title: Sorafenib in patients with progressive epithelioid hemangioendothelioma: a phase 2 study by the French Sarcoma Group (GSF/GETO).
abstract: BACKGROUND
  There is no standard treatment for progressive epithelioid hemangioendothelioma (EHE). To investigate the significant vascularization of EHE, the activity/toxicity of sorafenib in patients with progressive EHE was explored.
  
  
  METHODS
  In this multicenter, 1-stage, phase 2 trial of sorafenib (800 mg daily), the primary endpoint, which was chosen by default, was the 9-month progression-free rate. All patients had documented progressive disease at the time of study entry.
  
  
  RESULTS
  Fifteen patients were enrolled between June 2009 and February 2011. The median age was 57 years (range, 31-76 years), and the ratio of men to women was 9:6. The performance status was zero in 10 patients and 1 in 5 patients. Twelve patients had metastases, mainly in the lung (12 patients), liver (5 patients), and bone (3 patients). Five patients had received prior chemotherapy (doxorubicin in 5 patients and taxane in 3 patients). The median sorafenib treatment duration was 124 days (range, from 27 to >271 days). Seven patients required dose reductions or transient treatment discontinuation. The 9-month progression-free rate was 30.7% (4 of 13 patients). The 2-month, 4-month, and 6-month progression-free rate was 84.6% (11 of 13 patients), 46.4% (6 of 13 patients), and 38.4% (5 of 13 patients), respectively. Two partial responses were observed that lasted 2 months and 9 months.
  
  
  CONCLUSIONS
  Further clinical trials exploring sorafenib as treatment of progressive EHE are needed.
authors.affiliation: Department of Medical Oncology, Claudius Regaud Institute, Toulouse, France.
authors.forename: Christine
authors.initials: C
authors.lastname: Chevreau
authors.forename: Axel
authors.initials: A
authors.lastname: Le Cesne
authors.forename: Isabelle
authors.initials: I
authors.lastname: Ray-Coquard
authors.forename: Antoine
authors.initials: A
authors.lastname: Italiano
authors.forename: Angela
authors.initials: A
authors.lastname: Cioffi
authors.forename: Nicolas
authors.initials: N
authors.lastname: Isambert
authors.forename: Yves Marie
authors.initials: YM
authors.lastname: Robin
authors.forename: Charles
authors.initials: C
authors.lastname: Fournier
authors.forename: Stéphanie
authors.initials: S
authors.lastname: Clisant
authors.forename: Loic
authors.initials: L
authors.lastname: Chaigneau
authors.forename: Jacques-Olivier
authors.initials: JO
authors.lastname: Bay
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Bompas
authors.forename: Eric
authors.initials: E
authors.lastname: Gauthier
authors.forename: Jean Y
authors.initials: JY
authors.lastname: Blay
authors.forename: Nicolas
authors.initials: N
authors.lastname: Penel
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D005602:France; D006258:Head and Neck Neoplasms / Q000188:drug therapy; D018323:Hemangioendothelioma, Epithelioid / Q000188:drug therapy* / Q000556:secondary; D006801:Humans; D008113:Liver Neoplasms / Q000188:drug therapy; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D035583:Rare Diseases; D000077157:Sorafenib; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D009536:Niacinamide; D000077157:Sorafenib
keywords: epithelioid hemangioendothelioma; orphan diseases; phase 2 trial; sarcoma; sorafenib