title: Survival and cost-effectiveness of sorafenib therapy in advanced hepatocellular carcinoma: An analysis of the SEER-Medicare database.
abstract: Sorafenib is the only chemotherapeutic approved for treatment of advanced hepatocellular carcinoma (HCC). However, its effectiveness in patients with Child-Pugh class B cirrhosis and any moderating effects of health system characteristics are unclear. We examined the survival and cost-effectiveness associated with sorafenib in elderly patients with advanced HCC. We performed an analysis of Medicare beneficiaries with HCC diagnoses from 2007 to 2009. We compared advanced stage patients with HCC (American Joint Committee on Cancer stage III/IV) who received sorafenib within 6 months of diagnosis (and were otherwise untreated) to advanced stage patients with HCC who received no therapy (control). We performed univariate and multivariate analyses to identify predictors of survival. Incremental cost-effectiveness ratios (ICERs) were calculated for sorafenib-treated and control patients. We included 228 sorafenib-treated patients and 870 control patients. The median survival of the sorafenib-treated patients was 150.5 days versus 62 days for control patients. On multivariate analysis, significant predictors of improved survival were treatment with sorafenib (hazard ratio [HR], 0.66; 95% confidence interval [CI], 0.57-0.77), being seen at a National Cancer Institute-designated cancer center (HR, 0.77; 95% CI, 0.62-0.97), and being seen at a transplantation center (HR, 0.77; 95% CI, 0.65-0.93). Predictors of worse survival included stage IV disease (HR, 1.40; 95% CI, 1.24-1.58), decompensated cirrhosis (HR, 1.49; 95% CI, 1.30-1.70), and treatment in an urban setting (HR, 1.45; 95% CI, 1.21-1.73.) Although sorafenib use was associated with a survival benefit (HR, 0.61; 95% CI, 0.47-0.79) among patients with decompensated cirrhosis, the median survival benefit was 31 days, and it was not cost-effective (ICER, $224,914 per life year gained).
  
  
  
  Sorafenib is associated with improved survival in elderly patients with advanced HCC; however, it is not cost-effective among those with hepatic decompensation. (Hepatology 2017;65:122-133).
authors: Division of Gastroenterology, University of Michigan, Ann Arbor, MI.
authors: Neehar D
authors: ND
authors: Parikh
authors: College of Pharmacy, University of Michigan, Ann Arbor, MI.
authors: Vincent D
authors: VD
authors: Marshall
authors: Division of Digestive and Liver Disease, UT Southwestern Medical Center, Dallas, TX.
authors: Amit G
authors: AG
authors: Singal
authors: Department of Surgery, University of Michigan, Ann Arbor, MI.
authors: Hari
authors: H
authors: Nathan
authors: Division of Gastroenterology, University of Michigan, Ann Arbor, MI.
authors: Anna S
authors: AS
authors: Lok
authors: Department of Public Health Sciences, University of Virginia, Charlottesville, VA.
authors: Rajesh
authors: R
authors: Balkrishnan
authors: Kidney Epidemiology and Cost Center, University of Michigan, Ann Arbor, MI.
authors: Vahakn
authors: V
authors: Shahinian
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000191:economics* / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000191:economics / Q000401:mortality* / Q000473:pathology; D003362:Cost-Benefit Analysis; D016208:Databases, Factual; D005260:Female; D006801:Humans; D008103:Liver Cirrhosis; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000191:economics / Q000401:mortality* / Q000473:pathology; D008297:Male; D006278:Medicare; D009367:Neoplasm Staging; D009536:Niacinamide / Q000031:analogs & derivatives* / Q000191:economics / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000191:economics* / Q000627:therapeutic use*; D016016:Proportional Hazards Models; D018426:SEER Program; D000077157:Sorafenib; D015996:Survival Rate; D014481:United States
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib