title: Final results from a multicenter, international, pivotal study of romidepsin in refractory cutaneous T-cell lymphoma.
abstract: OBJECTIVE
  The primary objective of this study was to confirm the efficacy of romidepsin in patients with treatment refractory cutaneous T-cell lymphoma (CTCL).
  
  
  METHODS
  This international, pivotal, single-arm, open-label, phase II study was conducted in patients with stage IB to IVA CTCL who had received one or more prior systemic therapies. Patients received romidepsin as an intravenous infusion at a dose of 14 mg/m(2) on days 1, 8, and 15 every 28 days. Response was determined by a composite assessment of total tumor burden including cutaneous disease, lymph node involvement, and blood (Sézary cells).
  
  
  RESULTS
  Ninety-six patients were enrolled and received one or more doses of romidepsin. Most patients (71%) had advanced stage disease (≥ IIB). The response rate was 34% (primary end point), including six patients with complete response (CR). Twenty-six of 68 patients (38%) with advanced disease achieved a response, including five CRs. The median time to response was 2 months, and the median duration of response was 15 months. A clinically meaningful improvement in pruritus was observed in 28 (43%) of 65 patients, including patients who did not achieve an objective response. Median duration of reduction in pruritus was 6 months. Drug-related adverse events were generally mild and consisted mainly of GI disturbances and asthenic conditions. Nonspecific, reversible ECG changes were noted in some patients.
  
  
  CONCLUSIONS
  Romidepsin has significant and sustainable single-agent activity (including improvement in pruritus) and an acceptable safety profile, making it an important therapeutic option for treatment refractory CTCL.
authors.affiliation: Guy's and St Thomas' National Health Service Foundation Trust, London, United Kingdom. sean.whittaker@kcl.ac.uk
authors.forename: Sean J
authors.initials: SJ
authors.lastname: Whittaker
authors.forename: Marie-France
authors.initials: MF
authors.lastname: Demierre
authors.forename: Ellen J
authors.initials: EJ
authors.lastname: Kim
authors.forename: Alain H
authors.initials: AH
authors.lastname: Rook
authors.forename: Adam
authors.initials: A
authors.lastname: Lerner
authors.forename: Madeleine
authors.initials: M
authors.lastname: Duvic
authors.forename: Julia
authors.initials: J
authors.lastname: Scarisbrick
authors.forename: Sunil
authors.initials: S
authors.lastname: Reddy
authors.forename: Tadeusz
authors.initials: T
authors.lastname: Robak
authors.forename: Jürgen C
authors.initials: JC
authors.lastname: Becker
authors.forename: Alexey
authors.initials: A
authors.lastname: Samtsov
authors.forename: William
authors.initials: W
authors.lastname: McCulloch
authors.forename: Youn H
authors.initials: YH
authors.lastname: Kim
mesh_terms: D000368:Aged; D000903:Antibiotics, Antineoplastic / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D001247:Asthenia / Q000139:chemically induced; D047630:Depsipeptides / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D019008:Drug Resistance, Neoplasm; D005260:Female; D006801:Humans; D007262:Infusions, Intravenous; D016410:Lymphoma, T-Cell, Cutaneous / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D009325:Nausea / Q000139:chemically induced; D009367:Neoplasm Staging; D011446:Prospective Studies; D012867:Skin / Q000187:drug effects* / Q000473:pathology; D012878:Skin Neoplasms / Q000188:drug therapy*; D016896:Treatment Outcome; D014839:Vomiting / Q000139:chemically induced
chemical_list: D000903:Antibiotics, Antineoplastic; D047630:Depsipeptides; C087123:romidepsin