Safety and efficacy of sorafenib in Japanese patients with hepatocellular carcinoma in clinical practice: a subgroup analysis of GIDEON

J Gastroenterol. 2016 Dec;51(12):1150-1160. doi: 10.1007/s00535-016-1204-2. Epub 2016 Apr 22.


Background: GIDEON was a prospective, global, non-interventional study evaluating the safety of sorafenib in patients with unresectable hepatocellular carcinoma in real-world practice. The aim of this subgroup analysis was to assess the safety and efficacy of sorafenib as used by Japanese patients.

Methods: In Japan, 508 patients were valid for safety analysis. Efficacy and safety were evaluated by the Child-Pugh score.

Results: The number of patients with Child-Pugh A and B was 432 (85.0 %) and 58 (11.4 %), respectively. The median overall survival time and time to progression in patients with Child-Pugh A and Child-Pugh B were 17.4 and 4.9 months, 3.7 and 2.3 months, respectively. The most common drug-related adverse events (AEs) included hand-foot skin reaction (47.8 %), diarrhea (35.8 %) and hypertension (24.2 %). The incidences of all or drug-related AEs were similar between patients with Child-Pugh A and B. However, all or drug-related serious AEs, AEs resulting in permanent discontinuation of sorafenib and deaths were observed more frequently in patients with Child-Pugh B compared with Child-Pugh A. Duration of treatment tended to be shorter as the Child-Pugh score worsened.

Conclusions: Sorafenib was well tolerated by Japanese HCC patients in clinical settings. Patients with Child-Pugh B had shorter duration of treatment and higher incidence of SAEs. It is important to carefully evaluate patients' conditions and assess the benefit and risk before making a decision to treat patients with sorafenib.

Keywords: GIDEON; Hepatocellular carcinoma; Japanese; Sorafenib.

Publication types

  • Multicenter Study
  • Observational Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects*
  • Antineoplastic Agents / therapeutic use
  • Carcinoma, Hepatocellular / drug therapy*
  • Carcinoma, Hepatocellular / pathology
  • Chemoembolization, Therapeutic / methods
  • Dose-Response Relationship, Drug
  • Female
  • Humans
  • Kaplan-Meier Estimate
  • Liver Neoplasms / drug therapy*
  • Liver Neoplasms / pathology
  • Male
  • Middle Aged
  • Neoplasm Staging
  • Niacinamide / administration & dosage
  • Niacinamide / adverse effects
  • Niacinamide / analogs & derivatives*
  • Niacinamide / therapeutic use
  • Phenylurea Compounds / administration & dosage
  • Phenylurea Compounds / adverse effects*
  • Phenylurea Compounds / therapeutic use
  • Product Surveillance, Postmarketing / methods
  • Prospective Studies
  • Sorafenib
  • Treatment Outcome
  • Young Adult


  • Antineoplastic Agents
  • Phenylurea Compounds
  • Niacinamide
  • Sorafenib