Treatment Options for Sickle Cell Disease

被引：38

作者：

Meier, Emily Riehm ^{[1
]}

机构：

[1] Indiana Hemophilia & Thrombosis Ctr, Sickle Cell Res, 8326 Naab Rd, Indianapolis, IN 46260 USA

来源：

PEDIATRIC CLINICS OF NORTH AMERICA | 2018年 / 65卷 / 03期

关键词：

Sickle cell disease; Hydroxyurea; Transfusion; Hematopoletic stem cell transplant; Fetal hemoglobin; DONOR MARROW TRANSPLANTATION; DOPPLER FLOW VELOCITIES; HYDROXYUREA THERAPY; FETAL-HEMOGLOBIN; GENE-THERAPY; SIMPLE TRANSFUSION; ANEMIA PATIENTS; YOUNG-CHILDREN; IRON OVERLOAD; RISK-FACTORS;

D O I：

10.1016/j.pcl.2018.01.005

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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.

引用

页码：427 / +

页数：18

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