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Review
. 2021 Aug 25;10(17):3790.
doi: 10.3390/jcm10173790.

The Role of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Leukemia

Mattia Algeri  1 , Pietro Merli  1 , Franco Locatelli  1   2 , Daria Pagliara  1
Affiliations
Review

The Role of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Leukemia

Mattia Algeri et al. J Clin Med. .

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) offers potentially curative treatment for many children with high-risk or relapsed acute leukemia (AL), thanks to the combination of intense preparative radio/chemotherapy and the graft-versus-leukemia (GvL) effect. Over the years, progress in high-resolution donor typing, choice of conditioning regimen, graft-versus-host disease (GvHD) prophylaxis and supportive care measures have continuously improved overall transplant outcome, and recent successes using alternative donors have extended the potential application of allotransplantation to most patients. In addition, the importance of minimal residual disease (MRD) before and after transplantation is being increasingly clarified and MRD-directed interventions may be employed to further ameliorate leukemia-free survival after allogeneic HSCT. These advances have occurred in parallel with continuous refinements in chemotherapy protocols and the development of targeted therapies, which may redefine the indications for HSCT in the coming years. This review discusses the role of HSCT in childhood AL by analysing transplant indications in both acute lymphoblastic and acute myeloid leukemia, together with current and most promising strategies to further improve transplant outcome, including optimization of conditioning regimen and MRD-directed interventions.

Keywords: acute lymphoblastic leukemia; acute myeloid leukemia; allogeneic stem cell transplantation; alternative donors; conditioning regimen; minimal residual disease.

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Conflict of interest statement

F.L. reports advisory board membership for Amgen, Novartis, Bellicum Pharmaceutical, Neovii, and is on the Speakers’ Bureau for Amgen, Novartis, Miltenyi, Medac, Jazz Pharmaceutical, Gilead, Sanofi and Takeda, outside the submitted work.

References

    1. Hunger S.P., Lu X., Devidas M., Camitta B.M., Gaynon P.S., Winick N.J., Reaman G.H., Carroll W.L. Improved survival for children and adolescents with acute lymphoblastic leukemia between 1990 and 2005: A report from the children’s oncology group. J. Clin. Oncol. 2012;30:1663–1669. doi: 10.1200/JCO.2011.37.8018. - DOI - PMC - PubMed
    1. Bunin N.J., Davies S.M., Aplenc R., Camitta B.M., DeSantes K.B., Goyal R.K., Kapoor N., Kernan N.A., Rosenthal J., Smith F.O., et al. Unrelated donor bone marrow transplantation for children with acute myeloid leukemia beyond first remission or refractory to chemotherapy. J. Clin. Oncol. 2008;26:4326–4332. doi: 10.1200/JCO.2008.16.4442. - DOI - PMC - PubMed
    1. Pession A., Masetti R., Rizzari C., Putti M.C., Casale F., Fagioli F., Luciani M., Lo Nigro L., Menna G., Micalizzi C., et al. Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia. Blood. 2013;122:170–178. doi: 10.1182/blood-2013-03-491621. - DOI - PubMed
    1. Rasche M., Zimmermann M., Borschel L., Bourquin J.P., Dworzak M., Klingebiel T., Lehrnbecher T., Creutzig U., Klusmann J.H., Reinhardt D. Successes and challenges in the treatment of pediatric acute myeloid leukemia: A retrospective analysis of the AML-BFM trials from 1987 to 2012. Leukemia. 2018;32:2167–2177. doi: 10.1038/s41375-018-0071-7. - DOI - PMC - PubMed
    1. Rubnitz J.E., Inaba H., Dahl G., Ribeiro R.C., Bowman W.P., Taub J., Pounds S., Razzouk B.I., Lacayo N.J., Cao X., et al. Minimal residual disease-directed therapy for childhood acute myeloid leukaemia: Results of the AML02 multicentre trial. Lancet Oncol. 2010;11:543–552. doi: 10.1016/S1470-2045(10)70090-5. - DOI - PMC - PubMed

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