107th Congress

Diamond-Blackfan Anemia Act

H.R. 3014

Background

Diamond-Blackfan Anemia (DBA) is a rare disorder of the bone marrow caused by a mutation in a gene that codes for the production of red blood cells. Current treatments, such as steroids and blood transfusions, have potential longterm side effects. For instance, steroid treatment may cause osteoporosis or impaired growth, and transfusions may cause iron overload. As patients reach adulthood, they have an increased risk of developing acute leukemia or bone marrow failure. A bone marrow transplant, an inherently risky procedure, is currently the only cure for DBA. However, only 25 percent of patients are able to find an acceptable bone marrow donor.

Provisions of the Legislation/Impact on NIH

H.R. 3014, the Diamond-Blackfan Anemia Act, would have required the Director of the National Heart, Lung, and Blood Institute (NHLBI), in coordination with the Office of Rare Diseases, to expand and intensify research and related activities on DBA. In carrying out this mandate, the Director of NHLBI would have been required to convene a scientific workshop to evaluate the state of DBA research, determine important areas of DBA research, and develop a comprehensive plan for conducting and supporting DBA research. In accordance with the comprehensive plan, NHLBI would have been required to conduct and support research on the pathophysiology of DBA, the relationship between DBA and predisposition to cancer, and the development of improved treatments and an ultimate cure for DBA. Finally, NHLBI would have been required to facilitate the expansion, maintenance, and use of the DBA National Registry.

Status and Outlook

H.R. 3014 was introduced by Representative Carolyn McCarthy (D-NY) on October 3, 2001, and was referred to the House Energy and Commerce Committee. On October 15, it was sent to the House Energy and Commerce Subcommittee on Health. There was no further action on this legislation during the 107th Congress.