title: Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents.
abstract: BACKGROUND
  The relative efficacy of allogeneic hematopoietic cell transplantation (allo-HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo-HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan.
  
  
  METHODS
  We compared a fludarabine (Flu) and melphalan (Mel)-based regimen (RTC; n = 34) with total body irradiation (TBI) and/or busulfan (Bu)-based conditioning (MAC; n = 102) in demographic- and disease-criteria-matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2).
  
  
  RESULTS
  The incidence of engraftment, early complications, grade II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25% vs. 26%) and non-relapse mortality (13% vs. 11%) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3-year overall survival (OS) rates after Flu/Mel-RTC and MAC were comparable (mean, 72% [range, 51-85%] and 68% [range, 58-77%], respectively).
  
  
  CONCLUSIONS
  The results suggest that the Flu/Mel-RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry-based analysis has several limitations, RTC deserves to be further investigated in prospective trials.
authors.affiliation: Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto, Japan; Division of Pediatrics, Matsushita Memorial Hospital, Moriguchi, Japan.
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Ishida
authors.forename: Souichi
authors.initials: S
authors.lastname: Adachi
authors.forename: Daiichiro
authors.initials: D
authors.lastname: Hasegawa
authors.forename: Yasuhiro
authors.initials: Y
authors.lastname: Okamoto
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Goto
authors.forename: Jiro
authors.initials: J
authors.lastname: Inagaki
authors.forename: Masami
authors.initials: M
authors.lastname: Inoue
authors.forename: Katsuyoshi
authors.initials: K
authors.lastname: Koh
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Keisei
authors.initials: K
authors.lastname: Kawa
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Atsuta
authors.forename: Kazuko
authors.initials: K
authors.lastname: Kudo
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000175:diagnosis* / Q000453:epidemiology / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007564:Japan; D015470:Leukemia, Myeloid, Acute / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D009364:Neoplasm Recurrence, Local / Q000175:diagnosis* / Q000453:epidemiology / Q000401:mortality; D009367:Neoplasm Staging; D011379:Prognosis; D012074:Remission Induction; D012189:Retrospective Studies; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D014916:Whole-Body Irradiation
chemical_list: D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008558:Melphalan
keywords: acute myeloid leukemia; childhood; hematopoietic stem cell transplantation; melphalan; reduced intensity conditioning; reduced toxicity conditioning