title: Safety and efficacy of sunitinib in patients with advanced hepatocellular carcinoma: an open-label, multicentre, phase II study.
abstract: BACKGROUND
  Hepatocellular carcinoma (HCC) tumour spread is partly dependent on neoangiogenesis. In this open-label, multicentre, phase II trial done in Europe and Asia, sunitinib, a multitargeted tyrosine-kinase inhibitor with anti-angiogenic properties, was assessed in patients with advanced unresectable HCC.
  
  
  METHODS
  Between February and July, 2006, eligible patients were enrolled and treated with repeated cycles of oral sunitinib (50 mg/day for 4 weeks, followed by 2 weeks off treatment). The primary endpoint of this Simon two-stage phase II trial was objective response rate according to Response Evaluation Criteria in Solid Tumours (RECIST) criteria, with an expected response rate of 15%. This trial is registered with ClinicalTrials.gov, number NCT00247676.
  
  
  RESULTS
  Of 37 patients enrolled, one (2.7%) patient experienced a confirmed partial response, giving an overall objective response rate of 2.7% (95% CI 0.1-14.2); on the basis of this, the trial did not proceed to the second stage. 13 (35%) of 37 patients achieved stable disease for over 3 months. Commonly observed grade 3 and 4 adverse events included thrombocytopenia (14 of 37; 37.8%), neutropenia (nine of 37; 24.3%), asthenia (five of 37; 13.5%), hand-foot syndrome (four of 37; 10.8%), and anaemia (four of 37; 10.8%). There were four deaths among the 37 patients (10.8%) that were possibly related to treatment.
  
  
  CONCLUSIONS
  Sunitinib showed pronounced toxicities at a dose of 50 mg/day in patients with unresectable HCC. The response rate was low, and the study did not meet the primary endpoint based on RECIST criteria.
  
  
  BACKGROUND
  Pfizer Oncology.
authors: Beaujon University Hospital, Clichy, France. sandrine.faivre@bjn.aphp.fr
authors: Sandrine
authors: S
authors: Faivre
authors: Eric
authors: E
authors: Raymond
authors: Eveline
authors: E
authors: Boucher
authors: Jean
authors: J
authors: Douillard
authors: Ho Y
authors: HY
authors: Lim
authors: Jun S
authors: JS
authors: Kim
authors: Magaly
authors: M
authors: Zappa
authors: Silvana
authors: S
authors: Lanzalone
authors: Xun
authors: X
authors: Lin
authors: Samuel
authors: S
authors: Deprimo
authors: Charles
authors: C
authors: Harmon
authors: Ana
authors: A
authors: Ruiz-Garcia
authors: Maria J
authors: MJ
authors: Lechuga
authors: Ann Lii
authors: AL
authors: Cheng
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000401:mortality; D005260:Female; D006801:Humans; D007211:Indoles / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D011758:Pyrroles / Q000627:therapeutic use*; D000077210:Sunitinib
chemical_list: D000970:Antineoplastic Agents; D007211:Indoles; D011758:Pyrroles; D000077210:Sunitinib