title: Favorable outcomes after allogeneic hematopoietic stem cell transplantation in children with high-risk or advanced acute myeloid leukemia.
abstract: The role of allogeneic hematopoietic stem cell transplantation (HSCT), including transplantation from an alternative donor (AD), has not been clearly defined for children with high-risk or advanced acute myeloid leukemia (AML). We retrospectively reviewed outcomes in 29 children (median age at HSCT, 6.7 y; range, 1.0-16.2 y) with high-risk or advanced AML who underwent allogeneic HSCT at the Asan Medical Center between 1998 and 2008. Donors included a matched sibling donor (MSD) for 7 patients (24%), an unrelated volunteer for 21 patients (72%), and a haploidentical mother for 1 patient (3%). The 3-year estimates of overall survival and event-free survival (EFS) were 77% [95% confidence interval (CI), 65%-99%] and 70% (95% CI, 57%-93%), respectively, whereas the cumulative incidences of relapse and transplant-related mortality were 33% (95% CI, 5%-58%) and 7% (95% CI, 0%-44%), respectively. The 3-year EFS rates did not differ between MSD and AD HSCT. Univariate analysis showed that age ≥ 10 years at diagnosis was the only factor associated with poorer EFS. Development of acute graft-versus-host disease predicted a significantly lower incidence of relapse. These findings may provide further evidence that allogeneic HSCT is a curative therapy for children with high-risk or advanced AML, and suggest the efficacy of AD transplantation.
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: Kyung Nam
authors.initials: KN
authors.lastname: Koh
authors.forename: Meerim
authors.initials: M
authors.lastname: Park
authors.forename: Bo Eun
authors.initials: BE
authors.lastname: Kim
authors.forename: Keun Wook
authors.initials: KW
authors.lastname: Bae
authors.forename: Ho Joon
authors.initials: HJ
authors.lastname: Im
authors.forename: Jong Jin
authors.initials: JJ
authors.lastname: Seo
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D020732:Cytogenetic Analysis; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality*; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality*; D006801:Humans; D015994:Incidence; D007223:Infant; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality* / Q000628:therapy*; D008297:Male; D011237:Predictive Value of Tests; D012008:Recurrence; D012189:Retrospective Studies; D012307:Risk Factors; D012720:Severity of Illness Index; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome