title: Reports of clinical benefit of plitidepsin (Aplidine), a new marine-derived anticancer agent, in patients with advanced medullary thyroid carcinoma.
abstract: OBJECTIVE
  To assess clinical benefit of plitidepsin (Aplidine) in patients with advanced medullary thyroid carcinoma (MTC).
  
  
  METHODS
  We retrospectively reported the outcome of 10 patients with advanced MTC among 215 patients who have entered the phase I program with plitidepsin.
  
  
  RESULTS
  Median number of cycles was 5. Using World Health Organization criteria, 1 among 5 patients with measurable disease displayed a confirmed partial response, whereas 8 patients experienced a stable disease, and 1 patient had a progressive disease, corresponding to a disease control rate of 90%. Two patients treated at the maximum tolerated dose experienced muscular dose-limiting toxicity possibly related to palmitoyl transferase inhibition. One of these 2 patients was able to continue therapy with no dose reduction with the prophylactic addition of l-carnitine, which is used in the treatment of the carnitine palmitoyl transferase deficiency type 2.
  
  
  CONCLUSIONS
  Plitidepsin seems to be able to induce clinical benefit in patients with pretreated MTC, and its toxicity has been manageable at the recommended dose.
authors.affiliation: Department of Medical Oncology, Beaujon University Hospital, Clichy, France. christophe.letourneau@uhn.on.ca
authors.forename: Christophe
authors.initials: C
authors.lastname: Le Tourneau
authors.forename: Sandrine
authors.initials: S
authors.lastname: Faivre
authors.forename: Eva
authors.initials: E
authors.lastname: Ciruelos
authors.forename: María J
authors.initials: MJ
authors.lastname: Domínguez
authors.forename: José A
authors.initials: JA
authors.lastname: López-Martín
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Izquierdo
authors.forename: José
authors.initials: J
authors.lastname: Jimeno
authors.forename: Eric
authors.initials: E
authors.lastname: Raymond
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001859:Bone Neoplasms / Q000188:drug therapy / Q000556:secondary; D018276:Carcinoma, Medullary / Q000188:drug therapy* / Q000556:secondary; D047630:Depsipeptides / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008113:Liver Neoplasms / Q000188:drug therapy / Q000556:secondary; D008175:Lung Neoplasms / Q000188:drug therapy / Q000556:secondary; D008297:Male; D008387:Marine Toxins / Q000627:therapeutic use*; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D010456:Peptides, Cyclic; D015996:Survival Rate; D013964:Thyroid Neoplasms / Q000188:drug therapy* / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D047630:Depsipeptides; D008387:Marine Toxins; D010456:Peptides, Cyclic; C098980:plitidepsin