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Diamond Blackfan anemia treatment: past, present, and future |
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| Authors: | Narla Anupama Vlachos Adrianna Nathan David G |
| Affiliation: | a Brigham and Women's Hospital, Department of Medicine, Boston, MA b Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA c Children's Hospital, Boston, MA d Steven and Alexandra Cohen Children's Medical Center of New York, New Hyde Park, NY e Hofstra North Shore-LIJ School of Medicine, New Hyde Park, NY |
| 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. |
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