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Review
. 2021 Jun;16(3):235-246.
doi: 10.1007/s11899-021-00611-x. Epub 2021 Feb 25.

Pediatric Neoplasms Presenting with Monocytosis

Jacob R Greenmyer  1 , Mira Kohorst  2
Affiliations
Review

Pediatric Neoplasms Presenting with Monocytosis

Jacob R Greenmyer et al. Curr Hematol Malig Rep. 2021 Jun.

Abstract

Purpose of review: Juvenile myelomonocytic leukemia (JMML) is a rare but severe pediatric neoplasm with hematopoietic stem cell transplant as its only established curative option. The development of targeted therapeutics for JMML is being guided by an understanding of the pathobiology of this condition. Here, we review JMML with an emphasis on genetics in order to (i) demonstrate the relationship between JMML genotype and clinical phenotype and (ii) explore potential genetic targets of novel JMML therapies.

Recent findings: DNA hypermethylation studies have demonstrated consistently that methylation is related to disease severity. Increasing understanding of methylation in JMML may open the door to novel therapies, such as DNA methyltransferase inhibitors. The PI3K/AKT/MTOR, JAK/STAT, and RAF/MEK/ERK pathways are being investigated as therapeutic targets for JMML. Future therapy for JMML will be driven by an increased understanding of pathobiology. Targeted therapeutic approaches hold potential for improving outcomes in patients with JMML.

Keywords: JMML; Monocytosis; Pediatric.

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