title: A phase III, randomized, double-blind, placebo-controlled study of peldesine (BCX-34) cream as topical therapy for cutaneous T-cell lymphoma.
abstract: BACKGROUND
  The purine nucleoside phosphorylase inhibitor peldesine is a new agent being evaluated as a T-cell inhibitor.
  
  
  OBJECTIVE
  We attempted to determine the efficacy of peldesine (BCX-34) in a 1% dermal cream formulation as a treatment for cutaneous T-cell lymphoma (CTCL).
  
  
  METHODS
  Ninety patients with patch and plaque phase CTCL, histologically confirmed by a referee dermatopathologist, were enrolled in a randomized, double-blind, placebo-controlled study. BCX-34 dermal cream 1% or the vehicle cream (as a placebo control) was applied twice daily to the entire skin surface for up to 24 weeks. Efficacy of the topical therapy was assessed in terms of complete or partial (> or = 50%) clearing of patches and plaques.
  
  
  RESULTS
  Of the 89 patients able to be examined, 43 received BCX-34 and 46 received the placebo vehicle cream. One patient withdrew early and was not analyzed. The two groups were well balanced for potential prognostic factors. A total of 28% (12/43) of the patients treated with BCX-34 showed a response, but 24% (11/46) of patients who received vehicle also responded (P =.677).
  
  
  CONCLUSIONS
  Although BCX-34 dermal cream 1% was not significantly better than the control as therapy for patch and plaque-phase CTCL, this appears to be the first published placebo-controlled trial evaluating treatment for CTCL. Whether the vehicle cream has more than a placebo therapeutic effect is unclear. The relatively high (24%) placebo response rate should be kept in mind in assessing other treatments for early-stage CTCL.
authors.affiliation: Department of Dermatology, University of Texas, M. D. Anderson Cancer Center, Houston, TX, USA.
authors.forename: M
authors.initials: M
authors.lastname: Duvic
authors.forename: E A
authors.initials: EA
authors.lastname: Olsen
authors.forename: G A
authors.initials: GA
authors.lastname: Omura
authors.forename: J C
authors.initials: JC
authors.lastname: Maize
authors.forename: E C
authors.initials: EC
authors.lastname: Vonderheid
authors.forename: C A
authors.initials: CA
authors.lastname: Elmets
authors.forename: J L
authors.initials: JL
authors.lastname: Shupack
authors.forename: M F
authors.initials: MF
authors.lastname: Demierre
authors.forename: T M
authors.initials: TM
authors.lastname: Kuzel
authors.forename: D Y
authors.initials: DY
authors.lastname: Sanders
mesh_terms: D000279:Administration, Cutaneous; D000328:Adult; D000368:Aged; D004311:Double-Blind Method; D004791:Enzyme Inhibitors / Q000008:administration & dosage / Q000494:pharmacology*; D005260:Female; D006147:Guanine / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000494:pharmacology*; D006801:Humans; D016410:Lymphoma, T-Cell, Cutaneous / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D010919:Placebos; D016896:Treatment Outcome
chemical_list: D004791:Enzyme Inhibitors; D010919:Placebos; D006147:Guanine; C104194:peldesine