TY - JOUR
T1 - Diamond Blackfan anemia treatment
T2 - Past, present, and future
AU - Narla, Anupama
AU - Vlachos, Adrianna
AU - Nathan, David G.
PY - 2011/4
Y1 - 2011/4
N2 - 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.
AB - 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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UR - http://www.scopus.com/inward/citedby.url?scp=79952747383&partnerID=8YFLogxK
U2 - 10.1053/j.seminhematol.2011.01.004
DO - 10.1053/j.seminhematol.2011.01.004
M3 - Article
C2 - 21435508
AN - SCOPUS:79952747383
SN - 0037-1963
VL - 48
SP - 117
EP - 123
JO - Seminars in Hematology
JF - Seminars in Hematology
IS - 2
ER -