RECIST 1.1 for Response Evaluation Apply Not Only to Chemotherapy-Treated Patients But Also to Targeted Cancer Agents: A Pooled Database Analysis

RECIST working group

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Abstract

PURPOSE: The mode of action of targeted cancer agents (TCAs) differs from classic chemotherapy, which leads to concerns about the role of RECIST in evaluating tumor response in trials with TCAs. We investigated the performance of RECIST using a pooled database from 50 clinical trials with at least one TCA. METHODS: We examined the impact of the number of target lesions (TLs) on within-patient variability of tumor response. The prognostic effect of TL response (at 12 weeks or on study on the basis of a maximum five TLs) on survival was studied through landmark and time-dependent Cox models adjusted for baseline tumor load, occurrence of new lesions, or unequivocal progression of nontarget disease. RESULTS: Data were obtained from 23,259 patients with cancer (36% lung, 28% colorectal, 11% breast, and 25% other); 15,620 received TCAs, predominantly transduction or angiogenesis inhibitors, as a single agent (37%), combined with other TCAs (7%), or as chemotherapy (56%); 28% received chemotherapy only; and 5% received best supportive care or placebo. A total of 17,222 patients contributed to the analyses. Within-patient variability decreased with increasing number of TLs, similarly for TCAs (with/without chemotherapy) and chemotherapy only. Mixed responses occurred proportionally in all treatment classes. Landmark analyses showed an ordinal relationship between percentage change from baseline to 12 weeks and overall survival, and demonstrated a clear distinction between tumor shrinkage and progressive disease according to RECIST. Time-dependent analysis showed no marked improvement in the ability to predict survival on the basis of TL tumor growth compared with nontarget progression or new lesion occurrence, regardless of treatment. Similar results were seen for major tumor types and different classes of TCAs. CONCLUSION: This work reinforces that RECIST version 1.1 perform well for response assessment of TCAs.

Original languageEnglish (US)
Pages (from-to)1102-1110
Number of pages9
JournalJournal of clinical oncology : official journal of the American Society of Clinical Oncology
Volume37
Issue number13
DOIs
StatePublished - May 1 2019

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Databases
Drug Therapy
Neoplasms
Response Evaluation Criteria in Solid Tumors
Survival
Angiogenesis Inhibitors
Tumor Burden
Proportional Hazards Models
Disease Progression
Lung Neoplasms
Breast
Placebos
Clinical Trials
Therapeutics
Growth

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

@article{78c8cdcf10eb478c85d2f23aa1c0bb32,
title = "RECIST 1.1 for Response Evaluation Apply Not Only to Chemotherapy-Treated Patients But Also to Targeted Cancer Agents: A Pooled Database Analysis",
abstract = "PURPOSE: The mode of action of targeted cancer agents (TCAs) differs from classic chemotherapy, which leads to concerns about the role of RECIST in evaluating tumor response in trials with TCAs. We investigated the performance of RECIST using a pooled database from 50 clinical trials with at least one TCA. METHODS: We examined the impact of the number of target lesions (TLs) on within-patient variability of tumor response. The prognostic effect of TL response (at 12 weeks or on study on the basis of a maximum five TLs) on survival was studied through landmark and time-dependent Cox models adjusted for baseline tumor load, occurrence of new lesions, or unequivocal progression of nontarget disease. RESULTS: Data were obtained from 23,259 patients with cancer (36{\%} lung, 28{\%} colorectal, 11{\%} breast, and 25{\%} other); 15,620 received TCAs, predominantly transduction or angiogenesis inhibitors, as a single agent (37{\%}), combined with other TCAs (7{\%}), or as chemotherapy (56{\%}); 28{\%} received chemotherapy only; and 5{\%} received best supportive care or placebo. A total of 17,222 patients contributed to the analyses. Within-patient variability decreased with increasing number of TLs, similarly for TCAs (with/without chemotherapy) and chemotherapy only. Mixed responses occurred proportionally in all treatment classes. Landmark analyses showed an ordinal relationship between percentage change from baseline to 12 weeks and overall survival, and demonstrated a clear distinction between tumor shrinkage and progressive disease according to RECIST. Time-dependent analysis showed no marked improvement in the ability to predict survival on the basis of TL tumor growth compared with nontarget progression or new lesion occurrence, regardless of treatment. Similar results were seen for major tumor types and different classes of TCAs. CONCLUSION: This work reinforces that RECIST version 1.1 perform well for response assessment of TCAs.",
author = "{RECIST working group} and Saskia Liti{\`e}re and Ga{\"e}lle Isaac and {De Vries}, {Elisabeth G.E.} and Jan Bogaerts and Alice Chen and Janet Dancey and Robert Ford and Stephen Gwyther and Otto Hoekstra and Erich Huang and Nancy Lin and Yan Liu and Mandrekar, {Sumithra J} and Schwartz, {Lawrence H.} and Lalitha Shankar and Patrick Therasse and Lesley Seymour",
year = "2019",
month = "5",
day = "1",
doi = "10.1200/JCO.18.01100",
language = "English (US)",
volume = "37",
pages = "1102--1110",
journal = "Journal of Clinical Oncology",
issn = "0732-183X",
publisher = "American Society of Clinical Oncology",
number = "13",

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TY - JOUR

T1 - RECIST 1.1 for Response Evaluation Apply Not Only to Chemotherapy-Treated Patients But Also to Targeted Cancer Agents

T2 - A Pooled Database Analysis

AU - RECIST working group

AU - Litière, Saskia

AU - Isaac, Gaëlle

AU - De Vries, Elisabeth G.E.

AU - Bogaerts, Jan

AU - Chen, Alice

AU - Dancey, Janet

AU - Ford, Robert

AU - Gwyther, Stephen

AU - Hoekstra, Otto

AU - Huang, Erich

AU - Lin, Nancy

AU - Liu, Yan

AU - Mandrekar, Sumithra J

AU - Schwartz, Lawrence H.

AU - Shankar, Lalitha

AU - Therasse, Patrick

AU - Seymour, Lesley

PY - 2019/5/1

Y1 - 2019/5/1

N2 - PURPOSE: The mode of action of targeted cancer agents (TCAs) differs from classic chemotherapy, which leads to concerns about the role of RECIST in evaluating tumor response in trials with TCAs. We investigated the performance of RECIST using a pooled database from 50 clinical trials with at least one TCA. METHODS: We examined the impact of the number of target lesions (TLs) on within-patient variability of tumor response. The prognostic effect of TL response (at 12 weeks or on study on the basis of a maximum five TLs) on survival was studied through landmark and time-dependent Cox models adjusted for baseline tumor load, occurrence of new lesions, or unequivocal progression of nontarget disease. RESULTS: Data were obtained from 23,259 patients with cancer (36% lung, 28% colorectal, 11% breast, and 25% other); 15,620 received TCAs, predominantly transduction or angiogenesis inhibitors, as a single agent (37%), combined with other TCAs (7%), or as chemotherapy (56%); 28% received chemotherapy only; and 5% received best supportive care or placebo. A total of 17,222 patients contributed to the analyses. Within-patient variability decreased with increasing number of TLs, similarly for TCAs (with/without chemotherapy) and chemotherapy only. Mixed responses occurred proportionally in all treatment classes. Landmark analyses showed an ordinal relationship between percentage change from baseline to 12 weeks and overall survival, and demonstrated a clear distinction between tumor shrinkage and progressive disease according to RECIST. Time-dependent analysis showed no marked improvement in the ability to predict survival on the basis of TL tumor growth compared with nontarget progression or new lesion occurrence, regardless of treatment. Similar results were seen for major tumor types and different classes of TCAs. CONCLUSION: This work reinforces that RECIST version 1.1 perform well for response assessment of TCAs.

AB - PURPOSE: The mode of action of targeted cancer agents (TCAs) differs from classic chemotherapy, which leads to concerns about the role of RECIST in evaluating tumor response in trials with TCAs. We investigated the performance of RECIST using a pooled database from 50 clinical trials with at least one TCA. METHODS: We examined the impact of the number of target lesions (TLs) on within-patient variability of tumor response. The prognostic effect of TL response (at 12 weeks or on study on the basis of a maximum five TLs) on survival was studied through landmark and time-dependent Cox models adjusted for baseline tumor load, occurrence of new lesions, or unequivocal progression of nontarget disease. RESULTS: Data were obtained from 23,259 patients with cancer (36% lung, 28% colorectal, 11% breast, and 25% other); 15,620 received TCAs, predominantly transduction or angiogenesis inhibitors, as a single agent (37%), combined with other TCAs (7%), or as chemotherapy (56%); 28% received chemotherapy only; and 5% received best supportive care or placebo. A total of 17,222 patients contributed to the analyses. Within-patient variability decreased with increasing number of TLs, similarly for TCAs (with/without chemotherapy) and chemotherapy only. Mixed responses occurred proportionally in all treatment classes. Landmark analyses showed an ordinal relationship between percentage change from baseline to 12 weeks and overall survival, and demonstrated a clear distinction between tumor shrinkage and progressive disease according to RECIST. Time-dependent analysis showed no marked improvement in the ability to predict survival on the basis of TL tumor growth compared with nontarget progression or new lesion occurrence, regardless of treatment. Similar results were seen for major tumor types and different classes of TCAs. CONCLUSION: This work reinforces that RECIST version 1.1 perform well for response assessment of TCAs.

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U2 - 10.1200/JCO.18.01100

DO - 10.1200/JCO.18.01100

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VL - 37

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JO - Journal of Clinical Oncology

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SN - 0732-183X

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