ArticleNeuro-oncology2015
Combining progression-free survival and overall survival as a novel composite endpoint for glioblastoma trials.
Article in Neuro-oncology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 2 of them syntheses that pooled 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.
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.
Who cites it
13 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Evaluation of two-stage designs of Phase 2 single-arm trials in glioblastoma: a systematic review.BMC medical research methodology · 2022Pooled it
- The clinical trials landscape for glioblastoma: is it adequate to develop new treatments?Neuro-oncology · 2018Pooled it
- The Diagnostic Ability of Follow-Up Imaging Biomarkers after Treatment of Glioblastoma in the Temozolomide Era: Implications from Proton MR Spectroscopy and Apparent Diffusion Coefficient Mapping.BioMed research international · 2015Trial
- Experimental Evaluation of QY-69: A Butyrylcholinesterase Inhibitor with Anti-Glioblastoma Efficacy.Current neuropharmacology · 2026Article
- Novel Clinical Trial Designs in Neuro-Oncology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022Review
- Composite endpoint to evaluate complement inhibition therapy in patients with paroxysmal nocturnal hemoglobinuria.European journal of haematology · 2022Article
- Review
- Review
- Individualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT): A Bayesian Adaptive Platform Trial to Develop Precision Medicines for Patients With Glioblastoma.JCO precision oncology · 2019Article
- Arguing for Adaptive Clinical Trials in Sepsis.Frontiers in immunology · 2018Review
- Unbiased estimation for response adaptive clinical trials.Statistical methods in medical research · 2017Article
- Leveraging molecular datasets for biomarker-based clinical trial design in glioblastoma.Neuro-oncology · 2017Article
- Getting it first versus getting it right: weighing the value of and evidence for progression-free survival as a surrogate endpoint for overall survival in glioblastoma.Neuro-oncology · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
backgroundThe use of auxiliary endpoints may provide efficiencies for clinical trial design, but such endpoints may not have intrinsic clinical relevance or clear linkage to more meaningful endpoints. The purpose of this study was to generate a novel endpoint that considers both overall survival (OS) and earlier events such as progression-free survival (PFS) and determine whether such an endpoint could increase efficiency in the design of glioblastoma clinical trials.
methodsRecognizing that the association between PFS and OS varies depending on therapy and tumor type, we developed a statistical model to predict OS based on PFS as the trial progresses. We then evaluated the efficiency of our model using simulations of adaptively randomized trials incorporating PFS and OS distributions from prior published trials in neuro-oncology.
resultsWhen treatment effects on PFS and OS are concordant, our proposed approach results in efficiency gains compared with randomization based on OS alone while sacrificing minimal efficiency compared with using PFS as the primary endpoint. When treatment effects are limited to PFS, our approach provides randomization probabilities that are close to those based on OS alone.
conclusionUse of OS as the primary endpoint, combined with statistical modeling of the relationship between OS and PFS during the course of the trial, results in more robust and efficient trial designs than using either endpoint alone.
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Registered trials
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.