Student Scientific Society, Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland.
Student Scientific Society, Laboratory of Genetic Diagnostics, Medical University of Lublin, Gębali 6, 20-093 Lublin, Poland.
Molecules. 2022 Jun 18;27(12):3911. doi: 10.3390/molecules27123911.
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.
急性髓细胞白血病（AML）约占所有儿童白血病病例的 15-20%。患有急性髓细胞白血病的儿童的总生存率不超过 82%，5 年无事件生存率为 46%-69%。如此不理想的结果是由于这种疾病中发生的许多突变和表观遗传变化，这些变化对治疗的敏感性和复发率产生了不利影响。我们描述了近年来为应对这些挑战而开发的各种分子靶向治疗方法，并正在临床试验中进行研究。这些新的治疗方法最初在成人 AML 中引入，现在正慢慢开始改变儿科 AML 的治疗方法。尽管针对新药的临床试验结果令人鼓舞，但仍需要进行更多涉及大量儿科患者的临床研究，以改善儿童 AML 的治疗效果。
