title: Outcome of hematopoietic stem cell transplantation for pediatric patients with therapy-related acute myeloid leukemia or myelodysplastic syndrome.
abstract: BACKGROUND
  Therapy-related myelodysplastic syndrome (t-MDS) and acute myeloid leukemia (t-AML) carry a poor prognosis. We analyzed the results of allogeneic HSCT in 38 children to determine which factors, if any, affected outcome.
  
  
  METHODS
  The effects of demographic, donor, and disease-related factors were analyzed to determine their effects on overall and disease-free survival (OS, DFS), relapse, and non-relapse mortality (NRM).
  
  
  RESULTS
  OS and DFS for t-AML and t-MDS were similar. Three-year OS and EFS were the same (15.4 +/- 5.8%) and the 3-year NRM was 59.6 +/- 8.4%. The 1-year cumulative risk of grade III-IV acute graft-versus-host disease (GVHD) and relapse were 23.7 +/- 7.0% and 18.7 +/- 6.5%, respectively. The percentage of pre-transplant bone marrow (BM) blasts was positively associated with relapse (P = 0.05), while the percentage of BM blasts at diagnosis of therapy-related disease tended to associate with NRM (P = 0.07). Alternative donor and matched sibling donor grafts had similar outcomes. NRM was higher among patients who did not develop acute GVHD as compared to those with grade 1-2 acute GVHD (69.2 +/- 14.2% vs. +/- 12.7%, respectively), while NRM was 100% in patients with grade III-IV acute GVHD (P = 0.007).
  
  
  CONCLUSIONS
  The percentage of BM blasts is associated with relapse in these disorders. High rates of NRM negatively impact the outcome of allogeneic HSCT for children with t-AML and t-MDS. Future studies should focus on reducing NRM.
authors.affiliation: Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105-2794, USA. paul.woodard@stjude.org
authors.forename: Paul
authors.initials: P
authors.lastname: Woodard
authors.forename: Raymond
authors.initials: R
authors.lastname: Barfield
authors.forename: Gregory
authors.initials: G
authors.lastname: Hale
authors.forename: Edwin
authors.initials: E
authors.lastname: Horwitz
authors.forename: Wing
authors.initials: W
authors.lastname: Leung
authors.forename: Raul
authors.initials: R
authors.lastname: Ribeiro
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Rubnitz
authors.forename: Deo Kumar
authors.initials: DK
authors.lastname: Srivistava
authors.forename: Xin
authors.initials: X
authors.lastname: Tong
authors.forename: Usman
authors.initials: U
authors.lastname: Yusuf
authors.forename: Susana
authors.initials: S
authors.lastname: Raimondi
authors.forename: Ching-Hon
authors.initials: CH
authors.lastname: Pui
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
authors.forename: John M
authors.initials: JM
authors.lastname: Cunningham
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000401:mortality / Q000628:therapy*; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000628:therapy*; D016609:Neoplasms, Second Primary / Q000401:mortality / Q000628:therapy*; D016896:Treatment Outcome