title: Exploring the efficacy and safety of single-agent sorafenib in a cohort of Italian patients with hepatocellular carcinoma.
abstract: This study investigates the effectiveness and safety of sorafenib in a heterogeneous cohort of Child-Pugh A, B and C patients with advanced hepatocellular carcinoma in a clinical-practice scenario. Adult patients with hepatocellular carcinoma and treated with sorafenib 800 mg/day were eligible for this multicentric retrospective observational study. Safety analyses were performed and the effectiveness of sorafenib was assessed in terms of time to progression (TTP) and overall survival (OS). In total, 93 patients were enrolled: 14 were Child-Pugh A, 70 were Child-Pugh B and nine were Child-Pugh C. No differences in the frequency of grade 3 adverse events among different Child-Pugh classes were reported. In the overall cohort, median OS was 12 months (95% CI: 11.7-12.8 months) and TTP was 3 months (95% CI: 2.5-3.4 months). The Child-Pugh score had a statistically significant effect on TTP: 6.6 months in Child-Pugh A, 2.8 months in Child-Pugh B and 2.0 months in Child-Pugh C patients (p = 0.012). To our knowledge, this study includes the largest cohort of Caucasian Child-Pugh B and C patients ever treated with sorafenib. Although the retrospective design of this study does not allow reaching any definite conclusion, the results could lend some preliminary support to the safety and the effectiveness of sorafenib monotherapy in patients with Child-Pugh B and Child-Pugh C liver function.
authors.affiliation: Oncologia Medica, Università Campus Bio-Medico, Roma, Italy. d.santini@unicampus.it
authors.forename: Daniele
authors.initials: D
authors.lastname: Santini
authors.forename: Raffaele
authors.initials: R
authors.lastname: Addeo
authors.forename: Bruno
authors.initials: B
authors.lastname: Vincenzi
authors.forename: Alice
authors.initials: A
authors.lastname: Calvieri
authors.forename: Liliana
authors.initials: L
authors.lastname: Montella
authors.forename: Marianna
authors.initials: M
authors.lastname: Silletta
authors.forename: Michele
authors.initials: M
authors.lastname: Caraglia
authors.forename: Umberto
authors.initials: U
authors.lastname: Vespasiani
authors.forename: Antonio
authors.initials: A
authors.lastname: Picardi
authors.forename: Salvatore
authors.initials: S
authors.lastname: Del Prete
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Tonini
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000473:pathology; D015331:Cohort Studies; D018450:Disease Progression; D005260:Female; D006801:Humans; D007558:Italy; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000009:adverse effects* / Q000627:therapeutic use*; D012189:Retrospective Studies; D000077157:Sorafenib; D016896:Treatment Outcome
chemical_list: D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib