Allogeneic stem cell transplantation for children with acute myeloid leukemia in second complete remission

Franca Fagioli, Marco Zecca, Franco Locatelli, Edoardo Lanino, Cornelio Uderzo, Paolo Di Bartolomeo, J. Massimo Berger, Claudio Favre, Roberto Rondelli, Andrea Pession, Chiara Messina

Research output: Contribution to journalArticlepeer-review


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 transplantrelated 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-versushost 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.

Original languageEnglish
Pages (from-to)575-583
Number of pages9
JournalJournal of Pediatric Hematology/Oncology
Issue number8
Publication statusPublished - Aug 2008


  • Allogeneic hematopoietic stem cell transplantation
  • AML in second CR
  • Leukemia relapse
  • Pediatric patients

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Oncology
  • Hematology
  • Medicine(all)


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