摘要
Advanced cholangiocarcinoma is associated with poor prognostic survival and has limited therapeutic options available at present. The importance of angiogenesis and expression of pro-angiogenic factors in intrahepatic forms of cholangiocarcinoma suggest that therapies targeting angiogenesis might be useful for the treatment of this disease. Here we report three cases of patients with advanced intrahepatic cholangiocarcinoma progressive after standard chemotherapy and treated with sunitinib 50 mg/d in 6-wk cycles of 4 wk on treatment followed by 2 wk off treatment(Schedule 4/2). In all three patients, sunitinib treatment was associated with a sustained disease control superior to 4 mo, patients achieving either a partial response or stable disease. A reduction in tumor size and density was observed in all cases, suggesting tumor necrosis as a result of sunitinib treatment in these patients. In addition, sunitinib was generally well tolerated and the occurrence of side effects was managed with standard medical interventions, as required. Our results suggest that sunitinib therapy maybe associated with favorable outcomes and tolerability in patients with advanced cholangiocarcinoma. Those observations contributed to launch a prospective phase Ⅱ multicenter trial investigating sunitinib in advanced intrahepatic cholangiocarcinoma(SUN-CK study; NCT01718327).
Advanced cholangiocarcinoma is associated with poorprognostic survival and has limited therapeutic optionsavailable at present. The importance of angiogenesis andexpression of pro-angiogenic factors in intrahepatic formsof cholangiocarcinoma suggest that therapies targetingangiogenesis might be useful for the treatment of thisdisease. Here we report three cases of patients withadvanced intrahepatic cholangiocarcinoma progressiveafter standard chemotherapy and treated with sunitinib50 mg/d in 6-wk cycles of 4 wk on treatment followedby 2 wk off treatment （Schedule 4/2）. In all threepatients, sunitinib treatment was associated with asustained disease control superior to 4 mo, patientsachieving either a partial response or stable disease. Areduction in tumor size and density was observed in allcases, suggesting tumor necrosis as a result of sunitinibtreatment in these patients. In addition, sunitinib wasgenerally well tolerated and the occurrence of side effectswas managed with standard medical interventions, asrequired. Our results suggest that sunitinib therapy maybe associated with favorable outcomes and tolerabilityin patients with advanced cholangiocarcinoma. Thoseobservations contributed to launch a prospective phaseⅡ multicenter trial investigating sunitinib in advancedintrahepatic cholangiocarcinoma （SUN-CK study; NCT01718327）.
基金
Pfizer Inc,funding ACUMED(Tytherington,UK)for manuscript editing
