Purpose: Effective treatment options for intrahepatic cholangiocarcinoma (ICC) are limited. This study was intended to explore the efficacy and safety of apatinib in advanced ICC with lymph node metastasis or distant metastasis.
Patients and Methods: The efficacy and toxicity of apatinib were evaluated in patients with ICC between November 2017 and March 2020 at the Second Affiliated Hospital of Anhui Medical University. Survival analysis was estimated using Kaplan–Meier method.
Results: Ten patients with advanced ICC were enrolled. The median progression-free survival (PFS) was 3.0 months (95% CI: 1.450– 4.550). No patient achieved a complete response (CR). One patient gained partial response (PR), and 6 patients had stable disease (SD). The objective response rate (ORR) was 10%, and the disease control rate (DCR) was 70%. The common treatment-related adverse events were hypertension (20%), proteinuria (30%), hand and foot syndrome (10%) or emesis (10%). No grade 3/4 toxicities occurred. Toxicities were mild and tolerable.
Conclusion: Apatinib is potentially an effective treatment option with tolerable toxicities for patients with advanced ICC.
Keywords: intrahepatic cholangiocarcinoma, apatinib, efficacy, toxicity, progression-free survival