Division of Hematology and Oncology, Department of Medicine, State University of New York Upstate Medical University, Syracuse, NY, United States of America.
Division of Hematology and Oncology, Department of Medicine, State University of New York Downstate Health Sciences University, Brooklyn, NY, United States of America.
Blood Rev. 2022 May;53:100925. doi: 10.1016/j.blre.2021.100925. Epub 2022 Jan 1.
For more than two decades, hydroxyurea was the only therapeutic agent approved by the Food and Drug Administration (FDA) for sickle cell disease (SCD). Although curative allogeneic hematopoietic stem cell transplants (allo-HSCT) were also available, only very few patients underwent the procedure due to lack of matched-related donors. However, therapeutic options for SCD patients increased dramatically in the last few years. Three new agents, l-glutamine, crizanlizumab, and voxelotor, were approved by the FDA for use in SCD patients. The number of SCD patients who underwent allo-HSCT also increased as a result of advances in the prevention of graft failure and graft-versus-host disease from using mismatched donor HSC. More recently gene therapy was made available on clinical trials. The increased treatment options for SCD have led to a sense of optimism and excitement among many physicians that these new approaches would alter the clinical course and disease burden. Although these newer agents do provide hope to SCD patients, the hyped-up responses need to be evaluated in the context of reality. In this review, we will discuss and compare these new agents and cell-based therapy, evaluate their clinical and economic impacts, and examine their roles in reducing the disease burden.
二十多年来，羟脲一直是食品和药物管理局（FDA）唯一批准用于治疗镰状细胞病（SCD）的治疗药物。尽管有治愈性的异体造血干细胞移植（allo-HSCT）也可获得，但由于缺乏匹配的相关供体，只有极少数患者接受了该手术。然而，在过去几年中，SCD 患者的治疗选择急剧增加。三种新药物，L-谷氨酰胺、crizanlizumab 和 voxelotor，被 FDA 批准用于 SCD 患者。由于在使用非匹配供体 HSC 预防移植物失败和移植物抗宿主病方面取得了进展，接受 allo-HSCT 的 SCD 患者数量也有所增加。最近，基因治疗也可用于临床试验。SCD 的治疗选择增加，使许多医生感到乐观和兴奋，他们认为这些新方法将改变临床过程和疾病负担。尽管这些新药物确实为 SCD 患者带来了希望，但需要在现实背景下评估其被夸大的反应。在这篇综述中，我们将讨论和比较这些新药物和基于细胞的治疗方法，评估它们的临床和经济影响，并研究它们在减轻疾病负担方面的作用。
