TriangleHEALTH PROFESSIONAL · SOURCE READING
Cabozantinib
Source: Primary Liver Cancer Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 17, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
Evidence (cabozantinib):
An international, double-blind, placebo-controlled, phase III trial (CELESTIAL [NCT01908426]) that enrolled patients from 19 countries in Asia, Europe, North America, Australia, and New Zealand included 707 patients with advanced HCC and Child-Pugh class A disease. Patients had previously received sorafenib and progressed on at least one previous systemic therapy. Patients were randomly assigned in a 2:1 ratio to receive either cabozantinib (60 mg/day) or matching placebo. The primary end point was median OS.[18]
An international, double-blind, placebo-controlled, phase III trial (CELESTIAL [NCT01908426]) that enrolled patients from 19 countries in Asia, Europe, North America, Australia, and New Zealand included 707 patients with advanced HCC and Child-Pugh class A disease. Patients had previously received sorafenib and progressed on at least one previous systemic therapy. Patients were randomly assigned in a 2:1 ratio to receive either cabozantinib (60 mg/day) or matching placebo. The primary end point was median OS.[18]
The median OS was 10.2 months for patients who received cabozantinib and 8.0 months for patients who received placebo (HR, 0.76; 95% CI, 0.63–0.92, P = .005).[18][Level of evidence A1]
Source links and citations
An international, double-blind, placebo-controlled, phase III trial (CELESTIAL [NCT01908426]) that enrolled patients from 19 countries in Asia, Europe, North America, Australia, and New Zealand included 707 patients with advanced HCC and Child-Pugh class A disease. Patients had previously received sorafenib and progressed on at least one previous systemic therapy. Patients were randomly assigned in a 2:1 ratio to receive either cabozantinib (60 mg/day) or matching placebo. The primary end point was median OS.[18]
The median PFS was 1.9 months for patients who received placebo and 5.2 months for patients who received cabozantinib (HR, 0.44; 95% CI, 0.36–0.52, P < .001).
An international, double-blind, placebo-controlled, phase III trial (CELESTIAL [NCT01908426]) that enrolled patients from 19 countries in Asia, Europe, North America, Australia, and New Zealand included 707 patients with advanced HCC and Child-Pugh class A disease. Patients had previously received sorafenib and progressed on at least one previous systemic therapy. Patients were randomly assigned in a 2:1 ratio to receive either cabozantinib (60 mg/day) or matching placebo. The primary end point was median OS.[18]
Grade 3 or 4 side effects occurred in 68% of patients who received cabozantinib compared with 37% who received placebo. Common grade 3 or 4 side effects of cabozantinib included hand-foot syndrome (17%), hypertension (16%), abnormal aspartate aminotransferase level (12%), diarrhea (11%), and fatigue (10%).
While these findings are statistically significant for OS and PFS, the absolute improvement to OS was small, toxicity (including financial toxicity) was high, and the quality-of-life data are lacking to help guide selection of regimen and who should receive treatment. These factors should all be considered and individualized for each patient.
Studies cited in this source section
Source citation is not a determination that a study applies to you.
Publication references
Read the original reference and check its publication notices.
Preserved source evidence · Not medical advice