Straight to the Point—The Novel Strategies to Cure Pediatric AML

International Journal of Molecular Sciences

Monika Lejman¹  Mateusz Jurek²  Izabela Dziatkiewicz² 

[1] Laboratory of Genetic Diagnostics, II Faculty of Pediatrics, Medical University of Lublin, A. Gębali 6, 20-093 Lublin, Poland;Student Scientific Society, Laboratory of Genetic Diagnostics, II Faculty of Pediatrics, Medical University of Lublin, A. Gębali 6, 20-093 Lublin, Poland;

关键词: AML;    pediatric;    targeted therapy;    CAR-T;   

DOI  :  10.3390/ijms23041968

来源: DOAJ

【 摘 要 】

Although the outcome has improved over the past decades, due to improved supportive care, a better understanding of risk factors, and intensified chemotherapy, pediatric acute myeloid leukemia remains a life-threatening disease, and overall survival (OS) remains near 70%. According to French-American-British (FAB) classification, AML is divided into eight subtypes (M0–M7), and each is characterized by a different pathogenesis and response to treatment. However, the curability of AML is due to the intensification of standard chemotherapy, more precise risk classification, improvements in supportive care, and the use of minimal residual disease to monitor response to therapy. The treatment of childhood AML continues to be based primarily on intensive, conventional chemotherapy. Therefore, it is essential to identify new, more precise molecules that are targeted to the specific abnormalities of each leukemia subtype. Here, we review abnormalities that are potential therapeutic targets for the treatment of AML in the pediatric population.

【 授权许可】

Unknown