title: Ridaforolimus as a single agent in advanced endometrial cancer: results of a single-arm, phase 2 trial.
abstract: BACKGROUND
  This open-label, multicentre, phase 2 trial evaluated the efficacy and tolerability of the mammalian target of rapamycin inhibitor ridaforolimus in women with advanced endometrial cancer.
  
  
  METHODS
  Women with measurable recurrent or persistent endometrial cancer and documented disease progression were treated with ridaforolimus 12.5 mg intravenously once daily for 5 consecutive days every 2 weeks in a 4-week cycle. The primary end point was clinical benefit response, defined as an objective response or prolonged stable disease of 16 weeks or more.
  
  
  RESULTS
  In all, 45 patients were treated with single-agent ridaforolimus. Clinical benefit was achieved by 13 patients (29%), including 5 (11%) with confirmed partial responses and 8 (18%) with prolonged stable disease. All patients with clinical benefit response received ridaforolimus for more than 4 months. In this heavily pretreated population, the 6-month progression-free survival was 18%. Ridaforolimus was generally well tolerated: adverse events were predictable and manageable, consistent with prior studies in other malignancies. Overall, the most common adverse events were diarrhoea (58%) and mouth sores (56%); most common grade 3 or higher adverse events were anaemia (27%) and hyperglycaemia (11%).
  
  
  CONCLUSIONS
  Single-agent ridaforolimus has antitumor activity and acceptable tolerability in advanced endometrial cancer patients. Further clinical evaluation of ridaforolimus is warranted.
authors: Division of Gynecology, University of Milan-Bicocca, Milano, Italy. nicoletta.colombo@ieo.it
authors: N
authors: N
authors: Colombo
authors: D S
authors: DS
authors: McMeekin
authors: P E
authors: PE
authors: Schwartz
authors: C
authors: C
authors: Sessa
authors: P A
authors: PA
authors: Gehrig
authors: R
authors: R
authors: Holloway
authors: P
authors: P
authors: Braly
authors: D
authors: D
authors: Matei
authors: A
authors: A
authors: Morosky
authors: P F
authors: PF
authors: Dodion
authors: M H
authors: MH
authors: Einstein
authors: F
authors: F
authors: Haluska
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D016889:Endometrial Neoplasms / Q000188:drug therapy* / Q000473:pathology; D005260:Female; D006801:Humans; D008875:Middle Aged; D019233:Retreatment; D020123:Sirolimus / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors*
chemical_list: D018906:Antineoplastic Agents, Alkylating; C515074:ridaforolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus
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