Medical Treatment for Diamond Blackfan Anemia
Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal.
Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA.
This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.
|Fanconi's Anemia Hematologic Disease||Drug: Antithymocyte globulin Drug: Cyclosporine||Phase 2|
|Study Design:||Primary Purpose: Treatment|
|Official Title:||Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A|
|Study Start Date:||July 1998|
|Estimated Study Completion Date:||July 2005|
Please refer to this study by its ClinicalTrials.gov identifier: NCT00001749
|United States, Maryland|
|National Heart, Lung and Blood Institute (NHLBI)|
|Bethesda, Maryland, United States, 20892|