Apatinib as first-line treatment in patients with advanced hepatocellular carcinoma: a phase II clinical trial

Abstract

Background:The prognosis for advanced hepatocellular carcinoma (HCC) remains clinically unsatisfying.Apatinib has proven to be a very effective treatment for advanced HCC in our previous retrospective study.Our aim in this study was to evaluate the efficacy, safety, and toxicity of apatinib in patients with advanced HCC.Methods: This single-arm, open-label phase II clinical trial enrolled patients with advanced HCC. These patients received apatinib, 500 mg once daily, until disease progression, unacceptable toxicity, consent withdrawal, or death. One treatment cycle consisted of 4 weeks of apatinib treatment. The response evaluation criteria in solid tumors (RECIST) was used to assess tumor response every 1-2 cycles. The primary endpoint was the objective response rate (ORR), while the secondary endpoints were the overall survival (OS), progression-free survival (PFS), disease control rate (DCR), and toxicity. Results:Between December 2016 and June 2018, 23 patients were enrolled in the study, 22 of whom were available for response evaluation. The cutoff date was August 10, 2018. The overall ORR and DCR were 30.4% and 65.2%, respectively. The median OS and PFS were 13.8 (95% CI: 5.3-22.3) and 8.7 (95% CI: 5.9-11.1) months, respectively. The most common treatment-related adverse events were proteinuria (39.1%), hypertension (34.8%), and hand-foot-skin reaction (34.8%).Conclusions: Apatinib showed robust clinical activity in patients with advanced HCC. Moreover, apatinib was safe to use, well tolerated, and had acceptable toxicity. (NCT03046979).

Mechanisms of Pharmacoresistance in Hepatocellular Carcinoma: New Drugs but Old Problems

“…This is the case of donafenib (targeting RAF and VEGFR) (NCT02645981) 69 and apatinib (targeting VEGFR-2) (NCT02329860 and NCT03046979). 70 The efficacy of a sequential therapy based on TKIs is also being evaluated. A recent study has demonstrated that lenvatinib is a good option as a third-line therapy for those patients progressing under treatments with sorafenib and regorafenib.…”

Sorafenib Versus Apatinib Both Combined Transarterial Chemoembolization for Hepatocellular Carcinoma With Portal Vein Tumor Thrombosis: A Comparative Retrospective Study

“…In China, apatinib has been approved recently for the treatment of HCC due to its satisfactory efficiency. A randomized clinical trial showed that apatinib prolongs the OS of BCLC stage B or C HCC patients, which has also been validated in several retrospective studies (17)(18)(19)(20). In addition, several researches indicated that apatinib is effective in advanced HCC patients with PVTT when combined with TACE (21,22).…”

Efficacy and Safety of Apatinib Treatment for Advanced Cholangiocarcinoma After Failed Gemcitabine-Based Chemotherapy: An Open-Label Phase II Prospective Study

“…Notably, in another phase II clinical trial, for intermediate and advanced hepatocellular carcinoma patients, the patients receiving apatinib as the first-line therapy exhibited a tremendous potential in the long-term curative effect (mPFS: 8.7 months; mOS:13.8 months) ( 47 ). Therefore, we intend to conduct a new prospective study to fill the gap that there is a limited related theoretical basis for the therapeutic efficacy of apatinib in the first-line treatment of recurrent or metastatic advanced cholangiocarcinoma in the future.…”