期刊
PEDIATRIC CLINICS OF NORTH AMERICA
卷 65, 期 3, 页码 427-+出版社
W B SAUNDERS CO-ELSEVIER INC
DOI: 10.1016/j.pcl.2018.01.005
关键词
Sickle cell disease; Hydroxyurea; Transfusion; Hematopoletic stem cell transplant; Fetal hemoglobin
类别
资金
- Pfizer (ASPIRE IIR) [WI222809]
- Consultancy - CVS Caremark
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.
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