Journal
SEMINARS IN HEMATOLOGY
Volume 48, Issue 2, Pages 117-123Publisher
W B SAUNDERS CO-ELSEVIER INC
DOI: 10.1053/j.seminhematol.2011.01.004
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Funding
- NHLBI NIH HHS [R01 HL082945, K08 HL078818-05, K08 HL078818] Funding Source: Medline
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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.
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