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Review
. 2023 Nov 15;16(11):1614.
doi: 10.3390/ph16111614.

A Review of Childhood Acute Myeloid Leukemia: Diagnosis and Novel Treatment

Serena Tseng  1 , Mu-En Lee  2 , Pei-Chin Lin  1   3
Affiliations
Review

A Review of Childhood Acute Myeloid Leukemia: Diagnosis and Novel Treatment

Serena Tseng et al. Pharmaceuticals (Basel). .

Abstract

Acute myeloid leukemia (AML) is the second most common hematologic malignancy in children. The incidence of childhood AML is much lower than acute lymphoblastic leukemia (ALL), which makes childhood AML a rare disease in children. The role of genetic abnormalities in AML classification, management, and prognosis prediction is much more important than before. Disease classifications and risk group classifications, such as the WHO classification, the international consensus classification (ICC), and the European LeukemiaNet (ELN) classification, were revised in 2022. The application of the new information in childhood AML will be upcoming in the next few years. The frequency of each genetic abnormality in adult and childhood AML is different; therefore, in this review, we emphasize well-known genetic subtypes in childhood AML, including core-binding factor AML (CBF AML), KMT2Ar (KMT2A/11q23 rearrangement) AML, normal karyotype AML with somatic mutations, unbalanced cytogenetic abnormalities AML, NUP98 11p15/NUP09 rearrangement AML, and acute promyelocytic leukemia (APL). Current risk group classification, the management algorithm in childhood AML, and novel treatment modalities such as targeted therapy, immune therapy, and chimeric antigen receptor (CAR) T-cell therapy are reviewed. Finally, the indications of hematopoietic stem cell transplantation (HSCT) in AML are discussed.

Keywords: KMT2A/11q23 rearrangement; acute myeloid leukemia (AML); acute promyelocytic leukemia (APL); childhood; core-binding factor (CBF); hematopoietic stem cell transplantation (HSCT).

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Percentage of different cytogenetic/molecular genetic features of childhood AML in Taiwan (1996~2019). The pie chart showed the percentages of different genetic subtypes in childhood AML diagnosed between 1996 and 2019. The total number of patients enrolled was 860. (Adapted from Table 2 in Yang et al., 2021 [9]).
Figure 2
Figure 2
Five-year (a) event-free survival, (b) relapse-free survival, and (c) overall survival rates according to major cytogenetic alterations. (From Figure 2 in Yang et al., 2021 with permission [9]).
Figure 3
Figure 3
Recommended risk classification based on genetic alterations using prognostically important genetic abnormalities in childhood AML and MRD (minimal residual disease), adapted from [8].
Figure 4
Figure 4
Recommended treatment of childhood AML [26]. Reprinted from Blood, Vol 138 Issue 12 Pages 1009–1018, Jeffrey E. Rubnitz, Gertjan J.L. Kaspers, How I treat pediatric acute myeloid leukemia, Visual abstract, 2021, with permission.
See this image and copyright information in PMC

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