title: Phase II study of clofarabine in pediatric patients with refractory or relapsed acute lymphoblastic leukemia.
abstract: OBJECTIVE
  To evaluate the efficacy and safety of clofarabine, a novel deoxyadenosine analog, in pediatric patients with refractory or relapsed acute lymphoblastic leukemia (ALL).
  
  
  METHODS
  In a phase II, open-label, multicenter study, 61 pediatric patients with refractory or relapsed ALL received clofarabine 52 mg/m2 intravenously over 2 hours daily for 5 days, every 2 to 6 weeks. The median age was 12 years (range, 1 to 20 years), and the median number of prior regimens was three (range, two to six regimens).
  
  
  RESULTS
  The response rate was 30%, consisting of seven complete remissions (CR), five CRs without platelet recovery (CRp), and six partial remissions. Remissions were durable enough to allow patients to proceed to hematopoietic stem-cell transplantation (HSCT) after clofarabine. Median CR duration in patients who did not receive HSCT was 6 weeks, with four patients maintaining CR or CRp for 8 weeks or more (8+, 12, 37+, and 48 weeks) on clofarabine therapy alone. The most common adverse events of grade > or = 3 were febrile neutropenia, anorexia, hypotension, and nausea.
  
  
  CONCLUSIONS
  Clofarabine is active as a single agent in pediatric patients with multiple relapsed or refractory ALL. The toxicity profile is as expected in this heavily pretreated patient population. Studies exploring rational combinations of clofarabine with other agents are ongoing in an effort to maximize clinical benefit.
authors.affiliation: Department of Hematology-Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA. sima.jeha@stjude.org
authors.forename: Sima
authors.initials: S
authors.lastname: Jeha
authors.forename: Paul S
authors.initials: PS
authors.lastname: Gaynon
authors.forename: Bassem I
authors.initials: BI
authors.lastname: Razzouk
authors.forename: Janet
authors.initials: J
authors.lastname: Franklin
authors.forename: Richard
authors.initials: R
authors.lastname: Kadota
authors.forename: Violet
authors.initials: V
authors.lastname: Shen
authors.forename: Lori
authors.initials: L
authors.lastname: Luchtman-Jones
authors.forename: Michael
authors.initials: M
authors.lastname: Rytting
authors.forename: Lisa R
authors.initials: LR
authors.lastname: Bomgaars
authors.forename: Susan
authors.initials: S
authors.lastname: Rheingold
authors.forename: Kim
authors.initials: K
authors.lastname: Ritchey
authors.forename: Edythe
authors.initials: E
authors.lastname: Albano
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Arceci
authors.forename: Stewart
authors.initials: S
authors.lastname: Goldman
authors.forename: Timothy
authors.initials: T
authors.lastname: Griffin
authors.forename: Arnold
authors.initials: A
authors.lastname: Altman
authors.forename: Bruce
authors.initials: B
authors.lastname: Gordon
authors.forename: Laurel
authors.initials: L
authors.lastname: Steinherz
authors.forename: Steven
authors.initials: S
authors.lastname: Weitman
authors.forename: Peter
authors.initials: P
authors.lastname: Steinherz
mesh_terms: D000227:Adenine Nucleotides; D000293:Adolescent; D000328:Adult; D001087:Arabinonucleosides / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D000077866:Clofarabine; D005260:Female; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy* / Q000473:pathology; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D000227:Adenine Nucleotides; D001087:Arabinonucleosides; D000077866:Clofarabine