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Prepublished online as a Blood First Edition Paper on October 10, 2002; DOI 10.1182/blood-2002-03-0764.
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Blood, 15 February 2003, Vol. 101, No. 4, pp. 1611-1619
TRANSPLANTATION
Factors influencing outcome and incidence of long-term
complications in children who underwent autologous stem cell
transplantation for acute myeloid leukemia in first complete
remission
Franco 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 Sociè, and
Francesco Frassoni for the European Blood and Marrow
Transplantation Acute Leukemia Working Party
From Oncoematologia Pediatrica, Istituto di Ricovero e
Cura a Carattere Scientifico Policlinico San Matteo, Università
di Pavia, Italy; European Data Management Office, Paris,
France; Hospital Materno Infantil, Vall d'Hebron,
Barcelona, Spain; Dipartimento di Biotecnologie Cellulari
ed Ematologia, Università La Sapienza, Roma, Italy;
Dipartimento di Ematologia ed Oncologia, Istituto G. Gaslini, Genova,
Italy; Clinica Pediatrica, Università di Padova,
Italy; Bone Marrow Transplantation Unit, Schneider
Children's Medical Center of Israel, Petach-Tikva,
Israel; Clinica Pediatrica, Università di Torino,
Italy; Hospital Infantil La Fe, Valencia,
Spain; Oncology Unit, Children Hospital at Westmead,
Sydney, Australia; Cliniques Universitaires St Luc,
Brussels, Belgium; Clinica Pediatrica, Università di Bologna,
Italy; Department of Hematology, Bone Marrow
Transplantation Unit, Hôpital Saint-Louis, Paris,
France; Divisione di Ematologia II, Ospedale San Martino,
Genova, Italy.
To evaluate factors influencing outcome and incidence of long-term
complications, we analyzed, in a retrospective, multicenter study, 387 children who underwent autologous hematopoietic stem cell
transplantation (HSCT) for acute myeloid leukemia (AML) in first
complete remission (CR). Median follow-up time from transplantation was
60 months. Transplantation of bone marrow cells was performed in 318 children, whereas in 60 patients peripheral blood progenitor cells
(PBPCs) were used. In multivariate analysis, we investigated the
variables influencing probability of hematopoietic recovery, transplantation-related mortality (TRM), relapse, and leukemia-free survival (LFS). We found that use of PBPCs as stem cell sources and use
of BCNU
(N,N-bis[2-chloroethyl]-N-nitrosourea),
amsacrine, VP-16, and cytosine arabinoside (BAVC) as a preparative
regimen were associated with faster neutrophil recovery. Infusion of
PBPCs, young age of patients, use of BAVCs, and absence of 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 the BAVC regimen, and it was decreased
after in vitro purging of hematopoietic progenitors and in children
with a French-American-British classification of M3 and a time interval
of 170 days or more between CR and HSCT. These 2 latter variables
favorably influenced the probability of LFS, which was, by contrast,
reduced with the BAVC regimen. Thirty-three percent of patients
surviving more than 18 months experienced at least one late sequela;
use of total body irradiation was the only predictive factor. The
results obtained in this analysis can be of help in designing
prospective studies of autologous HSCT in children with AML in first CR.
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