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

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.

• Authors: 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 Grothey, Volker Heinemann, Howard S Hochster, Richard S Kaplan, Scott Kopetz, Roberto Labianca, Christopher H Lieu, Neal J Meropol, Timothy J Price, Richard L Schilsky, Hans-Joachim Schmoll, Einat Shacham-Shmueli, Qian Shi, Alberto F Sobrero, John Souglakos, Eric Van Cutsem, John Zalcberg, Martijn G H van Oijen, Cornelis J A Punt, Miriam Koopman
• Journal: European journal of cancer (Oxford, England : 1990) (Eur J Cancer) Vol. 100 Pg. 35-45 (09 2018) ISSN: 1879-0852 [Electronic] England
• PMID: 29936065 (Publication Type: Journal Article, Practice Guideline)
• Copyright: Copyright © 2018 Elsevier Ltd. All rights reserved.

Chemical References

• Biomarkers, Tumor
• KRAS protein, human
• BRAF protein, human
• Proto-Oncogene Proteins B-raf
• Proto-Oncogene Proteins p21(ras)

Topics

• Biomarkers, Tumor (genetics)
• Clinical Trials, Phase III as Topic (standards)
• Colorectal Neoplasms (genetics, pathology, therapy)
• Consensus
• DNA Mismatch Repair
• Delphi Technique
• Humans
• Karnofsky Performance Status
• Liver Neoplasms (genetics, secondary, therapy)
• Microsatellite Instability
• Mutation
• Patient Selection
• Proto-Oncogene Proteins B-raf (genetics)
• Proto-Oncogene Proteins p21(ras) (genetics)
• Treatment Outcome