Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia
Piotr Obszański,
Anna Kozłowska,
Jakub Wańcowiat,
Julia Twardowska,
Monika Lejman,
Joanna Zawitkowska
Affiliations
Piotr Obszański
Student Scientific Society, Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
Anna Kozłowska
Student Scientific Society, Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
Jakub Wańcowiat
Student Scientific Society, Laboratory of Genetic Diagnostics, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
Julia Twardowska
Student Scientific Society, Laboratory of Genetic Diagnostics, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
Monika Lejman
Laboratory of Genetic Diagnostics, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
Joanna Zawitkowska
Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland
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.
