Erlotinib is effective in pancreatic cancer with epidermal growth factor receptor mutations: a randomized, open-label, prospective trial

// Jack P. Wang 1,2,3,* , Chen-Yi Wu 4,5,6,* , Yi-Cheng Yeh 2,7 , Yi-Ming Shyr 2,8 , Ying-Ying Wu 1,2 , Chen-Yu Kuo 1,2,9,10 , Yi-Ping Hung 1,2,4,10 , Ming-Huang Chen 2,10 , Wei-Ping Lee 2,11 , Jiing-Chyuan Luo 1,2 , Yee Chao 1,2,12 and Chung-Pin Li 1,2 1 Division of Gastroenterology, Department of Medicine, Taipei Veterans General Hospital, Taipei, Taiwan 2 National Yang-Ming University School of Medicine, Taipei, Taiwan 3 Division of Gastroenterology, Department of Internal Medicine, Renai Branch, Taipei City Hospital, Taipei, Taiwan 4 Institute of Public Health, National Yang-Ming University, Taipei, Taiwan 5 Department of Dermatology, Taipei Veterans General Hospital, Taipei, Taiwan 6 Department of Dermatology, Heping Fuyou Branch, Taipei City Hospital, Taipei, Taiwan 7 Department of Pathology, Taipei Veterans General Hospital, Taipei, Taiwan 8 Division of General Surgery, Department of Surgery, Taipei Veterans General Hospital, Taipei, Taiwan 9 Department of Medicine, National Yang-Ming University Hospital, Yilan, Taiwan 10 Division of Oncology, Department of Medicine, Taipei Veterans General Hospital, Taipei, Taiwan 11 Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan 12 Department of Oncology Medicine, Taipei Veterans General Hospital, Taipei, Taiwan * These authors contributed equally to this manuscript Correspondence to: Chung-Pin Li, email: // Keywords : adenocarcinoma; pancreas; erlotinib; epidermal growth factor receptor Received : November 18, 2014 Accepted : May 12, 2015 Published : May 20, 2015 Abstract Objective To analyze the efficacy of gemcitabine with or without erlotinib for pancreatic cancer, and to determine the predictive role of epidermal growth factor receptor ( EGFR ) and KRAS mutations in these patients. Methods This was a single-center, randomized, open-label, prospective trial. Eighty-eight chemotherapy-naive metastatic pancreatic cancer patients were randomized for treatment with gemcitabine or gemcitabine plus erlotinib. EGFR and KRAS mutations were analyzed, respectively. The primary endpoint was the disease control rate. Results Disease control rate (64% vs. 25%; P < 0.001), progression-free survival (median 3.8 vs. 2.4 months; P < 0.001), and overall survival (median 7.2 vs. 4.4 months; P < 0.001) were better in the gemcitabine plus erlotinib group than in the gemcitabine alone group. In the gemcitabine plus erlotinib group, disease control (85% vs. 33%; P = 0.001), progression-free survival (median 5.9 vs . 2.4 months; P = 0.004), and overall survival (median 8.7 vs . 6.0 months; P = 0.044) were better in patients with EGFR mutations than in those without EGFR mutations. KRAS mutation was not associated with treatment response or survival. Conclusions Gemcitabine plus erlotinib is more effective than gemcitabine alone for treating metastatic pancreatic cancer patients, especially those with EGFR mutations. ClinicalTrials.gov number, NCT01608841.