title: Efficacy and safety of sorafenib in a subset of patients with advanced soft tissue sarcoma from a Phase II randomized discontinuation trial.
abstract: OBJECTIVE
  Phase II multi-disease randomized discontinuation trial to assess the safety and efficacy of sorafenib including patients with advanced soft tissue sarcoma (STS).
  
  
  METHODS
  Sorafenib (400 mg twice daily) was initially administered for 12 weeks. Patients with: ≥25% tumour shrinkage continued sorafenib; ≥25% tumour growth discontinued; other patients were randomized and received sorafenib or placebo.
  
  
  RESULTS
  Twenty-six patients (median age 55 years) were enrolled. Common drug-related adverse events, including fatigue, hand-foot skin reaction, rash or gastrointestinal disturbances, were manageable, reversible and generally low grade. Fatigue, skin toxicity, nausea, diarrhoea and hypertension occurred at grade ≥3 in 19% of patients. After 12 weeks eight (31%) patients had not progressed. Three patients who experienced tumour shrinkage and continued on sorafenib, and five (19%) were randomized either to continue sorafenib or to receive placebo. Of the three patients randomized to sorafenib, one achieved a partial response and two had SD. Overall one patient achieved a partial response and three further patients achieved minor responses.
  
  
  CONCLUSIONS
  There was evidence of disease activity in STS as defined by tumor regressions including one objective partial response. Further investigation in STS is warranted.
authors.affiliation: The Royal Marsden Hospital, Downs Rd, Sutton, Surrey, SM2 5PT, UK.
authors.forename: Simon
authors.initials: S
authors.lastname: Pacey
authors.forename: Mark J
authors.initials: MJ
authors.lastname: Ratain
authors.forename: Keith T
authors.initials: KT
authors.lastname: Flaherty
authors.forename: Stanley B
authors.initials: SB
authors.lastname: Kaye
authors.forename: Lisa
authors.initials: L
authors.lastname: Cupit
authors.forename: Eric K
authors.initials: EK
authors.lastname: Rowinsky
authors.forename: Chenghua
authors.initials: C
authors.lastname: Xia
authors.forename: Peter J
authors.initials: PJ
authors.lastname: O'Dwyer
authors.forename: I R
authors.initials: IR
authors.lastname: Judson
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use; D001557:Benzenesulfonates / Q000009:adverse effects / Q000627:therapeutic use*; D003710:Demography; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D011725:Pyridines / Q000009:adverse effects / Q000627:therapeutic use*; D012509:Sarcoma / Q000000981:diagnostic imaging / Q000188:drug therapy*; D012983:Soft Tissue Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy*; D000077157:Sorafenib; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents; D001557:Benzenesulfonates; D010671:Phenylurea Compounds; D011725:Pyridines; D009536:Niacinamide; D000077157:Sorafenib