Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.
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 busulfan, cyclophosphamide and melphalan; [corrected] 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.
1] Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy [2] Department of Pediatric Science, University of Pavia, Pavia, Italy.
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F
Locatelli
Pediatric Oncology and Hematology Unit 'Lalla Seragnoli', Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
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R
Masetti
Pediatric Oncology and Hematology Unit 'Lalla Seragnoli', Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
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Rondelli
Department of Pediatric Onco-Hematology, IRCCS, Policlinico San Matteo Foundation, Pavia, Italy.
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M
Zecca
Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino, Italy.
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F
Fagioli
Department of Pediatric Hematology, San Gerardo Hospital, Monza, Italy.
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A
Rovelli
Department of Pediatric Hematology and Oncology, University of Padova, Padova, Italy.
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C
Messina
Department of Pediatric Hematology Oncology, IRCCS G. Gaslini Institute, Genova, Italy.
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E
Lanino
Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy.
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A
Bertaina
Department of Pediatrics, University of Pisa, Pisa, Italy.
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C
Favre
Department of Pediatric Onco-Hematology, IRCCS, Policlinico San Matteo Foundation, Pavia, Italy.
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Giorgiani
BMT Unit, Department of Pediatric Hemato-Oncology, Santobono-Pausilipon Hospital, Napoli, Italy.
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M
Ripaldi
Pediatric Hematology/Oncology, ARNAS Ospedale Civico di Palermo, Palermo, Italy.
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O
Ziino
Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy.
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G
Palumbo
Department of Pediatric Hematology and Oncology, University of Padova, Padova, Italy.
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M
Pillon
Pediatric Oncology and Hematology Unit 'Lalla Seragnoli', Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
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Pession
1] Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy [2] Department of Medical Sciences, Catholic University Medical School, Rome, Italy.
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Rutella
Pediatric Oncology and Hematology Unit 'Lalla Seragnoli', Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
A
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Prete
D059786:Abnormal Karyotype; D000293:Adolescent; D064591:Allografts; D064592:Autografts; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality* / Q000473:pathology / Q000628:therapy*; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage*; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods*; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics
D019653:Myeloablative Agonists; C497970:FLT3 protein, human; D051941:fms-Like Tyrosine Kinase 3