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Acute Leukemia

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

Bone Marrow Transplantation volume 50, pages 181–188 (2015)Cite this article

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An Erratum to this article was published on 04 February 2015

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Abstract

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.

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  • 04 February 2015

    This article has been corrected since Advance Online Publication and an erratum is also printed in this issue.

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Acknowledgements

This study was supported by research grants awarded by Associazione Italiana per la Ricerca sul Cancro (5 x 1000 Special Grant #9962 to FL), by PRIN (Progetti di Rilevante Interesse Nazionale) 2010 to FL and 2012 to SR, by Ospedale Bambino Gesù, Roma, (Progetto di Ricerca Corrente 2012–2013) to AB and FL, and FILAS (Adult Stem Cells) to SR.

Author Contributions

FL designed the study, interpreted data, performed transplantation and wrote the article; RM designed the study, checked data and performed transplantation; RR analyzed data; MZ, FF, AR, CM and EL designed the study, performed transplantation and followed patients; AB, CF, GG, MR, GP, MP and OZ performed transplantation and followed patients; AP designed the study, performed transplantation and interpreted the data; SR interpreted the data and wrote the paper; ArPr contributed to study design, interpreted the data and performed transplantation.

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Authors and Affiliations

  1. Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy

    F Locatelli, A Bertaina, G Palumbo & S Rutella

  2. Department of Pediatric Science, University of Pavia, Pavia, Italy

    F Locatelli

  3. Department of Pediatrics, Pediatric Oncology and Hematology Unit ‘Lalla Seragnoli’, University of Bologna Sant’Orsola-Malpighi Hospital, Bologna, Italy

    R Masetti, R Rondelli, A Pession & A Prete

  4. Department of Pediatric Onco-Hematology, IRCCS, Policlinico San Matteo Foundation, Pavia, Italy

    M Zecca & G Giorgiani

  5. Stem Cell Transplantation and Cellular Therapy Division, Pediatric Onco-Hematology, Regina Margherita Children's Hospital, Torino, Italy

    F Fagioli

  6. Department of Pediatric Hematology, San Gerardo Hospital, Monza, Italy

    A Rovelli

  7. Department of Pediatric Hematology and Oncology, University of Padova, Padova, Italy

    C Messina & M Pillon

  8. Department of Pediatric Hematology Oncology, IRCCS G. Gaslini Institute, Genova, Italy

    E Lanino

  9. Department of Pediatrics, University of Pisa, Pisa, Italy

    C Favre

  10. Department of Pediatric Hemato-Oncology, BMT Unit, Santobono-Pausilipon Hospital, Napoli, Italy

    M Ripaldi

  11. Pediatric Hematology/Oncology, ARNAS Ospedale Civico di Palermo, Palermo, Italy,

    O Ziino

  12. Department of Medical Sciences, Catholic University Medical School, Rome, Italy

    S Rutella

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  1. F Locatelli
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Correspondence to F Locatelli.

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List of participating centers and investigators

Department of Pediatric Onco-Hematology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Policlinico San Matteo Foundation, Pavia. Franco Locatelli (till January 2010), Marco Zecca, Giovanna Giorgiani [38 HSCT].Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma. Franco Locatelli (since January 2010), Alice Bertaina, Maurizio Caniglia, Giuseppe Palumbo, Sergio Rutella [30 HSCT]. Department of Pediatric Hematology and Oncology, University of Padova, Padova. Chiara Messina, Marta Pillon [29 HSCT]. Department of Pediatric Hematology, San Gerardo Hospital, Monza. Adriana Balduzzi, Attilio Rovelli [27 HSCT]. BMT Unit, Department of Pediatric Hemato-Oncology, Santobono-Pausilipon Hospital, Napoli. Mimmo Ripaldi [24 HSCT]. Department of Pediatric Hematology Oncology, IRCCS G. Gaslini Institute, Genova. Edoardo Lanino, Giorgio Dini [23 HSCT]. Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino. Franca Fagioli [15 HSCT]. Pediatric Oncology and Hematology Unit ‘Lalla Seragnoli’, Department of Pediatrics, University of Bologna Sant’Orsola-Malpighi Hospital, Bologna. Riccardo Masetti, Arcangelo Prete, Andrea Pession [12 HSCT]. Department of Pediatrics, University of Pisa, Pisa. Claudio Favre [9 HSCT]. Pediatric Hematology/Oncology, ARNAS Ospedale Civico di Palermo. Ottavio Ziino, Paolo D’Angelo [8 HSCT]. Pediatric Hematology/Oncology, University Hospital, Catania. Luca Lo Nigro [8 HSCT]. BMT Unit, Ospedale di Pescara. Paolo Di Bartolomeo [6 HSCT]. BMT Unit, Ospedale Pediatrico Burlo Garofalo, Trieste. Marco Rabusin [6 HSCT]. BMT Unit, Department of Pediatric Hematology/Oncology, Ospedale Pediatrico Meier, Florence. Desiree Caselli [5 HSCT]. BMT Unit, Pediatric Hematology/Oncology, Ospedale Silvestrini, Perugia. Franco Aversa [3 HSCT].

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Locatelli, F., Masetti, R., Rondelli, R. et al. Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.. Bone Marrow Transplant 50, 181–188 (2015). https://doi.org/10.1038/bmt.2014.246

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