TY - JOUR
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
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
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
N1 - Publisher Copyright:
© 2018 Elsevier Ltd
PY - 2018/9
Y1 - 2018/9
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.
AB - 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.
KW - Clinical trials
KW - Colorectal cancer
KW - Delphi survey
KW - Metastatic disease
KW - Patient characteristics
KW - Prognosis
KW - Stratification
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U2 - 10.1016/j.ejca.2018.05.010
DO - 10.1016/j.ejca.2018.05.010
M3 - Article
C2 - 29936065
AN - SCOPUS:85048766700
SN - 0959-8049
VL - 100
SP - 35
EP - 45
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -