Division of Hematology/Oncology, University of North Carolina, Chapel Hill, NC 27599-7305, USA.
Hematology Am Soc Hematol Educ Program. 2009:54-61. doi: 10.1182/asheducation-2009.1.54.
Despite an increased understanding of the pathophysiology of sickle cell disease (SCD), there remains a paucity of available agents for the prevention and treatment of specific SCD-related complications. Recently, there has been significant progress in the development of novel drugs for this disease. These agents, which increase the production of fetal hemoglobin, improve red blood cell hydration, increase the availability of nitric oxide and possess anti-inflammatory effects, are in varying stages of clinical development. With the complex pathophysiology of SCD, it is unlikely that a single agent will prevent or treat all the sequelae of this disease. As a result, patients may benefit from treatment with a combination of agents that possess different mechanisms of action. This overview discusses selected novel agents that appear promising in SCD.
尽管对镰状细胞病（SCD）的病理生理学有了更多的了解，但仍缺乏可用于预防和治疗特定 SCD 相关并发症的药物。最近，新型药物的开发取得了重大进展。这些药物可增加胎儿血红蛋白的产生、改善红细胞的水合作用、增加一氧化氮的可用性并具有抗炎作用，处于不同的临床开发阶段。由于 SCD 的病理生理学复杂，单一药物不太可能预防或治疗这种疾病的所有后遗症。因此，患者可能受益于具有不同作用机制的药物联合治疗。本文综述了在 SCD 中表现出前景的一些新型药物。
