Treatment Options for Sickle Cell Disease
Treatment Options for Sickle Cell Disease
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DOI：
10.1016/j.pcl.2018.01.005
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发表时间：
2018-06-01
影响因子：
2.6
通讯作者：
Meier, Emily Riehm
中科院分区：
文献类型：
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作者：
Meier, Emily Riehm
镰状细胞病（SCD）并发症开始于镰状血红蛋白（HbS）的聚合。因此，SCD疗法集中于预防HbS产生或减少HbS的循环量。羟基脲治疗已变得越来越普遍，而红细胞输注的循证适应症数量很少。造血干细胞移植是SCD的一种治疗选择，但只有不到25%的患者有合适的供体。本文重点介绍了支持性和预防性护理的改善以及羟基脲的益处。红细胞输注，造血干细胞移植和基因治疗试验的适应症也进行了总结。
Sickle cell disease (SCD) complications begin with the polymerization of sickle hemoglobin (HbS). Thus, SCD therapies are focused on preventing HbS production or reducing the circulating amount of HbS. Hydroxyurea treatment has become more widespread, whereas the number of evidence-based indications for erythrocyte transfusion is small. Hematopoietic stem cell transplant is a curative option for SCD, but less than 25% of patients have a suitable donor. This article focuses on supportive and preventive care improvements and the benefits of hydroxyurea. Indications for erythrocyte transfusion, hematopoietic stem cell transplant, and gene therapy trials are also summarized.