The results of this double blind, placebo controlled trial show that patients with stage IV adenocarcinoma of the colon or rectum have increased overall survival when treated with regorafenib. The median OS in the regorafenib group was 8.8 months compared to 6.3 months with placebo (Hazard Ratio (HR) = 0.550, p=0.0002). Secondary endpoints showed similar results with an increase in PFS for patients receiving regorafenib 3.2 months compared to 1.7 months, (HR= 0.311, p=<0.0001); and disease control rate (DCR) 52% vs 7%, respectively.
The study enrolled 204 patients in 25 centres in mainland China, Hong Kong, Taiwan, Republic of Korea, and Vietnam. The patients were randomised (2:1) to receive best supportive care plus either oral regorafenib 160 mg daily or placebo during the first 3 weeks of each 4-week cycle. Treatment was continued until disease progression, unacceptable toxicity, or consent withdrawal.
Entry criteria included disease progression within 3 months of completing standard therapy, ECOG PS 0 or 1, and at least 2 prior treatment lines for mCRC. The primary endpoint was overall survival (OS). Secondary endpoints included progression-free survival (PFS), tumour response, disease control rate (DCR), and safety.
The most frequent treatment-emergent NCI-CTCAE grade≥3 adverse events in regorafenib-treated patients were hand-foot skin reaction (16%), hypertension (12%), hyperbilirubinemia (12%), elevated liver enzymes (AST 10%, ALT8%), hypophosphatemia (9%), anaemia (7%), and hyperlipasemia (7%). There were no reports of liver failure or pancreatitis.
Abstracts from the 16th ESMO World Congress on Gastrointestinal Cancer are published in Annals of Oncology, Volume 25 suppl 2 June 2014 (Ann Oncol 2014 Jun; 25 (Suppl 2): 1-117)