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.
Int J Mol Sci. 2022 Feb 10;23(4):1968. doi: 10.3390/ijms23041968.
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.
尽管过去几十年来，由于支持性治疗的改善、对风险因素的更好理解以及强化化疗，儿科急性髓系白血病的预后有所改善，但它仍是一种危及生命的疾病，总体生存率（OS）仍接近 70%。根据法美英（FAB）分类，AML 分为八个亚型（M0-M7），每个亚型的发病机制和对治疗的反应都不同。然而，AML 的可治愈性归因于标准化疗的强化、更精确的风险分类、支持性治疗的改进以及使用微小残留病来监测治疗反应。儿童 AML 的治疗仍主要基于强化、常规化疗。因此，识别新的、更精确的针对每个白血病亚型特定异常的靶向分子至关重要。在这里，我们综述了儿科 AML 治疗的潜在治疗靶点的异常。
