title: Efficacy of trabectedin in advanced soft tissue sarcoma: beyond lipo- and leiomyosarcoma.
abstract: OBJECTIVE
  Trabectedin is effective in leiomyosarcoma and liposarcoma, especially the myxoid variant, related to the presence of the FUS-CHOP transcript. We evaluated the efficacy of trabectedin in specific subgroups of patients with soft tissue sarcomas (STS).
  
  
  METHODS
  Seventy-two patients with advanced anthracycline-pretreated STS, who received trabectedin at a dose of 1.5 mg/m(2) every 3 weeks by continuous 24-hour infusion, were retrospectively analyzed. Best response rate according to Response Evaluation Criteria In Solid Tumors (RECIST) criteria and severe adverse events (AEs) according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE v4.02) were evaluated. Secondary endpoints included progression-free survival and overall survival (OS).
  
  
  RESULTS
  Median age was 48 (range, 20-75) years, with a median Eastern Cooperative Oncology Group performance status of 0. The median number of previous chemotherapy regimens was 1 (range, 0-5). Median number of trabectedin cycles was 3 (range, 1-17). About 69/72 patients (95.8%) were evaluable for response: 9 patients (13%) achieved partial response and 26 (37.7%) stable disease. According to histotype, clinical benefit (partial response + stable disease) was reported in synovial sarcoma (n=5), retroperitoneal liposarcoma (n=10), myxoid liposarcoma (n=5), leiomyosarcoma (n=8), high-grade undifferentiated pleomorphic sarcoma (n=5), Ewing/peripheral primitive neuroectodermal tumor (n=1), and malignant peripheral nerve sheath tumor (n=1). Any grade AEs were noncumulative, reversible, and manageable. G3/G4 AEs included anemia (n=1, 1.4%), neutropenia (n=7, 9.6%), liver toxicity (n=6, 8.3%), and fatigue (n=2, 2.8%). With a median follow-up time of 11 (range, 2-23) months, median progression-free survival and OS of the entire cohort were 2.97 months and 16.5 months, respectively.
  
  
  CONCLUSIONS
  Our experience confirms trabectedin as an effective therapeutic option for metastatic lipo- and leiomyosarcoma and suggests promise in synovial sarcomas and high-grade undifferentiated pleomorphic sarcoma.
authors.affiliation: Department of Medical Oncology and Haematology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Rita
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: Department of Medical Oncology and Haematology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Marrari
authors.affiliation: Department of Medical Oncology and Haematology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Marchetti
authors.affiliation: Department of Surgical Oncology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Mussi
authors.affiliation: Department of Radiology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Luca
authors.initials: L
authors.lastname: Balzarini
authors.affiliation: Department of Radiology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Fabio Romano
authors.initials: FR
authors.lastname: Lutman
authors.affiliation: Department of Surgical Oncology, Orthopaedic Institute "G. Pini", Milan, Italy.
authors.forename: Primo
authors.initials: P
authors.lastname: Daolio
authors.affiliation: Department of Surgical Oncology, Orthopaedic Institute "G. Pini", Milan, Italy.
authors.forename: Stefano
authors.initials: S
authors.lastname: Bastoni
authors.affiliation: Department of Medical Oncology and Haematology, Humanitas Cancer Center, IRCCS, Rozzano, Italy ; Department of Medical Oncology, Adelaide and Meath Hospital, Incorporating the National Children's Hospital (AMNCH), Dublin, Ireland.
authors.forename: Alexia Francesca
authors.initials: AF
authors.lastname: Bertuzzi
authors.affiliation: Department of Surgical Oncology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Vittorio
authors.initials: V
authors.lastname: Quagliuolo
authors.affiliation: Department of Medical Oncology and Haematology, Humanitas Cancer Center, IRCCS, Rozzano, Italy.
authors.forename: Armando
authors.initials: A
authors.lastname: Santoro
mesh_terms: D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000009:adverse effects / Q000627:therapeutic use*; D004149:Dioxoles / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007890:Leiomyosarcoma / Q000188:drug therapy / Q000473:pathology; D008080:Liposarcoma / Q000188:drug therapy / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009362:Neoplasm Metastasis; D012189:Retrospective Studies; D012509:Sarcoma / Q000188:drug therapy* / Q000473:pathology; D015996:Survival Rate; D044005:Tetrahydroisoquinolines / Q000009:adverse effects / Q000627:therapeutic use*; D000077606:Trabectedin; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D018906:Antineoplastic Agents, Alkylating; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin
keywords: leiomyosarcoma; liposarcoma; soft tissue sarcoma; trabectedin