Department of Pediatrics, Division of Pediatric Hematology/Oncology, Albert Einstein College of Medicine, Children's Hospital at Montefiore, Bronx, NY.
Department of Pediatrics, Division of Pediatric Hematology/Oncology, Icahn School of Medicine at Mount Sinai, Kravis Children's Hospital, New York, NY.
Semin Hematol. 2018 Apr;55(2):68-75. doi: 10.1053/j.seminhematol.2018.04.007. Epub 2018 Apr 20.
Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These downstream targets are the focus of emerging treatments with considerable potential to ameliorate disease manifestations. This review summarizes the progress on development of these agents.
镰状细胞病是一种遗传性血红蛋白病，其特征为溶血性贫血、频繁的疼痛发作、生活质量差、终末器官损伤和寿命缩短。尽管主要事件是异常血红蛋白的聚合，但血管阻塞的下游病理生理学是由改变的、黏附性镰状红细胞、中性粒细胞、内皮细胞和血小板之间的异型相互作用引起的。缺血再灌注损伤、溶血和氧化损伤都导致炎症加剧和止血系统激活。这些下游靶点是新兴治疗方法的重点，这些方法具有改善疾病表现的巨大潜力。这篇综述总结了这些药物的开发进展。
