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 Ane-
mia.
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled,
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Diamond-Blackfan 
Anemia Act’’. 
SEC. 2. FINDINGS. 
The Congress finds as follows:
(1) Diamond-Blackfan Anemia (in this section 
referred to as ‘‘DBA’’) is a rare genetic bone mar-
row failure disorder, which develops in infancy and 
results in severe anemia due to failure to make red 
blood cells. 
(2) Treatments for DBA, including the use of 
steroids (such as prednisone) and blood transfusions, 
have potential long-term side effects, including 
osteoporosis, impaired growth because of the 
steroids, and iron overload because of the trans-
fusions. DBA patients have an increased risk of leu-
kemia, solid tumors, and complete bone marrow fail-
ure. 
(3) The only cure for DBA is a bone marrow 
transplant, a procedure that carries serious risks 
and, since most patients lack an acceptable donor, is 
an option for only about 25 percent of patients. 
(4) Although there are a few studies focusing 
on other bone marrow failure syndromes and one in-
tramural study investigating the efficacy of stem cell 
treatment for DBA patients, there are almost no 
studies conducted at or with extramural funding 
from the National Institutes of Health that are spe-
cifically aimed at the identification, treatment, and 
cure of DBA.
SEC. 3. DIAMOND-BLACKFAN ANEMIA. 
The Public Health Service Act is amended by insert-
ing after section 424B (42 U.S.C. 285b-7b) the following: 
‘‘DIAMOND-BLACKFAN ANEMIA 
‘‘SEC. 424C. (a) IN GENERAL.—The Director of the 
Institute, in coordination with the National Heart, Lung, 
and Blood Institute and the Office of Rare Diseases, shall 
expand and intensify research and related activities of the 
Institute with regard to Diamond-Blackfan Anemia. 
‘‘(b) PROGRAMS.—In carrying out subsection (a): 
‘‘(1) SCIENTIFIC WORKSHOP.—The Director of 
the Institute shall convene a scientific workshop for 
the following: 
‘‘(A) Evaluation of the state of research on 
Diamond-Blackfan Anemia. 
‘‘(B) Determination of areas of importance 
for such research (which may include red cell 
differentiation, ribosomal protein function, can-
cer predisposition, and gene discovery). 
‘‘(C) Development of a comprehensive plan 
for conducting and supporting such research. 
‘‘(2) RESEARCH.—In accordance with the com-
prehensive 
plan 
developed 
under 
subsection 
(b)(1)(C), the Director of the Institute shall conduct 
or support research on the following:
‘‘(A) The pathophysiology of Diamond-
Blackfan Anemia. 
‘‘(B) The relationship between Diamond-
Blackfan Anemia and predisposition to cancer. 
‘‘(C) Developing better treatments and ul-
timately a cure for Diamond-Blackfan Anemia. 
‘‘(3) DATABASE.—The Director of the Institute 
shall facilitate the expansion and maintenance of, as 
well as foster the use of, the Diamond-Blackfan 
Anemia National Registry.’’.
Æ