title: Sorafenib in metastatic uveal melanoma: efficacy, toxicity and health-related quality of life in a multicentre phase II study.
abstract: BACKGROUND
  The aim of the study was to analyse efficacy, safety, and health-related quality of life (HRQoL) for sorafenib treatment in patients with metastatic uveal melanoma.
  
  
  METHODS
  A multicentre, single-arm phase II trial was conducted. The primary objective was to determine the non-progression rate (RECIST) at 24 weeks for patients receiving sorafenib at a dose of 800 mg per day. Secondary endpoints included progression-free survival (PFS), overall survival (OS), toxicity, and HRQoL.
  
  
  RESULTS
  Thirty-two patients were included. Ten patients showed non-progression at 24 weeks (31.2%) without objective tumour responses. The estimated 24-week PFS was 31.2% (95% CI: 14.8%-47.6%) and the estimated 24-week OS was 62.5% (95% CI: 45.4%-79.6%). Ten patients (34.3%) had at least one grade 3 or 4 adverse reaction and 12 patients (41.4%) required dose modifications due to toxicity. At 24 weeks, no patient had an improvement in global HRQoL and 87.5% experienced a permanent increase in physical fatigue.
  
  
  CONCLUSIONS
  Sorafenib demonstrated non-progression at 24 weeks in 31.2% of patients. However, 41.4% of patients required dose modifications due to toxicity and no improvement in HRQoL was demonstrated.
authors.affiliation: Service d'ophtalmologie (Department of Ophthalmology), CHU Rennes, 35033 Rennes, France.|Faculté de Médecine (Faculty of Medicine), Université de Rennes 1, F35043 Rennes, France.|Centre universitaire d'ophtalmologie-Recherche (Ophthalmology-Research University Centre), Hôpital du Saint-Sacrement, Centre de Recherche du CHU de Québec (CHU Quebec Research Centre), G1S 4L8 Québec, Canada.|CHU Caen, F-14033 Caen France.
authors.forename: F
authors.initials: F
authors.lastname: Mouriaux
authors.affiliation: Institut Curie, Département d'Imagerie Médicale (Curie Institute, Department of Medical Imaging), F-75005 Paris, France.
authors.forename: V
authors.initials: V
authors.lastname: Servois
authors.affiliation: CHU Caen, F-14033 Caen France.
authors.forename: J J
authors.initials: JJ
authors.lastname: Parienti
authors.affiliation: Centre Eugène Marquis, F-35000 Rennes, France.
authors.forename: T
authors.initials: T
authors.lastname: Lesimple
authors.affiliation: Centre Antoine Lacassagne, F-06000 Nice, France.
authors.forename: A
authors.initials: A
authors.lastname: Thyss
authors.affiliation: Service Dermatologie (Dermatology Department), CHU de Bordeaux, F-33000 Bordeaux, France.
authors.forename: C
authors.initials: C
authors.lastname: Dutriaux
authors.affiliation: Centre Léon Bérard, F-69000 Lyon, France.
authors.forename: E M
authors.initials: EM
authors.lastname: Neidhart-Berard
authors.affiliation: Centre Oscar Lambret, F-59000 Lille, France.
authors.forename: N
authors.initials: N
authors.lastname: Penel
authors.affiliation: Centre François Baclesse, F-14000 Caen France.
authors.forename: C
authors.initials: C
authors.lastname: Delcambre
authors.affiliation: CHU Caen, F-14033 Caen France.
authors.forename: L
authors.initials: L
authors.lastname: Peyro Saint Paul
authors.affiliation: CHU Caen, F-14033 Caen France.
authors.forename: A D
authors.initials: AD
authors.lastname: Pham
authors.affiliation: Centre François Baclesse, F-14000 Caen France.
authors.forename: N
authors.initials: N
authors.lastname: Heutte
authors.affiliation: Département d'Oncologie Médicale, Institut Curie, (Department of Medical Oncology, Curie Institute), 75005 Paris, France.
authors.forename: S
authors.initials: S
authors.lastname: Piperno-Neumann
authors.affiliation: CHU Caen, F-14033 Caen France.|Centre François Baclesse, F-14000 Caen France.
authors.forename: F
authors.initials: F
authors.lastname: Joly
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008545:Melanoma / Q000188:drug therapy*; D009536:Niacinamide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000009:adverse effects* / Q000627:therapeutic use*; D011788:Quality of Life; D000077157:Sorafenib; D014604:Uveal Neoplasms / Q000188:drug therapy*; D000098943:Uveal Melanoma
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib