Trial reportJournal for immunotherapy of cancer2019
Are tumor size changes predictive of survival for checkpoint blockade based immunotherapy in metastatic melanoma?
Trial report in Journal for immunotherapy of cancer, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 17 papers, 1 of them a synthesis that pooled it.
What it found
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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.
Phase I Study of Single Agent Pembrolizumab (MK-3475) in Patients With Progressive Locally Advanced or Metastatic Carcinoma, Melanoma, and Non-Small Cell Lung Carcinoma (KEYNOTE 001)
Randomized, Phase II Study of Pembrolizumab (MK-3475) Versus Chemotherapy in Patients With Advanced Melanoma (KEYNOTE 002)
A Multicenter, Randomized, Controlled, Three-Arm, Phase III Study to Evaluate the Safety and Efficacy of Two Dosing Schedules of Pembrolizumab (MK-3475) Compared to Ipilimumab in Patients With Advanced Melanoma
Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Deciphering radiological stable disease to immune checkpoint inhibitors.Annals of oncology : official journal of the European Society for Medical Oncology · 2022Pooled it
- Early predictions of response and survival from a tumor dynamics model in patients with recurrent, metastatic head and neck squamous cell carcinoma treated with immunotherapy.CPT: pharmacometrics & systems pharmacology · 2021Trial
- ΔAverage of long and short axis diameter on non-contrast CT is associated with major pathological response to neoadjuvant immunotherapy in resectable non-small cell lung cancer.European radiology · 2026Article
- Evaluating the potential of candidate surrogate endpoints for overall survival in treatment-naive metastatic melanoma treated with anti-PD-1-based regimens with or without anti-CTLA-4 therapy.Journal for immunotherapy of cancer · 2026Article
- Early Tumor Shrinkage and Clinical Outcomes for Esophageal Squamous Cell Carcinoma Patients Treated with Immune Checkpoint Inhibitors: Real-World Observational Study.Annals of surgical oncology · 2026Observational
- Early radiological response pattern predicts long-term response in patients with non-small cell lung cancer treated with immune-checkpoint inhibitors.Cancer imaging : the official publication of the International Cancer Imaging Society · 2026Article
- Impact of Tumor Response and Response Duration on Survival Among Participants Receiving Pembrolizumab Plus Chemotherapy as First-Line Therapy for Non-Small-Cell Lung Cancer.Oncology and therapy · 2025Article
- Article
- Predictive markers of response to immune checkpoint inhibitor rechallenge in metastatic non-small cell lung cancer.Exploration of targeted anti-tumor therapy · 2024Article
- Assessing immunotherapy response: going beyond RECIST by integrating early tumor growth kinetics.Frontiers in immunology · 2024Article
- Article
- Comparison of sequential and joint nonlinear mixed effects modeling of tumor kinetics and survival following Durvalumab treatment in patients with metastatic urothelial carcinoma.Journal of pharmacokinetics and pharmacodynamics · 2023Article
- Prediction of overall survival in patients across solid tumors following atezolizumab treatments: A tumor growth inhibition-overall survival modeling framework.CPT: pharmacometrics & systems pharmacology · 2021Article
- Downregulation of Stemness Genes and Induction of Necrosis in Rat LA7 Cancer Stem Cells Induced Tumors Treated with Starved Fibroblasts Culture Supernatant.Reports of biochemistry & molecular biology · 2021Article
- Prognostic impact of early tumor shrinkage and depth of response in patients with microsatellite instability-high metastatic colorectal cancer receiving immune checkpoint inhibitors.Journal for immunotherapy of cancer · 2021Article
- Radiological dynamics and SITC-defined resistance types of advanced melanoma during anti-PD-1 monotherapy: an independent single-blind observational study on an international cohort.Journal for immunotherapy of cancer · 2021Observational
- Early tumor shrinkage identifies long-term disease control and survival in patients with lung cancer treated with atezolizumab.Journal for immunotherapy of cancer · 2020Article
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundIn oncology clinical development, objective response rate, disease control rate and early tumor size changes are commonly used as efficacy metrics for early decision-making. However, for immunotherapy trials, it is unclear whether these early efficacy metrics are still predictive of long-term clinical benefit such as overall survival. The goal of this paper is to identify appropriate early efficacy metrics predictive of overall survival for immunotherapy trials.
methodsBased on several checkpoint blockade based immunotherapy studies in metastatic melanoma, we evaluated the predictive value of early tumor size changes and RECIST-based efficacy metrics at various time points on overall survival. The cut-off values for tumor size changes to predict survival were explored via tree based recursive partitioning and validated by external data. Sensitivity analyses were performed for the cut-offs.
resultsThe continuous tumor size change metric and RECIST-based trichotomized response metric at different landmark time points were found to be statistically significantly associated with overall survival. The predictive values were higher at Week 12 and 18 than those at Week 24. The percentage of tumor size changes appeared to have comparable or lower predictive values than the RECIST-based trichotomized metric, and a cut-off of approximately 10% tumor reduction appeared to be reasonable for predicting survival.
conclusionsAn approximate 10% tumor reduction may be a reasonable cut-off for early decision-making while the RECIST-based efficacy metric remains the primary tool. Early landmark analysis is especially useful for decision making when accrual is fast. Composite response rate (utilizing different weights for PR/CR and SD) may be worth further investigation.
trial registrationClinical trials gov, NCT01295827 , Registered February 15, 2011; NCT01704287 , Registered October 11, 2012; NCT01866319 , Registered May 31, 2013.
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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.