title: A phase II evaluation of trabectedin in the treatment of advanced, persistent, or recurrent uterine leiomyosarcoma: a gynecologic oncology group study.
abstract: OBJECTIVE
  To estimate activity and safety of trabectedin 1.5 mg/m2 IV over 24 hours every 3 weeks (1 cycle) in uterine leiomyosarcoma.
  
  
  METHODS
  Patients with chemotherapy naive, advanced, persistent or recurrent uterine leiomyosarcoma, acceptable organ function and PS≤2 were eligible. A two-stage design was utilized. Three responses were required in the first stage to initiate the second stage; the target sample size was 40 for the combined stages. If the true response rate was 10%, the study design provided a 95% chance of correctly classifying the treatment as "inactive." Conversely, if the true response rate was 30%, then the average probability of correctly classifying the treatment as active would be 90%.
  
  
  RESULTS
  Twenty patients were eligible and evaluable. The median number of cycles was 10 (123 total cycles, range 2-29). The number of patients with partial responses was 2 (10%; 95% confidence interval of 1.2%-31.7%). Response durations were 3.3 and 5.7 months. Ten patients had stable disease (50%). The median progression-free survival (PFS) and overall survival were 5.8 months and greater than 26.1 months (median not reached), respectively. Observed grade 3/4 toxicity included: neutropenia 16/20 (1 infection); thrombocytopenia 3/20; metabolic 3/20; anemia, gastrointestinal and vascular 1/20 each. There were no treatment related deaths nor cases of liver failure.
  
  
  CONCLUSIONS
  Although a second stage of accrual was not indicated based on the overall response rate, the drug was well tolerated.
authors: Creighton University School of Medicine at St. Joseph's Hospital and Medical Center, Phoenix, AZ 85013, USA. Bradley.monk@chw.edu
authors: Bradley J
authors: BJ
authors: Monk
authors: John A
authors: JA
authors: Blessing
authors: Daron G
authors: DG
authors: Street
authors: Carolyn Y
authors: CY
authors: Muller
authors: James J
authors: JJ
authors: Burke
authors: Martee L
authors: ML
authors: Hensley
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D018906:Antineoplastic Agents, Alkylating / Q000009:adverse effects / Q000627:therapeutic use*; D004149:Dioxoles / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007262:Infusions, Intravenous; D007890:Leiomyosarcoma / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D011446:Prospective Studies; D015996:Survival Rate; D044005:Tetrahydroisoquinolines / Q000009:adverse effects / Q000627:therapeutic use*; D000077606:Trabectedin; D014594:Uterine Neoplasms / Q000188:drug therapy* / Q000473:pathology
chemical_list: D018906:Antineoplastic Agents, Alkylating; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin