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Review ARTICLE Provisionally accepted The full-text will be published soon. Notify me

Front. Pediatr. | doi: 10.3389/fped.2019.00463

Targeted therapies for pediatric AML: gaps and perspective

  • 1Giorgio Prodi Interdepartmental Cancer Research Centre, University of Bologna, Italy
  • 2Department of Medical and Surgical Sciences, University of Bologna, Italy

Acute Myeloid Leukemia (AML) is a hematopoietic disorder characterized by numerous cytogenetic and molecular aberrations that accounts for approximately 25% of childhood leukemia diagnoses. The outcome of children with AML has increased remarkably over the past 30 years, with current survival rates up to 70%, mainly due to intensification of standard chemotherapy and improvements in risk classification, supportive care, and minimal residual disease monitoring. However, childhood AML prognosis remains unfavorable and relapse rates are still around 30%. Therefore, novel therapeutic approaches are needed to increase the cure rate. In AML, the presence of gene mutations and rearrangements prompted the identification of effective targeted molecular strategies, including kinase inhibitors, cell pathway inhibitors and epigenetic modulators. This review will discuss several new drugs that recently received US Food and Drug Administration approval for AML treatment and promising strategies to treat childhood AML, including FLT3 inhibitors, epigenetic modulators and Hedgehog pathway inhibitors.

Keywords: Pediatric AML, targeted therapy, FLT-3 inhibitors, Hedgehog pathway inhibitors, DOT1L inhibitors

Received: 18 Jul 2019; Accepted: 24 Oct 2019.

Copyright: © 2019 Lonetti, Andrea and Masetti. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: PhD. Annalisa Lonetti, Giorgio Prodi Interdepartmental Cancer Research Centre, University of Bologna, Bologna, 40138, Emilia-Romagna, Italy,