Sickle Cell Disease A Brief Update

被引:57
作者
Azar, Shari [1 ]
Wong, Trisha E. [2 ,3 ]
机构
[1] Oregon Hlth & Sci Univ, Div Hematol & Med Oncol, Dept Med, 3181 Southwest Sam Jackson Pk Rd,Mailstop L586, Portland, OR 97239 USA
[2] Oregon Hlth & Sci Univ, Div Hematol Oncol, Dept Pediat, 3181 Southwest Sam Jackson Pk Rd,Mailstop CDRCP, Portland, OR 97239 USA
[3] Oregon Hlth & Sci Univ, Dept Pathol, Div Transfus Serv, 3181 Southwest Sam Jackson Pk Rd,Mailstop HRC9, Portland, OR 97239 USA
关键词
Sickle cell disease; Sickle cell anemia; Hemoglobinopathy; Hydroxyurea; Iron overload; Review; SILENT CEREBRAL INFARCTS; FETAL-HEMOGLOBIN; YOUNG-ADULTS; NITRIC-OXIDE; TRANSFUSION MANAGEMENT; MEDICATION ADHERENCE; CHELATION TREATMENT; PEDIATRIC-PATIENTS; CONJUGATE VACCINE; CONTROLLED-TRIAL;
D O I
10.1016/j.mcna.2016.09.009
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Sickle cell disease (SCD) is an inherited monogenic disease characterized by misshapen red blood cells that causes vaso-occlusive disease, vasculopathy, and systemic inflammation. Approximately 300,000 infants are born per year with SCD globally. Acute, chronic, and acute-on-chronic complications contribute to end-organ damage and adversely affect quantity and quality of life. Hematopoietic stem cell transplantation is the only cure available today, but is not feasible for the vast majority of people suffering from SCD. Fortunately, new therapies are in late clinical trials and more are in the pipeline, offering hope for this unfortunate disease, which has increasing global burden.
引用
收藏
页码:375 / +
页数:20
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