title: Trabectedin for inoperable or recurrent soft tissue sarcoma in adult patients: a retrospective cohort study.
abstract: BACKGROUND
  Trabectedin is an antineoplastic agent used for patients with soft tissue sarcoma (STS) who fail standard-of-care treatment. Real-world data of its performance is scarce. This study evaluates the safety and effectiveness of trabectedin for patients with advanced STS who were treated at a high-volume sarcoma center.
  
  
  METHODS
  A retrospective chart review was performed on 77 patients treated with trabectedin (24 h infusion q3w) between 01/2005 and 05/2014. Data regarding safety, objective radiological response, progression-free and overall survival were analyzed.
  
  
  RESULTS
  Median age at treatment onset was 52y [interquartile range (IQR): 45-61y]. Tumors included leiomyosarcoma (41.6%), liposarcoma (18.2%), and synovial sarcoma (13%). Trabectedin was provided as ≥ third-line chemotherapy in 71.4%. Median number of cycles was 2 (range: 1-17). Dose reduction and treatment delays occurred in 19.5 and 40.3%, respectively. Toxicities occurred in 78%, primarily for neutropenia or elevated liver enzymes. Two patients died secondary to trabectedin-induced rhabdomyolysis. Treatment was discontinued because of disease progression (84.7%), toxicity (10%), and patient preference (5%). Partial response or stable disease occurred in 14.1 and 33.8%, respectively, while 52.1% developed progressive disease. Median progression-free survival was 1.3 m (IQR: 0.7-3.5 m) and was significantly higher in patients lacking severe toxicities or progressive disease. Median overall survival was 6.7 m (IQR: 2.3-12.7 m) and was significantly higher in patients with leiomyosarcoma or liposarcoma relative to other histologies.
  
  
  CONCLUSIONS
  Trabectedin has an acceptable safety profile as an anti-tumor agent. Our data further suggest there may be some benefit in using trabectedin particularly in patients with leiomyo- or liposarcoma who failed standard-of-care agents.
authors.affiliation: Division of General Surgery, Department of Surgery, University of Toronto, Toronto, ON, Canada. fernando.angarita@mail.utoronto.ca.
authors.forename: Fernando A
authors.initials: FA
authors.lastname: Angarita
authors.affiliation: Division of General Surgery, Department of Surgery, University of Toronto, Toronto, ON, Canada. amandajcannell@gmail.com.
authors.forename: Amanda J
authors.initials: AJ
authors.lastname: Cannell
authors.affiliation: Department of Medical Oncology, Mount Sinai Hospital, Toronto, ON, Canada. albiruni.razak@uhn.ca.|Department of Medical Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada. albiruni.razak@uhn.ca.|Department of Medicine, University of Toronto, Toronto, ON, Canada. albiruni.razak@uhn.ca.
authors.forename: Albiruni R
authors.initials: AR
authors.lastname: Abdul Razak
authors.affiliation: Department of Pathology and Laboratory Medicine, Mount Sinai Hospital, Toronto, ON, Canada. BDickson@mtsinai.on.ca.|Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada. BDickson@mtsinai.on.ca.
authors.forename: Brendan C
authors.initials: BC
authors.lastname: Dickson
authors.affiliation: Department of Medical Oncology, Mount Sinai Hospital, Toronto, ON, Canada. martin.blackstein@utoronto.ca.|Department of Medical Oncology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada. martin.blackstein@utoronto.ca.|Department of Medicine, University of Toronto, Toronto, ON, Canada. martin.blackstein@utoronto.ca.
authors.forename: Martin E
authors.initials: ME
authors.lastname: Blackstein
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage*; D015331:Cohort Studies; D004149:Dioxoles / Q000008:administration & dosage* / Q000009:adverse effects; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D012189:Retrospective Studies; D012509:Sarcoma / Q000188:drug therapy* / Q000473:pathology; D044005:Tetrahydroisoquinolines / Q000008:administration & dosage* / Q000009:adverse effects; D000077606:Trabectedin
chemical_list: D000970:Antineoplastic Agents; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin