Diamond Blackfan Anemia Treatment: Past, Present, and Future

被引：41
作者：
Narla, Anupama [1 ,2 ,3 ]
Vlachos, Adrianna [4 ,5 ]
Nathan, David G. [1 ,2 ,3 ]
机构：
[1] Harvard Univ, Sch Med, Dana Farber Canc Inst, Boston, MA 02115 USA
[2] Brigham & Womens Hosp, Dept Med, Boston, MA 02115 USA
[3] Childrens Hosp, Boston, MA 02115 USA
[4] Steven & Alexandra Cohen Childrens Med Ctr New Yo, New Hyde Pk, NY USA
[5] Hofstra N Shore LIJ Sch Med, New Hyde Pk, NY USA
来源：
SEMINARS IN HEMATOLOGY | 2011年 / 48卷 / 02期
关键词：
ERYTHROID HYPOPLASTIC ANEMIA; RED-CELL APLASIA; MYELODYSPLASTIC SYNDROME; CYCLOSPORINE THERAPY; PROTEIN; PROLIFERATION; DIFFERENTIATION; IDENTIFICATION; GENE; RNA;
D O I：
10.1053/j.seminhematol.2011.01.004
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Despite significant improvements in our understanding of the pathophysiology of Diamond Blackfan anemia (DBA), there have been few advances in therapy. The cornerstones of treatment remain corticosteroids, chronic red blood cell transfusions, and hematopoietic stem cell transplantation, each of which is fraught with complications. In this article, we will review the history of therapies that have been offered to patients with DBA, summarize the current standard of care, including management of side effects, and discuss novel therapeutics that are being developed in the context of the research into the roles of ribosomal haplo-insufficiency and p53 activation in Diamond Blackfan anemia. Semin Hematol 48:117-123. (C) Published by Elsevier Inc.
引用
收藏
页码：117 / 123
页数：7
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