title: 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.
abstract: 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.
authors.affiliation: Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Università di Pavia, Italy. f.locatelli@smatteo.pv.it
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Myriam
authors.initials: M
authors.lastname: Labopin
authors.forename: Juan
authors.initials: J
authors.lastname: Ortega
authors.forename: Giovanna
authors.initials: G
authors.lastname: Meloni
authors.forename: Giorgio
authors.initials: G
authors.lastname: Dini
authors.forename: Chiara
authors.initials: C
authors.lastname: Messina
authors.forename: Isaac
authors.initials: I
authors.lastname: Yaniv
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Victoria
authors.initials: V
authors.lastname: Castel
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Shaw
authors.forename: Augustin
authors.initials: A
authors.lastname: Ferrant
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Gerard
authors.initials: G
authors.lastname: Sociè
authors.forename: Francesco
authors.initials: F
authors.lastname: Frassoni
mesh_terms: D000293:Adolescent; D000677:Amsacrine / Q000008:administration & dosage; D000704:Analysis of Variance; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage; D001854:Bone Marrow Cells; D002330:Carmustine / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage; D018572:Disease-Free Survival; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D006410:Hematopoiesis; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality; D006801:Humans; D007223:Infant; D015470:Leukemia, Myeloid, Acute / Q000628:therapy*; D008297:Male; D009504:Neutrophils; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality; D012008:Recurrence; D012074:Remission Induction; D012189:Retrospective Studies; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D000677:Amsacrine; D003561:Cytarabine; D005047:Etoposide; D002330:Carmustine