title: Pralatrexate is an effective treatment for relapsed or refractory transformed mycosis fungoides: a subgroup efficacy analysis from the PROPEL study.
abstract: Transformed mycosis fungoides (tMF) is an aggressive disease with a median survival of 12-24 months. In this retrospective analysis of 12 patients with tMF, treatment with pralatrexate resulted in an objective response of 25% per independent central review and 58% per investigator assessment. Pralatrexate was well tolerated, with no toxicity-related discontinuations, which makes this an additional option for tMF treatment.
  
  
  BACKGROUND
  Transformed mycosis fungoides (tMF) is an aggressive disease, with poor prognosis and a median survival of 24 months.
  
  
  METHODS
  In the Pralatrexate in Patients With Relapsed or Refractory Peripheral T-cell Lymphoma (PROPEL) study, 12 patients with tMF were treated with a median of 10 pralatrexate doses (starting dose of 30 mg/m(2)) administered weekly for 6 weeks in a 7-week cycle. The median number of prior systemic therapies was 3.
  
  
  RESULTS
  This retrospective analysis showed that the objective response rate in this subgroup was 25% (n = 3) per independent central review and 58% (n = 7) per investigator assessment, with this discrepancy likely attributed to challenges with photodocumentation of cutaneous lesions. The median duration of response and the median progression-free survival were 2.2 and 1.7 months, respectively, per central review, whereas median duration of response was 4.4 months, and median progression-free survival was 5.3 months per investigator assessment. Median survival was 13 months. Grade 1-3 mucositis was reported in 7 (58%) patients. Grade 4 adverse events were fatigue (n = 1) and thrombocytopenia (n = 1). Pralatrexate was well tolerated, with no toxicity-related discontinuations.
  
  
  CONCLUSIONS
  Based on these results, pralatrexate may be a treatment option for patients with relapsed or refractory tMF.
authors.affiliation: Department of Medical Oncology and Bone Marrow Transplantation, Yale Cancer Center, New Haven, CT 06510, USA. foss@yale.edu
authors.forename: Francine
authors.initials: F
authors.lastname: Foss
authors.forename: Steven M
authors.initials: SM
authors.lastname: Horwitz
authors.forename: Bertrand
authors.initials: B
authors.lastname: Coiffier
authors.forename: Nancy
authors.initials: N
authors.lastname: Bartlett
authors.forename: Leslie
authors.initials: L
authors.lastname: Popplewell
authors.forename: Barbara
authors.initials: B
authors.lastname: Pro
authors.forename: Lauren C
authors.initials: LC
authors.lastname: Pinter-Brown
authors.forename: Andrei
authors.initials: A
authors.lastname: Shustov
authors.forename: Richard R
authors.initials: RR
authors.lastname: Furman
authors.forename: Corinne
authors.initials: C
authors.lastname: Haioun
authors.forename: Tony
authors.initials: T
authors.lastname: Koutsoukos
authors.forename: Owen A
authors.initials: OA
authors.lastname: O'Connor
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000630:Aminopterin / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D018572:Disease-Free Survival; D005493:Folic Acid Antagonists / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D008875:Middle Aged; D009182:Mycosis Fungoides / Q000188:drug therapy*; D011379:Prognosis; D012008:Recurrence; D012189:Retrospective Studies; D016019:Survival Analysis
chemical_list: C418863:10-propargyl-10-deazaaminopterin; D005493:Folic Acid Antagonists; D000630:Aminopterin