Outcome of hematopoietic stem cell transplantation for pediatric patients with therapy-related acute myeloid leukemia or myelodysplastic syndrome.
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.
Woodard
Paul
P
Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105-2794, USA. paul.woodard@stjude.org
Barfield
Raymond
R
Hale
Gregory
G
Horwitz
Edwin
E
Leung
Wing
W
Ribeiro
Raul
R
Rubnitz
Jeffrey
J
Srivistava
Deo Kumar
DK
Tong
Xin
X
Yusuf
Usman
U
Raimondi
Susana
S
Pui
Ching-Hon
CH
Handgretinger
Rupert
R
Cunningham
John M
JM
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