Diamond Blackfan Anemia Treatment: Past, Present, and Future SEMINARS IN HEMATOLOGY Narla, A., Vlachos, A., Nathan, D. G. 2011; 48 (2): 117-123

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.

View details for DOI 10.1053/j.seminhematol.2011.01.004

View details for Web of Science ID 000289037800007

View details for PubMedID 21435508