Molecular Approaches to Treating Pediatric Leukemias

Frontiers in Pediatrics

article

Michaela Kuhlen¹  Jan-Henning Klusmann²  Jessica I. Hoell² 

[1] Swabian Children's Cancer Center, University Children's Hospital Augsburg;Department of Pediatric Hematology and Oncology, Martin Luther University Halle-Wittenberg

关键词: leukemia;    children;    targeted therapy;    precision medicine;    molecular approaches;   

DOI  :  10.3389/fped.2019.00368

学科分类：社会科学、人文和艺术（综合）

来源: Frontiers

【 摘 要 】

Over the past decades, striking progress has been made in the treatment of pediatric leukemia, approaching 90% overall survival in children with acute lymphoblastic leukemia (ALL) and 75% in children with acute myeloid leukemia (AML). This has mainly been achieved through multiagent chemotherapy including CNS prophylaxis and risk-adapted therapy within collaborative clinical trials. However, prognosis in children with refractory or relapsed leukemia remains poor and has not significantly improved despite great efforts. Hence, more effective and less toxic therapies are urgently needed. Our understanding of disease biology, molecular drivers, drug resistance and, thus, the possibility to identify children at high-risk for treatment failure has significantly improved in recent years. Moreover, several new drugs targeting key molecular pathways involved in leukemia development, cell growth, and proliferation have been developed and approved. These striking achievements are linked to the great hope to further improve survival in children with refractory and relapsed leukemia. This review gives an overview on current molecularly targeted therapies in children with leukemia, including kinase, and proteasome inhibitors, epigenetic and enzyme targeting, as well as apoptosis regulators among others.

【 授权许可】

CC BY