Drug Therapies for the Management of Sickle Cell Disease.
Drug Therapies for the Management of Sickle Cell Disease.
复制标题
DOI：
10.12688/f1000research.22433.1
复制
发表时间：
2020-01-01
期刊：
影响因子：
--
通讯作者：
Ataga, Kenneth I
中科院分区：
文献类型：
--
作者：
Rai, Parul;Ataga, Kenneth I
镰状细胞病 (SCD) 困扰着全世界数百万人，但在美国被称为孤儿病。在过去的几十年里，人们对 SCD 的病理生理学及其并发症的了解不断加深。虽然资源丰富国家的大多数 SCD 患者都能存活到成年，但 SCD 患者的预期寿命仍然比一般非裔美国人要短得多。 SCD 可以通过造血干细胞移植和可能的基因治疗来治愈，但大多数患者无法获得这些治疗方法，其中大多数患者居住在低收入和中等收入国家。直到最近，只有一种药物羟基脲被美国食品和药物管理局批准用于减轻疾病的严重程度。多种其他药物（L-谷氨酰胺、crizanlizumab 和 voxelotor）最近已被批准用于治疗 SCD，还有其他几种药物处于不同的临床测试阶段。治疗 SCD 的多种药物的可用性引发了与适当药物治疗的选择、多种药物的组合以及最近批准的产品的可负担性相关的问题。对新药开发的热情为低收入和中等收入国家的患者参与测试潜在的疾病缓解疗法提供了机会，并有可能为这些环境中的能力建设做出贡献。证明这些药物单独或联合使用可以预防或减少终末器官损伤，将为药物治疗在改善 SCD 结局中的作用提供额外的证据。
Sickle cell disease (SCD) afflicts millions of people worldwide but is referred to as an orphan disease in the United States. Over the past several decades, there has been an increasing understanding of the pathophysiology of SCD and its complications. While most individuals with SCD in resource-rich countries survive into adulthood, the life expectancy of patients with SCD remains substantially shorter than for the general African-American population. SCD can be cured using hematopoietic stem cell transplantation and possibly gene therapy, but these treatment approaches are not available to most patients, the majority of whom reside in low- and middle-income countries. Until relatively recently, only one drug, hydroxyurea, was approved by the US Food and Drug Administration to ameliorate disease severity. Multiple other drugs (L-glutamine, crizanlizumab, and voxelotor) have recently been approved for the treatment of SCD, with several others at various stages of clinical testing. The availability of multiple agents to treat SCD raises questions related to the choice of appropriate drug therapy, combination of multiple agents, and affordability of recently approved products. The enthusiasm for new drug development provides opportunities to involve patients in low- and middle-income nations in the testing of potentially disease-modifying therapies and has the potential to contribute to capacity building in these environments. Demonstration that these agents, alone or in combination, can prevent or decrease end-organ damage would provide additional evidence for the role of drug therapies in improving outcomes in SCD.