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American Society of Hematology, Blood, 12(138), p. 1009-1018, 2021

DOI: 10.1182/blood.2021011694

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How I treat pediatric acute myeloid leukemia

Journal article published in 2021 by Jeffrey E. Rubnitz ORCID, Gertjan J. L. Kaspers
This paper was not found in any repository; the policy of its publisher is unknown or unclear.
This paper was not found in any repository; the policy of its publisher is unknown or unclear.

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Abstract

Abstract Treatment outcomes for pediatric patients with acute myeloid leukemia (AML) have continued to lag behind outcomes reported for children with acute lymphoblastic leukemia (ALL), in part because of the heterogeneity of the disease, a paucity of targeted therapies, and the relatively slow development of immunotherapy compared with ALL. In addition, we have reached the limits of treatment intensity, and, even with outstanding supportive care, it is highly unlikely that further intensification of conventional chemotherapy alone will impact relapse rates. However, comprehensive genomic analyses and a more thorough characterization of the leukemic stem cell have provided insights that should lead to tailored and more effective therapies in the near future. In addition, new therapies are finally emerging, including the BCL-2 inhibitor venetoclax, CD33- and CD123-directed chimeric antigen receptor T-cell therapy, CD123-directed antibody therapy, and menin inhibitors. Here, we present 4 cases to illustrate some of the controversies regarding the optimal treatment of children with newly diagnosed or relapsed AML.