title: Outcome of acute promyelocytic leukemia (APL) in children and adolescents: an analysis in two consecutive trials of the European APL Group.
abstract: OBJECTIVE
  Acute promyelocytic leukemia (APL) is rare in children. All-trans-retinoic acid (ATRA) combined with chemotherapy, the reference treatment of APL, is generally considered to produce similar results in children and adults. However, previously published childhood APL studies have generally analyzed all patients age < 18 years as a group, without further dividing according to age.
  
  
  METHODS
  We compared disease characteristics and outcomes of children (age ≤ 12 years), adolescents (13 to 18 years), and adults (> 18 years) included in two multicenter APL clinical trials (APL 93 and 2000 trials).
  
  
  RESULTS
  Of the 833 patients age ≤ 60 years included in the two trials, 26 (3%), 58 (7%), and 749 (90%) were children, adolescents, and adults, respectively. Children had significantly higher baseline WBC counts (P < .001). The complete remission (CR) rate (92%, 100%, and 94.5%, respectively) and 5-year cumulative incidence of relapse (CIR; 28%, 20%, and 23%, respectively) did not differ between children, adolescents, and adults, whereas adolescents had significantly better overall survival (OS; 5-year OS, 93.6% v 80.4% in adults and 80.4% in children; P = .03). However, in children age ≤ 4 years, the 5-year CIR was 52%, compared with 17.6% in children age 5 to 12 years (P = .006), although most of the younger children who relapsed experienced durable salvage with autologous or allogeneic stem-cell transplantation.
  
  
  CONCLUSIONS
  Adolescents and children age > 4 years with APL treated with ATRA and chemotherapy have outcomes at least as favorable as those of adults. Younger children seem to experience more relapses and may require reinforcement of first-line treatment.
authors.affiliation: Assistance Publique-Hôpitaux de Paris, Hôpital Avicenne, Bobigny, France.
authors.forename: Cecile
authors.initials: C
authors.lastname: Bally
authors.forename: Jehane
authors.initials: J
authors.lastname: Fadlallah
authors.forename: Guy
authors.initials: G
authors.lastname: Leverger
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.forename: Alain
authors.initials: A
authors.lastname: Robert
authors.forename: Andre
authors.initials: A
authors.lastname: Baruchel
authors.forename: Agnes
authors.initials: A
authors.lastname: Guerci
authors.forename: Christian
authors.initials: C
authors.lastname: Recher
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Raffoux
authors.forename: Xavier
authors.initials: X
authors.lastname: Thomas
authors.forename: Thierry
authors.initials: T
authors.lastname: Leblanc
authors.forename: Nadia
authors.initials: N
authors.lastname: Idres
authors.forename: Bruno
authors.initials: B
authors.lastname: Cassinat
authors.forename: Norbert
authors.initials: N
authors.lastname: Vey
authors.forename: Christine
authors.initials: C
authors.lastname: Chomienne
authors.forename: Herve
authors.initials: H
authors.lastname: Dombret
authors.forename: Miguel
authors.initials: M
authors.lastname: Sanz
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: Lionel
authors.initials: L
authors.lastname: Adès
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005060:Europe; D005260:Female; D006801:Humans; D015473:Leukemia, Promyelocytic, Acute / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality*; D008297:Male; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local / Q000175:diagnosis / Q000401:mortality*; D011379:Prognosis; D018570:Risk Assessment; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome; D014212:Tretinoin / Q000008:administration & dosage*
chemical_list: D014212:Tretinoin