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Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia

Acute myeloid leukemia (AML) accounts for approximately 15–20% of all childhood leukemia cases. The overall survival of children with acute myeloid leukemia does not exceed 82%, and the 5-year event-free survival rates range from 46% to 69%. Such suboptimal outcomes are the result of numerous mutati...

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Autores principales: Obszański, Piotr, Kozłowska, Anna, Wańcowiat, Jakub, Twardowska, Julia, Lejman, Monika, Zawitkowska, Joanna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9230975/
https://www.ncbi.nlm.nih.gov/pubmed/35745032
http://dx.doi.org/10.3390/molecules27123911
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author Obszański, Piotr
Kozłowska, Anna
Wańcowiat, Jakub
Twardowska, Julia
Lejman, Monika
Zawitkowska, Joanna
author_facet Obszański, Piotr
Kozłowska, Anna
Wańcowiat, Jakub
Twardowska, Julia
Lejman, Monika
Zawitkowska, Joanna
author_sort Obszański, Piotr
collection PubMed
description Acute myeloid leukemia (AML) accounts for approximately 15–20% of all childhood leukemia cases. The overall survival of children with acute myeloid leukemia does not exceed 82%, and the 5-year event-free survival rates range from 46% to 69%. Such suboptimal outcomes are the result of numerous mutations and epigenetic changes occurring in this disease that adversely affect the susceptibility to treatment and relapse rate. We describe various molecular-targeted therapies that have been developed in recent years to meet these challenges and were or are currently being studied in clinical trials. First introduced in adult AML, novel forms of treatment are slowly beginning to change the therapeutic approach to pediatric AML. Despite promising results of clinical trials investigating new drugs, further clinical studies involving greater numbers of pediatric patients are still needed to improve the outcomes in childhood AML.
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spelling pubmed-92309752022-06-25 Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia Obszański, Piotr Kozłowska, Anna Wańcowiat, Jakub Twardowska, Julia Lejman, Monika Zawitkowska, Joanna Molecules Review Acute myeloid leukemia (AML) accounts for approximately 15–20% of all childhood leukemia cases. The overall survival of children with acute myeloid leukemia does not exceed 82%, and the 5-year event-free survival rates range from 46% to 69%. Such suboptimal outcomes are the result of numerous mutations and epigenetic changes occurring in this disease that adversely affect the susceptibility to treatment and relapse rate. We describe various molecular-targeted therapies that have been developed in recent years to meet these challenges and were or are currently being studied in clinical trials. First introduced in adult AML, novel forms of treatment are slowly beginning to change the therapeutic approach to pediatric AML. Despite promising results of clinical trials investigating new drugs, further clinical studies involving greater numbers of pediatric patients are still needed to improve the outcomes in childhood AML. MDPI 2022-06-18 /pmc/articles/PMC9230975/ /pubmed/35745032 http://dx.doi.org/10.3390/molecules27123911 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Obszański, Piotr
Kozłowska, Anna
Wańcowiat, Jakub
Twardowska, Julia
Lejman, Monika
Zawitkowska, Joanna
Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title_full Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title_fullStr Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title_full_unstemmed Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title_short Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
title_sort molecular-targeted therapy of pediatric acute myeloid leukemia
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9230975/
https://www.ncbi.nlm.nih.gov/pubmed/35745032
http://dx.doi.org/10.3390/molecules27123911
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