I 

108TH CONGRESS 

1ST SESSION H. R. 894

To amend the Public Health Service Act to require the Director of the 

National Institutes of Health to expand and intensify research regarding 

Diamond-Blackfan Anemia. 

IN THE HOUSE OF REPRESENTATIVES 

FEBRUARY 25, 2003

Mrs. MCCARTHY of New York introduced the following bill; which was 

referred to the Committee on Energy and Commerce 

A BILL

To amend the Public Health Service Act to require the 

Director of the National Institutes of Health to expand 

and intensify research regarding Diamond-Blackfan Anemia.

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled,

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Diamond-Blackfan 

5 Anemia Act’’. 

6 SEC. 2. FINDINGS. 

7 The Congress finds as follows: 
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1 (1) Diamond-Blackfan Anemia (in this section 

2 referred to as ‘‘DBA’’) is a rare genetic bone mar3 row failure disorder, which develops in infancy and 

4 results in severe anemia due to failure to make red 

5 blood cells. 

6 (2) Treatments for DBA, including the use of 

7 steroids (such as prednisone) and blood transfusions, 

8 have potential long-term side effects, including 

9 osteoporosis, impaired growth because of the 

10 steroids, and iron overload because of the trans11 fusions. DBA patients have an increased risk of leu12 kemia, solid tumors, and complete bone marrow fail13 ure. 

14 (3) The only cure for DBA is a bone marrow 

15 transplant, a procedure that carries serious risks 

16 and, since most patients lack an acceptable donor, is 

17 an option for only about 25 percent of patients. 

18 (4) Although there are a few studies focusing 

19 on other bone marrow failure syndromes and one in20 tramural study investigating the efficacy of stem cell 

21 treatment for DBA patients, there are almost no 

22 studies conducted at or with extramural funding 

23 from the National Institutes of Health that are spe24 cifically aimed at the identification, treatment, and 

25 cure of DBA. 
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1 SEC. 3. DIAMOND-BLACKFAN ANEMIA. 

2 The Public Health Service Act is amended by insert3 ing after section 424B (42 U.S.C. 285b-7b) the following: 

4 ‘‘DIAMOND-BLACKFAN ANEMIA

5 ‘‘SEC. 424C. (a) IN GENERAL.—The Director of the 

6 Institute, in coordination with the National Heart, Lung, 

7 and Blood Institute and the Office of Rare Diseases, shall 

8 expand and intensify research and related activities of the 

9 Institute with regard to Diamond-Blackfan Anemia. 

10 ‘‘(b) PROGRAMS.—In carrying out subsection (a): 

11 ‘‘(1) SCIENTIFIC WORKSHOP.—The Director of 

12 the Institute shall convene a scientific workshop for 

13 the following: 

14 ‘‘(A) Evaluation of the state of research on 

15 Diamond-Blackfan Anemia. 

16 ‘‘(B) Determination of areas of importance 

17 for such research (which may include red cell 

18 differentiation, ribosomal protein function, can19 cer predisposition, and gene discovery). 

20 ‘‘(C) Development of a comprehensive plan 

21 for conducting and supporting such research. 

22 ‘‘(2) RESEARCH.—In accordance with the com23 prehensive plan developed under subsection 

24 (b)(1)(C), the Director of the Institute shall conduct 

25 or support research on the following: 
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1 ‘‘(A) The pathophysiology of Diamond2 Blackfan Anemia. 

3 ‘‘(B) The relationship between Diamond4 Blackfan Anemia and predisposition to cancer. 

5 ‘‘(C) Developing better treatments and ul6 timately a cure for Diamond-Blackfan Anemia. 

7 ‘‘(3) DATABASE.—The Director of the Institute 

8 shall facilitate the expansion and maintenance of, as 

9 well as foster the use of, the Diamond-Blackfan 

10 Anemia National Registry.’’.

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