Ballas Samir K
Department of Medicine, Cardeza Foundation for Hematologic Research, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA, USA.
Clin Hemorheol Microcirc. 2018;68(2-3):105-128. doi: 10.3233/CH-189002.
Sickle cell disease (SCD) is an inherited disorder of hemoglobin structure that has no established cure in adult patients. Cure has been achieved in selected children with sickle cell anemia (SCA) using allogeneic bone marrow transplantation or cord blood transplantation. SCD is essentially a triumvirate of (1) pain syndromes, (2) anemia and its sequelae and (3) organ failure, including infection. Pain, however, is the hallmark of SCD and dominates its clinical picture throughout the life of the patients. The prevalence of these complications varies with age from infancy through adult life. However, pain, infections and anemia requiring blood transfusion occur throughout the life span of affected patients. The overall medical care of patients with SCD in developed countries has improved such that their life expectancy has almost doubled since 1951. Currently, there are at least five major approaches for the general management of SCD and its complications. These include (i) symptomatic management, (ii) supportive management, (iii) preventive management, (iv) abortive management, and (v) curative therapy.
镰状细胞病（SCD）是一种血红蛋白结构的遗传性疾病，成年患者尚无既定的治愈方法。通过异基因骨髓移植或脐血移植，部分镰状细胞贫血（SCA）患儿已实现治愈。SCD本质上是由（1）疼痛综合征、（2）贫血及其后遗症以及（3）器官衰竭（包括感染）组成的三联征。然而，疼痛是SCD的标志，在患者一生中主导着其临床表现。这些并发症的患病率随年龄从婴儿期到成年期而有所不同。然而，疼痛、感染和需要输血的贫血在受影响患者的整个生命周期中都会出现。发达国家SCD患者的总体医疗护理有所改善，自1951年以来他们的预期寿命几乎翻了一番。目前，SCD及其并发症的一般管理至少有五种主要方法。这些方法包括（i）对症治疗、（ii）支持治疗、（iii）预防治疗、（iv）顿挫治疗和（v）治愈性治疗。
