Higgins Tara, Menditto Melissa A, Katartzis Stephanie, Matson Kelly L
Department of Pharmacy (TH), UF Health Shands Hospital, Gainesville, FL.
University of Rhode Island, College of Pharmacy (MAM, SK, KLM), Kingston, RI.
J Pediatr Pharmacol Ther. 2022;27(3):206-213. doi: 10.5863/1551-6776-27.3.206. Epub 2022 Mar 21.
Sickle cell disease is a chronic and life-limiting disorder. Approximately 100,000 Americans are affected with sickle cell disease with most being African Americans. Newborn screening for sickle cell is available in the United States, leading to early detection and management of the disease beginning in infancy. According to the 2014 National Heart, Lung, and Blood Institute sickle cell disease guidelines, supportive care has been primary management of sickle cell disease, with hydroxyurea being the only FDA-approved, disease-modifying pharmacotherapy available and allogeneic hematopoietic stem cell transplant the only cure. Since 2017, three new disease-modifying therapies have been approved by the FDA: L-glutamine, crizanlizumab, and voxelotor. This review will discuss pertinent trials, dosing, interactions, side effects, access, cost, and their role in sickle cell management.
镰状细胞病是一种慢性且危及生命的疾病。大约10万美国人患有镰状细胞病，其中大多数是非裔美国人。美国可进行镰状细胞病的新生儿筛查，从而在婴儿期就开始对该疾病进行早期检测和管理。根据2014年美国国立心肺血液研究所的镰状细胞病指南，支持性治疗一直是镰状细胞病的主要治疗方式，羟基脲是唯一获得美国食品药品监督管理局（FDA）批准的、可改变疾病进程的药物疗法，而异基因造血干细胞移植是唯一的治愈方法。自2017年以来，FDA已批准了三种新的疾病修饰疗法：L-谷氨酰胺、crizanlizumab和voxelotor。本综述将讨论相关试验、剂量、相互作用、副作用、可及性、成本及其在镰状细胞病管理中的作用。
