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.