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Prepublished online as a Blood First Edition Paper on October 10, 2002; DOI 10.1182/blood-2002-03-0764.

Submitted March 18, 2002
Accepted August 7, 2002
Factors influencing outcome and incidence of long-term complications in children given autologous stem cell transplantation for acute myeloid leukemia in 1st complete remission
Franco W Locatelli*, Myriam Labopin, Juan Ortega, Giovanna Meloni, Giorgio Dini, Chiara Messina, Isaac Yaniv, Franca Fagioli, Victoria Castel, Peter J Shaw, Augustin Ferrant, Andrea Pession, Gerard Socie, and Francesco Frassoni
Universita' di Pavia, Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy
EBMT, European Data Management Office, Paris, France
Vall d'Hebron, Hospital Materno Infantil, Barcelona, Spain
Universita' La Sapienza, Dipartimento di Biotecnologie Cellulari ed Ematologia, Rome, Italy
Istituto G. Gaslini, Dipartimento di Ematologia ed Oncologia, Genova, Italy
Universita' di Padova, Clinica Pediatrica, Padova, Italy
Schneider Children's Medical Center of Israel, BMT Unit, Petach-Tikva, Israel
Universita' di Torino, Clinica Pediatrica, Turin, Italy
Hospital Infantil La Fe, Valencia, Spain
Children Hospital at Westmead, Oncology Unit, Sydney, Australia
Cliniques Universitaires St Luc, Brussels, Belgium
Universita' di Bologna, Clinica Pediatrica, Bologna, Italy
Hopital Saint-Louis, Department of Hematology, BMT Unit, Paris, France
Ospedale San Martino, Divisione di Ematologia II, Genova, Italy
* Corresponding author; email: f.locatelli{at}smatteo.pv.it.
In order to evaluate factors influencing outcome and incidence of long-term complications, we analyzed, in a retrospective, multi-center study, 387 children given an autologous hematopoietic stem cell transplantation (HSCT) for acute myeloid leukemia (AML) in 1st complete remission (CR). Median follow-up time from transplantation was 60 months. Transplant of bone marrow cells was performed in 318 children, whereas in 60 patients peripheral blood progenitor cells (PBPC) were employed. In multivariate analysis, we investigated the variables influencing probability of hematopoietic recovery, transplant-related mortality (TRM), relapse, and leukemia-free survival (LFS). We found that use of PBPC as stem-cell source and use of BAVC as preparative regimen were associated with faster neutrophil recovery. Infusion of PBPC, young age, use of BAVC and no marrow purging predicted an accelerated platelet reconstitution. The 5-year Kaplan-Meier estimates of TRM, relapse and LFS were 3±1%, 39±3% and 60±3%, respectively. Relapse probability was increased in children given BAVC regimen and decreased after in vitro purging of hematopoietic progenitors, as well as in children with FAB M3 and a time interval between CR and HSCT 170 days. These 2 latter variables favourably influenced the probability of LFS, which was, by contrast, reduced with BAVC regimen. Thirty-three percent of patients surviving more than 18 months developed at least one late sequel, use of total body irradiation being the only predictive factor. The results obtained in this analysis can be of help in designing prospective studies autologous HSCT in children with AML in 1st CR.

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