Borst Alexandra
Comprehensive Vascular Anomalies Program, The Children's Hospital of Philadelphia, Philadelphia, PA.
Hematology Am Soc Hematol Educ Program. 2024 Dec 6;2024(1):709-717. doi: 10.1182/hematology.2024000599.
The last 2 decades of genetic discovery in the field of vascular anomalies have brought targeted medical therapies to the forefront of care patients with vascular anomalies and have broadened the role of hematologists/oncologists in this field. Many vascular anomalies have now been identified to be driven by somatic gain-of-function variants in the PI3K/AKT/ mTOR and Ras/MAPK intracellular signaling pathways. This has led to the introduction of various antiangiogenic agents that inhibit these pathways. Knowledge of the indications for and the safe administration of these agents in patients with vascular anomalies is now a crucial part of training for hematologists/oncologists.
在血管异常领域过去20年的基因发现,已使靶向药物治疗成为血管异常患者治疗的前沿方法,并扩大了血液科医生/肿瘤内科医生在该领域的作用。现在已确定许多血管异常是由PI3K/AKT/mTOR和Ras/MAPK细胞内信号通路中的体细胞功能获得性变异驱动的。这导致了多种抑制这些通路的抗血管生成药物的引入。了解这些药物在血管异常患者中的适应证和安全用药方法,现在已成为血液科医生/肿瘤内科医生培训的关键部分。