Review
doi: 10.1053/j.seminhematol.2011.01.004.
Diamond Blackfan anemia treatment: past, present, and future
Affiliations
- PMID: 21435508
- PMCID: PMC3073777
- DOI: 10.1053/j.seminhematol.2011.01.004
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Review
Diamond Blackfan anemia treatment: past, present, and future
Semin Hematol.
2011 Apr.
Abstract
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.
Copyright © 2011 Elsevier Inc. All rights reserved.
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References
-
- Diamond LK, Allen DM, Magill FB. Congenital (erythroid) hypoplastic anemia. A 25-year study. Am J Dis Child. 1961;102:403–15. - PubMed
-
- Josephs HW. Anaemia of infancy and early childhood. Medicine. 1936;15:307.
-
- Diamond LK, Blackfan KD. Hypoplastic anemia. Am J Dis Child. 1938;56:464.
-
- Gasser C. Aplastic anemia (chronic erythroblastophthisis) and cortisone. Schweiz Med Wochenschr. 1951;81(50):1241–2. - PubMed
-
- Allen DM, Diamond LK. Congenital (erythroid) hypoplastic anemia: cortisone treated. Am J Dis Child. 1961;102:416–23. - PubMed
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