Safety and efficacy of cabozantinib for patients with advanced hepatocellular carcinoma who advanced to Child-Pugh B liver function at study week 8: a retrospective analysis of the CELESTIAL randomised controlled trial

BMC Cancer. 2022 Apr 9;22(1):377. doi: 10.1186/s12885-022-09453-z.

Abstract

Background: Patients with hepatocellular carcinoma (HCC) and Child-Pugh B liver cirrhosis have poor prognosis and are underrepresented in clinical trials. The CELESTIAL trial, in which cabozantinib improved overall survival (OS) and progression-free survival (PFS) versus placebo in patients with HCC and Child-Pugh A liver cirrhosis at baseline, was evaluated for outcomes in patients who had Child-Pugh B cirrhosis at Week 8.

Methods: This was a retrospective analysis of adult patients with previously treated advanced HCC. Child-Pugh B status was assessed by the investigator. Patients were randomised 2:1 to cabozantinib (60 mg once daily) or placebo.

Results: Fifty-one patients receiving cabozantinib and 22 receiving placebo had Child-Pugh B cirrhosis at Week 8. Safety and tolerability of cabozantinib for the Child-Pugh B subgroup were consistent with the overall population. For cabozantinib- versus placebo-treated patients, median OS from randomisation was 8.5 versus 3.8 months (HR 0.32, 95% CI 0.18-0.58), median PFS was 3.7 versus 1.9 months (HR 0.44, 95% CI 0.25-0.76), and best response was stable disease in 57% versus 23% of patients.

Conclusions: These encouraging results with cabozantinib support the initiation of prospective studies in patients with advanced HCC and Child-Pugh B liver function.

Clinical trial registration: NCT01908426.

Keywords: Cabozantinib; Child–Pugh B; Hepatocellular carcinoma.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adult
  • Anilides
  • Antineoplastic Agents* / adverse effects
  • Carcinoma, Hepatocellular* / pathology
  • Humans
  • Liver Cirrhosis / drug therapy
  • Liver Neoplasms* / pathology
  • Prospective Studies
  • Pyridines
  • Retrospective Studies

Substances

  • Anilides
  • Antineoplastic Agents
  • Pyridines
  • cabozantinib

Associated data

  • ClinicalTrials.gov/NCT01908426