Consensus statement on essential patient characteristics in systemic treatment trials for metastatic colorectal cancer: Supported by the ARCAD Group

Kaitlyn K.H. Goey, Halfdan Sørbye, Bengt Glimelius, Richard A. Adams, Thierry André, Dirk Arnold, Jordan D. Berlin, György Bodoky, Aimery de Gramont, Eduardo Díaz-Rubio, Cathy Eng, Alfredo Falcone, Axel F Grothey, Volker Heinemann, Howard S. Hochster, Richard S. Kaplan, Scott Kopetz, Roberto Labianca, Christopher H. Lieu, Neal J. MeropolTimothy J. Price, Richard L. Schilsky, Hans Joachim Schmoll, Einat Shacham-Shmueli, Qian D Shi, Alberto F. Sobrero, John Souglakos, Eric Van Cutsem, John Zalcberg, Martijn G.H. van Oijen, Cornelis J.A. Punt, Miriam Koopman

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Background: Patient characteristics and stratification factors are key features influencing trial outcomes. However, there is substantial heterogeneity in reporting of patient characteristics and use of stratification factors in phase 3 trials investigating systemic treatment of metastatic colorectal cancer (mCRC). We aimed to develop a minimum set of essential baseline characteristics and stratification factors to include in such trials. Methods: We performed a modified, two-round Delphi survey among international experts with wide experience in the conduct and methodology of phase 3 trials of systemic treatment of mCRC. Results: Thirty mCRC experts from 15 different countries completed both consensus rounds. A total of 14 patient characteristics were included in the recommended set: age, performance status, primary tumour location, primary tumour resection, prior chemotherapy, number of metastatic sites, liver-only disease, liver involvement, surgical resection of metastases, synchronous versus metachronous metastases, (K)RAS and BRAF mutation status, microsatellite instability/mismatch repair status and number of prior treatment lines. A total of five patient characteristics were considered the most relevant stratification factors: RAS/BRAF mutation status, performance status, primary tumour sidedness and liver-only disease. Conclusions: This survey provides a minimum set of essential baseline patient characteristics and stratification factors to include in phase 3 trials of systemic treatment of mCRC. Inclusion of these patient characteristics and strata in study protocols and final study reports will improve interpretation of trial results and facilitate cross-study comparisons.

Original languageEnglish (US)
Pages (from-to)35-45
Number of pages11
JournalEuropean Journal of Cancer
Volume100
DOIs
StatePublished - Sep 1 2018

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Colorectal Neoplasms
Liver Diseases
Therapeutics
Neoplasm Metastasis
Neoplasms
Microsatellite Instability
Mutation
DNA Mismatch Repair
Drug Therapy
Liver
Surveys and Questionnaires

Keywords

  • Clinical trials
  • Colorectal cancer
  • Delphi survey
  • Metastatic disease
  • Patient characteristics
  • Prognosis
  • Stratification

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Consensus statement on essential patient characteristics in systemic treatment trials for metastatic colorectal cancer : Supported by the ARCAD Group. / Goey, Kaitlyn K.H.; Sørbye, Halfdan; Glimelius, Bengt; Adams, Richard A.; André, Thierry; Arnold, Dirk; Berlin, Jordan D.; Bodoky, György; de Gramont, Aimery; Díaz-Rubio, Eduardo; Eng, Cathy; Falcone, Alfredo; Grothey, Axel F; Heinemann, Volker; Hochster, Howard S.; Kaplan, Richard S.; Kopetz, Scott; Labianca, Roberto; Lieu, Christopher H.; Meropol, Neal J.; Price, Timothy J.; Schilsky, Richard L.; Schmoll, Hans Joachim; Shacham-Shmueli, Einat; Shi, Qian D; Sobrero, Alberto F.; Souglakos, John; Van Cutsem, Eric; Zalcberg, John; van Oijen, Martijn G.H.; Punt, Cornelis J.A.; Koopman, Miriam.

In: European Journal of Cancer, Vol. 100, 01.09.2018, p. 35-45.

Research output: Contribution to journalArticle

Goey, KKH, Sørbye, H, Glimelius, B, Adams, RA, André, T, Arnold, D, Berlin, JD, Bodoky, G, de Gramont, A, Díaz-Rubio, E, Eng, C, Falcone, A, Grothey, AF, Heinemann, V, Hochster, HS, Kaplan, RS, Kopetz, S, Labianca, R, Lieu, CH, Meropol, NJ, Price, TJ, Schilsky, RL, Schmoll, HJ, Shacham-Shmueli, E, Shi, QD, Sobrero, AF, Souglakos, J, Van Cutsem, E, Zalcberg, J, van Oijen, MGH, Punt, CJA & Koopman, M 2018, 'Consensus statement on essential patient characteristics in systemic treatment trials for metastatic colorectal cancer: Supported by the ARCAD Group', European Journal of Cancer, vol. 100, pp. 35-45. https://doi.org/10.1016/j.ejca.2018.05.010
Goey, Kaitlyn K.H. ; Sørbye, Halfdan ; Glimelius, Bengt ; Adams, Richard A. ; André, Thierry ; Arnold, Dirk ; Berlin, Jordan D. ; Bodoky, György ; de Gramont, Aimery ; Díaz-Rubio, Eduardo ; Eng, Cathy ; Falcone, Alfredo ; Grothey, Axel F ; Heinemann, Volker ; Hochster, Howard S. ; Kaplan, Richard S. ; Kopetz, Scott ; Labianca, Roberto ; Lieu, Christopher H. ; Meropol, Neal J. ; Price, Timothy J. ; Schilsky, Richard L. ; Schmoll, Hans Joachim ; Shacham-Shmueli, Einat ; Shi, Qian D ; Sobrero, Alberto F. ; Souglakos, John ; Van Cutsem, Eric ; Zalcberg, John ; van Oijen, Martijn G.H. ; Punt, Cornelis J.A. ; Koopman, Miriam. / Consensus statement on essential patient characteristics in systemic treatment trials for metastatic colorectal cancer : Supported by the ARCAD Group. In: European Journal of Cancer. 2018 ; Vol. 100. pp. 35-45.
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abstract = "Background: Patient characteristics and stratification factors are key features influencing trial outcomes. However, there is substantial heterogeneity in reporting of patient characteristics and use of stratification factors in phase 3 trials investigating systemic treatment of metastatic colorectal cancer (mCRC). We aimed to develop a minimum set of essential baseline characteristics and stratification factors to include in such trials. Methods: We performed a modified, two-round Delphi survey among international experts with wide experience in the conduct and methodology of phase 3 trials of systemic treatment of mCRC. Results: Thirty mCRC experts from 15 different countries completed both consensus rounds. A total of 14 patient characteristics were included in the recommended set: age, performance status, primary tumour location, primary tumour resection, prior chemotherapy, number of metastatic sites, liver-only disease, liver involvement, surgical resection of metastases, synchronous versus metachronous metastases, (K)RAS and BRAF mutation status, microsatellite instability/mismatch repair status and number of prior treatment lines. A total of five patient characteristics were considered the most relevant stratification factors: RAS/BRAF mutation status, performance status, primary tumour sidedness and liver-only disease. Conclusions: This survey provides a minimum set of essential baseline patient characteristics and stratification factors to include in phase 3 trials of systemic treatment of mCRC. Inclusion of these patient characteristics and strata in study protocols and final study reports will improve interpretation of trial results and facilitate cross-study comparisons.",
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T1 - Consensus statement on essential patient characteristics in systemic treatment trials for metastatic colorectal cancer

T2 - Supported by the ARCAD Group

AU - Goey, Kaitlyn K.H.

AU - Sørbye, Halfdan

AU - Glimelius, Bengt

AU - Adams, Richard A.

AU - André, Thierry

AU - Arnold, Dirk

AU - Berlin, Jordan D.

AU - Bodoky, György

AU - de Gramont, Aimery

AU - Díaz-Rubio, Eduardo

AU - Eng, Cathy

AU - Falcone, Alfredo

AU - Grothey, Axel F

AU - Heinemann, Volker

AU - Hochster, Howard S.

AU - Kaplan, Richard S.

AU - Kopetz, Scott

AU - Labianca, Roberto

AU - Lieu, Christopher H.

AU - Meropol, Neal J.

AU - Price, Timothy J.

AU - Schilsky, Richard L.

AU - Schmoll, Hans Joachim

AU - Shacham-Shmueli, Einat

AU - Shi, Qian D

AU - Sobrero, Alberto F.

AU - Souglakos, John

AU - Van Cutsem, Eric

AU - Zalcberg, John

AU - van Oijen, Martijn G.H.

AU - Punt, Cornelis J.A.

AU - Koopman, Miriam

PY - 2018/9/1

Y1 - 2018/9/1

N2 - Background: Patient characteristics and stratification factors are key features influencing trial outcomes. However, there is substantial heterogeneity in reporting of patient characteristics and use of stratification factors in phase 3 trials investigating systemic treatment of metastatic colorectal cancer (mCRC). We aimed to develop a minimum set of essential baseline characteristics and stratification factors to include in such trials. Methods: We performed a modified, two-round Delphi survey among international experts with wide experience in the conduct and methodology of phase 3 trials of systemic treatment of mCRC. Results: Thirty mCRC experts from 15 different countries completed both consensus rounds. A total of 14 patient characteristics were included in the recommended set: age, performance status, primary tumour location, primary tumour resection, prior chemotherapy, number of metastatic sites, liver-only disease, liver involvement, surgical resection of metastases, synchronous versus metachronous metastases, (K)RAS and BRAF mutation status, microsatellite instability/mismatch repair status and number of prior treatment lines. A total of five patient characteristics were considered the most relevant stratification factors: RAS/BRAF mutation status, performance status, primary tumour sidedness and liver-only disease. Conclusions: This survey provides a minimum set of essential baseline patient characteristics and stratification factors to include in phase 3 trials of systemic treatment of mCRC. Inclusion of these patient characteristics and strata in study protocols and final study reports will improve interpretation of trial results and facilitate cross-study comparisons.

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KW - Metastatic disease

KW - Patient characteristics

KW - Prognosis

KW - Stratification

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