andreas MockInstitute of Pathology, University of Munich (LMU), Munich, Germany.
Peter HorakGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Paolo AmbrosiniMedical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Simone RotaMedical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Monica NigerMedical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Luca AgnelliDepartment of Advanced Diagnostics, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Gabriele TinèBiostatistics for Clinical Research Unit, Epidemiology and Data Science Unit, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milano, Italy.
Dominic EdelmannDivision of Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Christophe Le TourneauDepartment of Drug Development and Innovation (D3i), Institut Curie, Paris, France.
Janessa LaskinDepartment of Medical Oncology, BC Cancer, Vancouver, BC, Canada.
Giancarlo PruneriMedical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Chi WangDivision of Cancer Biostatistics, Department of Internal Medicine, University of Kentucky, Lexington, KY, USA.
Daniel HübschmannComputational Oncology, Molecular Precision Oncology Program, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Filippo de BraudOncology Unit 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.
Stefan Fröhling *German Cancer Consortium (DKTK), Heidelberg, Germany.
Luigi Mariani *Biostatistics for Clinical Research Unit, Epidemiology and Data Science Unit, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milano, Italy.

Funding

University of Kentucky Markey Cancer Center Support Grant ECIA SupplementP30CA177558 · NCI · UNIVERSITY OF KENTUCKY · PI KATHLEEN L. O'CONNOR · 2013 to 2026
$38.3M
Statistical Methods for Identification and Evaluation of Predictive Biomarkers in CancerR21CA284179 · NCI · UNIVERSITY OF KENTUCKY · PI CHEN, LI, WANG, CHI · 2024 to 2024
$389k
NCI NIH HHS P30 CA177558NCI NIH HHS R21 CA284179NCI NIH HHS R21CA284179-01A1
6 · The paper itself

Abstract

Progression Free Survival Ratio (PFSratio), as defined as the ratio between PFS on investigational treatment (PFS2) and PFS on the last prior therapy (PFS1), is a popular endpoint in precision oncology (PO) studies. In this work, five methodologies for PFSratio-based trial analysis (count-based, Kaplan Meier, Kernel-based Kaplan Meier, parametric and midrank) and two for trial design (GBVE and Weibull) are benchmarked. The Kernel-based Kaplan Meier analysis is most recommended, as it handles informative censoring and does not require PFS1/PFS2 distribution assumptions. Sample size and power calculation methods perform best when applied to settings with expected high PFS1/PFS2 correlation and median ratio. Analysis of five clinical trials (MOSCATO 01, WINTHER, MASTER, SHIVA and POG570) from >800 patients revealed an overall weak PFS1/PFS2 correlation (Kendall's τ range 0.17-0.35), and an asymptotically unbiased median S

Identifiers

PMID41398053
PMCPMC12820104

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.