title: Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups: The GIDEON study.
abstract: GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) is a prospective, observational registry study evaluating the safety of sorafenib and treatment practices in hepatocellular carcinoma patients. This large global database allowed for assessment of the use and tolerability of sorafenib in patients with liver dysfunction.
  
  
  
  Baseline characteristics and medical/treatment history were collected in patients for whom a decision to treat with sorafenib had been made. Adverse event, dosing, and outcomes data were collected during follow-up.
  
  
  
  In the overall safety population (n=3202), 1968 patients (61%) had Child-Pugh A status and 666 (21%) had Child-Pugh B. The majority of Child-Pugh A (72%) and Child-Pugh B (70%) patients received an initial sorafenib dose of 800mg, consistent with the label, and dose reduction rates were 40% and 29%, respectively. The type and incidence of adverse events were generally consistent across Child-Pugh subgroups. The incidence of drug-related adverse events leading to discontinuation was similar between Child-Pugh A and Child-Pugh B patients (17% and 21%). In the intent-to-treat population (n=3213), median overall survival (months [95% confidence interval]) was longer in Child-Pugh A patients (13.6 [12.8-14.7]) compared with Child-Pugh B patients (5.2 [4.6-6.3]).
  
  
  
  In clinical practice, the safety profile of sorafenib appeared to be consistent across Child-Pugh A and Child-Pugh B patients. Findings suggest sorafenib may be safely used in some Child-Pugh B patients and indicate the importance of careful patient evaluation when making treatment decisions.
  
  
  
  The GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) study is a large prospective registry of patients with liver cancer who were treated with sorafenib. The aims were to evaluate the safety and tolerability of sorafenib among those in which the liver was not functioning properly. The study showed that the safety profile of sorafenib was consistent across patients with preserved liver function and those in which the liver was not functioning properly, and therefore, suggesting that sorafenib may be a valid treatment for some patients with liver impairment.
authors.affiliation: Department of Internal Medicine, Division of Digestive and Liver Diseases, University of Texas Southwestern Medical Center, Dallas, TX, USA. Electronic address: jorge.marrero@utsouthwestern.edu.
authors.forename: Jorge A
authors.initials: JA
authors.lastname: Marrero
authors.affiliation: Department of Gastroenterology and Hepatology, Kinki University School of Medicine, Osaka, Japan.
authors.forename: Masatoshi
authors.initials: M
authors.lastname: Kudo
authors.affiliation: Medical Oncology and Translational Research, University of California, San Francisco, CA, USA.
authors.forename: Alan P
authors.initials: AP
authors.lastname: Venook
authors.affiliation: Liver Cancer Institute and Zhongshan Hospital, Fudan University, Shanghai, China.
authors.forename: Sheng-Long
authors.initials: SL
authors.lastname: Ye
authors.affiliation: University Henri Poincaré by University of Lorraine, Nancy, France.
authors.forename: Jean-Pierre
authors.initials: JP
authors.lastname: Bronowicki
authors.affiliation: Hepatic Surgery Center, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
authors.forename: Xiao-Ping
authors.initials: XP
authors.lastname: Chen
authors.affiliation: Policlínica Metropolitana, Caracas, Venezuela.
authors.forename: Lucy
authors.initials: L
authors.lastname: Dagher
authors.affiliation: Kyorin University School of Medicine, Mitaka, Tokyo, Japan.
authors.forename: Junji
authors.initials: J
authors.lastname: Furuse
authors.affiliation: Vascular and Interventional Radiology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
authors.forename: Jean-Francois H
authors.initials: JH
authors.lastname: Geschwind
authors.affiliation: Hospital Angeles Clínica Londres, Mexico City, Mexico.
authors.forename: Laura Ladrón
authors.initials: LL
authors.lastname: de Guevara
authors.affiliation: University Hospital of Larissa, Larissa, Greece.
authors.forename: Christos
authors.initials: C
authors.lastname: Papandreou
authors.affiliation: Department of Digestive Surgery, Nihon University School of Medicine, Tokyo, Japan.
authors.forename: Tadatoshi
authors.initials: T
authors.lastname: Takayama
authors.affiliation: Virginia Commonwealth University Medical Center, Richmond, VA, USA.
authors.forename: Arun J
authors.initials: AJ
authors.lastname: Sanyal
authors.affiliation: The Catholic University of Korea, Seoul, Republic of Korea.
authors.forename: Seung Kew
authors.initials: SK
authors.lastname: Yoon
authors.affiliation: Global Medical Affairs, Bayer HealthCare Pharmaceuticals, Inc., Montville, NJ, USA.
authors.forename: Keiko
authors.initials: K
authors.lastname: Nakajima
authors.affiliation: Clinical Statistics, Bayer HealthCare Pharmaceuticals, Inc., Montville, NJ, USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Lehr
authors.affiliation: Global Medical Affairs and Pharmacovigilance, Bayer HealthCare Pharmaceuticals, Inc., Berlin, Germany.
authors.forename: Stephanie
authors.initials: S
authors.lastname: Heldner
authors.affiliation: Division of Diagnostic Imaging and Intervention, Pisa University Hospital and School of Medicine, Pisa, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Lencioni
mesh_terms: D000970:Antineoplastic Agents; D006528:Carcinoma, Hepatocellular; D002648:Child; D006801:Humans; D008113:Liver Neoplasms; D009536:Niacinamide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000627:therapeutic use*; D011446:Prospective Studies; D012042:Registries; D000077157:Sorafenib
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib
keywords: Child-Pugh; GIDEON; HCC; Hepatocellular carcinoma; Nexavar; Sorafenib