title: A phase II study of lenalidomide alone in relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes with chromosome 5 abnormalities.
abstract: This phase II study assessed the efficacy and safety of lenalidomide in patients with relapsed/refractory acute myeloid leukemia (N = 18) and high-risk myelodysplastic syndrome (N = 9) with chromosome 5 abnormalities. The overall complete remission rate with or without platelet recovery was 7% (2/27). Activity of lenalidomide was limited to patients with noncomplex cytogenetics.
  
  
  BACKGROUND
  Lenalidomide is effective in low-risk myelodysplastic syndromes (MDS) with deletion 5q. We conducted a phase II study to evaluate the safety and efficacy of lenalidomide in patients with relapsed/refractory acute myeloid leukemia (AML) and high-risk MDS with any chromosome 5 abnormality.
  
  
  METHODS
  Eighteen adults with AML and 9 with high-risk MDS were enrolled. Lenalidomide was given orally at doses 5 to 25 mg daily for 21 days of a 28-day cycle until disease progression or unacceptable adverse event.
  
  
  RESULTS
  Median age for all 27 patients was 64 years (range, 39-88 years) with a median of 2 previous therapies (range, 1-6 lines). Two patients (7%) with AML and 5q deletion and +8 cytogenetic abnormality in 2 separate clones achieved complete remission (CR) or CR without platelet recovery (CRp). Response durations were 4 and 6 months, respectively. No responses were seen in patients with chromosome 5 abnormality in a complex cytogenetic background. Twenty patients (74%) developed neutropenic fever or infection requiring hospitalization.
  
  
  CONCLUSIONS
  Clinical activity of lenalidomide as single agent in AML and high-risk MDS with chromosome 5 abnormalities appears to be limited to patients with noncomplex cytogenetics.
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Yiming
authors.initials: Y
authors.lastname: Chen
authors.forename: Hagop
authors.initials: H
authors.lastname: Kantarjian
authors.forename: Zeev
authors.initials: Z
authors.lastname: Estrov
authors.forename: Stefan
authors.initials: S
authors.lastname: Faderl
authors.forename: Farhad
authors.initials: F
authors.lastname: Ravandi
authors.forename: Kristy
authors.initials: K
authors.lastname: Rey
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.forename: Gautam
authors.initials: G
authors.lastname: Borthakur
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002869:Chromosome Aberrations; D002872:Chromosome Deletion; D002895:Chromosomes, Human, Pair 5; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000235:genetics*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics*; D012008:Recurrence; D012307:Risk Factors; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D013792:Thalidomide; D000077269:Lenalidomide