title: Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia.
abstract: We evaluated the outcome of 482 children with acute myeloid leukemia (AML) enrolled in the Associazione Italiana di Ematologia e Oncologia Pediatrica AML 2002/01 trial. Treatment was stratified according to risk group; hematopoietic stem cell transplantation (HSCT) was used in high-risk (HR) children. Patients with core binding factor leukemia achieving complete remission (CR) after the first induction course were considered standard risk (SR; 99 patients), whereas the others (n = 383) were assigned to the HR group. Allogeneic (ALLO) or autologous (AUTO) HSCT was employed, respectively, in 141 and 102 HR patients after consolidation therapy. CR, early death, and induction failure rates were 87%, 3%, and 10%, respectively. Relapse occurred in 24% of patients achieving CR. The 8-year overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 68%, 55%, and 63%, respectively. OS, EFS, and DFS for SR and HR patients were 83%, 63%, and 66% and 64%, 53%, and 62%. DFS was 63% and 73% for HR patients given AUTO-HSCT and ALLO-HSCT, respectively. In multivariate analysis, risk group, white blood cell >100 × 10(9)/L at diagnosis, and monosomal karyotype predicted poorer EFS. Risk-oriented treatment and broad use of HSCT result in a long-term EFS comparing favorably with previously published studies on childhood AML.
authors.affiliation: Clinica Pediatrica, Università di Bologna, Ospedale S. Orsola, Bologna, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Riccardo
authors.initials: R
authors.lastname: Masetti
authors.forename: Carmelo
authors.initials: C
authors.lastname: Rizzari
authors.forename: Maria Caterina
authors.initials: MC
authors.lastname: Putti
authors.forename: Fiorina
authors.initials: F
authors.lastname: Casale
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Matteo
authors.initials: M
authors.lastname: Luciani
authors.forename: Luca
authors.initials: L
authors.lastname: Lo Nigro
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Menna
authors.forename: Concetta
authors.initials: C
authors.lastname: Micalizzi
authors.forename: Nicola
authors.initials: N
authors.lastname: Santoro
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Testi
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Andrea
authors.initials: A
authors.lastname: Biondi
authors.forename: Martina
authors.initials: M
authors.lastname: Pigazzi
authors.forename: Sergio
authors.initials: S
authors.lastname: Rutella
authors.forename: Roberto
authors.initials: R
authors.lastname: Rondelli
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Basso
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D060830:Consolidation Chemotherapy; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D060828:Induction Chemotherapy; D007231:Infant, Newborn; D015470:Leukemia, Myeloid, Acute / Q000401:mortality / Q000628:therapy*; D008297:Male; D012008:Recurrence; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome