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 - Funding Information:
CJAP acted in an advisory role for Nordic-Pharma and Servier. HS reports grants from Novartis, Ipsen and Amgen and personal fees from Novartis, Celgene, Roche, Ipsen, Amgen, Shire and Merck. RA reports grants from Cancer Research UK, EME MRC and Astra Zeneca and personal fees and non-financial support from Amgen, Merck Serono, BMS and Servier. TA reports grants from Bayer and BMS and personal fees from Amgen, Bayer, BMS, Eli-Lilly, MSD Oncology, Servier, Xbiotech and Yakult. EDR reports grants from Merck and Amgen and personal fees from Merck, Amgen, MSD, Bayer, Genomica, Sanofi and Roche. CE reports grants from Keryx and Daiichi, consulting fees from Bayer and Sirtex and other support (speaker's bureau) from Genentech. AF reports grants and personal fees from Amgen, Bayer, Merck, Roche, Servier, Bristol, MSD and Lilly. VH reports grants from Merck, Amgen, Roche, Sanofi, Servier, Boehringer Ingelheim and Pfizer, personal fees from Merck, Roche, Sanofi, Sirtex, Bristol Mayer Squibb, MSD, Novartis and Boehringer Ingelheim and non-financial support from Merck, Roche, Sirtex and Bayer. HH reports consultancy fees from Bristol Meyers Squibb, Bayer, Merck and Genentech. SK reports personal fees (consultancy fees) from Amgen, Array, Bayer, Genentech, MolecularMatch and Taiho. NM reports grants from Genomic Health (research support) and is an employee of Flatiron Health. TP reports personal fees from Amgen. JS reports grants from Amgen, Roche and Sanofi, personal fees from Amgen, Merck, Roche, Sanofi and Servier and non-financial support from Amgen, Merck, Roche and Sanofi. The other authors declare no competing interests.
Publisher Copyright:
© 2018 Elsevier Ltd
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.
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
UR - http://www.scopus.com/inward/record.url?scp=85048766700&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2018.05.010
DO - 10.1016/j.ejca.2018.05.010
M3 - Article
AN - SCOPUS:85048766700
SN - 0959-8049
VL - 100
SP - 35
EP - 45
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -