Paving the way for immunotherapy in pediatric acute myeloid leukemia: Current knowledge and the way forward

J.B. Koedijk*, Inge van der Werf, Friso G. Calkoen, Stefan Nierkens, Gertjan J.L. Kaspers, Christian Michel Zwaan, Olaf Heidenreich

*Corresponding author for this work

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Immunotherapeutic agents may be an attractive option to further improve outcomes and to reduce treatment-related toxicity for pediatric AML. While improvements in outcome have been observed with immunotherapy in many cancer types, immunotherapy development and implementation into patient care for both adult and pediatric AML has been hampered by an incomplete understanding of the bone marrow environment and a paucity of tumor-specific antigens. Since only a minority of patients respond in most immunotherapy trials across different cancer types, it will be crucial to understand which children with AML are likely to respond to or may benefit from immunotherapies. Immune cell profiling efforts hold promise to answer this question, as illustrated by the development of predictive scores in solid cancers. Such information on the number and phenotype of immune cells during current treatment regimens will be pivotal to generate hypotheses on how and when to intervene with immunotherapy in pediatric AML. In this review, we discuss the current understanding of the number and phenotype of immune cells in the bone marrow in pediatric AML, ongoing immunotherapy trials and how comprehensive immune profiling efforts may pave the way for successful clinical trials (and, ultimately, implementation into patient care).

Original languageEnglish
Article number4364
Issue number17
Publication statusPublished - 28 Aug 2021

Bibliographical note

Funding Information:
Funding: This work was in part funded by a KiKa programme grant (329) to O.H.

Publisher Copyright:
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.

Research programs

  • EMC MM-02-54-03


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