title: Efficacy of trabectedin in patients with advanced or metastatic alveolar soft-part sarcoma.
abstract: BACKGROUND
  Alveolar soft-part sarcoma (ASPS) is a rare sarcoma often occurring in young patients that is characterized by the unbalanced translocation der(17)t(X;17) (p11;q25). Although it usually shows an indolent clinical course, the prognosis is usually poor in advanced disease. Since standard chemotherapy regimens used in soft-tissue sarcomas lack efficacy in ASPS, new therapeutic options are needed. We investigated the efficacy of trabectedin, which has demonstrated activity in a variety of cancer types including some of the most prevalent translocation-related sarcomas.
  
  
  METHODS
  7 patients with metastatic or advanced ASPS treated with trabectedin in the Sarcoma Center Berlin-Brandenburg and the University Hospital of Greifswald were analyzed for median progression-free survival (mPFS), overall survival (OS), and therapy-related toxicity.
  
  
  RESULTS
  In 6 patients with documented disease progression, disease stabilization was reached with trabectedin; only 1 patient experienced progressive disease. The mPFS and OS were 7 months and 21 months, respectively, since the start of trabectedin treatment. Overall, no severe Common Toxicity Criteria (CTC) grade 3 or 4 toxicity was observed.
  
  
  CONCLUSIONS
  The poor prognosis of patients with ASPS has so far been due to the unavailability of effective systemic treatments. Trabectedin can be considered the only currently registered drug with clinical activity in this disease.
authors.affiliation: HELIOS Klinikum Bad Saarow, Department of Hematology, Oncology, and Palliative Care, Sarcoma Center Berlin-Brandenburg, Bad Saarow, Germany. daniel.pink@helios-kliniken.de
authors.forename: Daniel
authors.initials: D
authors.lastname: Pink
authors.forename: Janina
authors.initials: J
authors.lastname: Bertz-Lepel
authors.forename: Christoph
authors.initials: C
authors.lastname: Busemann
authors.forename: Ulrich
authors.initials: U
authors.lastname: Bitz
authors.forename: Peter
authors.initials: P
authors.lastname: Reichardt
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use; D004149:Dioxoles / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D018234:Sarcoma, Alveolar Soft Part / Q000188:drug therapy* / Q000473:pathology / Q000556:secondary*; D044005:Tetrahydroisoquinolines / Q000627:therapeutic use*; D000077606:Trabectedin; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D018906:Antineoplastic Agents, Alkylating; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin