title: Allogeneic stem cell transplantation for children with acute myeloid leukemia in second complete remission.
abstract: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective therapy for patients with relapsed acute myeloid leukemia. In this retrospective, multicenter study, we analyzed the outcome of 63 children (median age, 7 y; range, 0.2 to 17) who received unmanipulated allo-HSCT in second complete remission. Either a matched family donor or an unrelated donor was used in 29 (46%) and 34 (54%) patients, respectively. The stem cell source was bone marrow in 53 children (84%), peripheral blood in 7 (11%), and cord blood in 3 patients (5%). Preparative regimen included total body irradiation in 25 patients (40%). The 5-year estimates of overall survival and leukemia-free survival were 53% [95% confidence interval (CI) 39-66] and 49% (95% CI 35-63), respectively, whereas the cumulative incidence of relapse and transplant-related mortality (TRM) were 26% (95% CI 16-41) and 25% (95% CI 15-40), respectively. In multivariate analysis, the use of a matched family donor predicted a better probability of LFS [relative risk (RR) 2.29, P=0.05]. Both chronic graft-versus-host disease occurrence and age at diagnosis greater than 11 years were associated with an increased TRM (RR 8.08, P=0.04 and RR 4.38, P=0.05, respectively). These results indicate that allo-HSCT is a procedure able to rescue a significant proportion of children with acute myeloid leukemia in second complete remission, especially if an human leukocyte antigen-compatible relative is employed as donor. Both leukemia recurrence and TRM contributed to treatment failure. Optimization of donor selection and of strategies for both prophylaxis and treatment of graft-versus-host disease may improve the results of unrelated donor allo-HSCT.
authors.affiliation: Oncoematologia Pediatrica, Ospedale Infantile Regina Margherita, Torino, Italy. franca.fagioli@unito.it
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Edoardo
authors.initials: E
authors.lastname: Lanino
authors.forename: Cornelio
authors.initials: C
authors.lastname: Uderzo
authors.forename: Paolo
authors.initials: P
authors.lastname: Di Bartolomeo
authors.forename: Massimo
authors.initials: M
authors.lastname: Berger
authors.forename: Claudio
authors.initials: C
authors.lastname: Favre
authors.forename: Roberto
authors.initials: R
authors.lastname: Rondelli
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Chiara
authors.initials: C
authors.lastname: Messina
mesh_terms: D000293:Adolescent; D017668:Age of Onset; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000209:etiology / Q000628:therapy; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000401:mortality / Q000628:therapy*; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000401:mortality / Q000628:therapy*; D012074:Remission Induction; D012189:Retrospective Studies; D033581:Stem Cell Transplantation / Q000009:adverse effects; D014184:Transplantation, Homologous