Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study

• Published in: Bone marrow transplantation (Basingstoke) Vol. 50; no. 2; pp. 181 - 188
• Main Authors: Locatelli, F, Masetti, R, Rondelli, R, Zecca, M, Fagioli, F, Rovelli, A, Messina, C, Lanino, E, Bertaina, A, Favre, C, Giorgiani, G, Ripaldi, M, Ziino, O, Palumbo, G, Pillon, M, Pession, A, Rutella, S, Prete, A
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.02.2015; Nature Publishing Group
• Subjects: 13; 692/308/2171; Abnormal Karyotype; Acute myelocytic leukemia; Acute myeloid leukemia; Adolescent; Allografts; Autografts; Blood; Bone marrow; Care and treatment; Cell Biology; Child; Child, Preschool; Children; Cord blood; Cord Blood Stem Cell Transplantation; Cyclophosphamide; Disease-Free Survival; Female; fms-Like Tyrosine Kinase 3 - genetics; Follow-Up Studies; Genetic aspects; Health aspects; Hematology; Hematopoietic Stem Cell Transplantation; Histocompatibility antigen HLA; Humans; Infant; Internal Medicine; Karyotypes; Leukemia; Leukemia, Myeloid, Acute - genetics; Leukemia, Myeloid, Acute - mortality; Leukemia, Myeloid, Acute - pathology; Leukemia, Myeloid, Acute - therapy; Male; Medicine; Medicine & Public Health; Melphalan; Myeloablative Agonists - administration & dosage; original-article; Patient outcomes; Peripheral blood; Public Health; Stem cell transplantation; Stem Cells; Survival Rate; Transplantation; Transplantation Conditioning - methods; Italy
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/bmt.2014.246

We analyzed the outcome of 243 children with high-risk (HR) AML in first CR1 enrolled in the AIEOP-2002/01 protocol, who were given either allogeneic (ALLO; n =141) or autologous (AUTO; n =102) hematopoietic SCT (HSCT), depending on the availability of a HLA-compatible sibling. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were eligible to be transplanted also from alternative donors. All patients received a myeloablative regimen combining BU, Cyclophosphamide and Melphalan; AUTO-HSCT patients received BM cells in most cases, while in children given ALLO-HSCT stem cell source was BM in 96, peripheral blood in 19 and cord blood in 26. With a median follow-up of 57 months (range 12–130), the probability of disease-free survival (DFS) was 73% and 63% in patients given either ALLO- or AUTO-HSCT, respectively ( P =NS). Although the cumulative incidence (CI) of relapse was lower in ALLO- than in AUTO-HSCT recipients (17% vs 28%, respectively; P =0.043), the CI of TRM was 7% in both groups. Patients transplanted with unrelated donor cord blood had a remarkable 92.3% 8-year DFS probability. Altogether, these data confirm that HSCT is a suitable option for preventing leukemia recurrence in HR children with CR1 AML.