H. Res. 524 

In the House of Representatives, U. S., 

October 15, 2007. 

Whereas Diamond-Blackfan Anemia (‘‘DBA’’) is a rare genetic bone marrow failure disorder affecting children and 

adults, 90 percent of whom are younger than 1 year of 

age when they are diagnosed, and results in severe anemia due to failure to produce red blood cells; 

Whereas individuals and families suffering with rare diseases 

such as DBA not only face the challenges of their debilitating and life-threatening diseases, but must also confront the consequences of their rare disease status; 

Whereas individuals suffering from rare diseases need access 

to treatment options and the potential for a cure; 

Whereas research is proving the study of complex, rare diseases such as DBA yield tremendous advancements in 

other, larger disease areas that affect millions of Americans; 

Whereas the children living with DBA have an increased risk 

of leukemia, solid tumors, and complete bone marrow 

failure, and 50 percent of patients with DBA are born 

with birth defects including abnormalities to the face, 

head, upper arm and hand, genitourinary, and heart with 

21 percent of affected patients having more than 1 defect; 
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Whereas the study of DBA will yield the true incidence of 

aplastic anemia, myelodysplastic syndrome, leukemia, and 

the predisposition to cancer in DBA and will serve as an 

important model for understanding the genetics of birth 

defects; 

Whereas 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, diabetes, and iron 

overload because of the transfusions; 

Whereas 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 available for 

only about 25 percent of patients; 

Whereas rare diseases, such as DBA, benefit greatly from 

well-established comprehensive care centers such as the 

DBA Comprehensive Clinical Care Center at Schneider 

Children’s Hospital in New Hyde Park, New York (the 

‘‘Center’’), which has become the multidimensional hub 

for the care and treatment of DBA patients across the 

country, as well as the home of the DBA Patient Registry which has become a valuable national resource for 

investigators utilizing the Center to accomplish research 

in a multitude of areas not specific only to DBA; 

Whereas the successful establishment of the Center became 

a model for how to diagnose, treat, and improve the lives 

of patients with rare diseases, while learning from the 

disorder to yield advancements in other areas of disease 

research; 

Whereas the success of the initial Center prompted the Centers for Disease Control and Prevention’s DBA Public 
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Health Outreach and Surveillance Program to establish 3 

additional DBA Centers in Texas, California, and Massachusetts to further patient access to information, treatment, and care by DBA experts, which has resulted in 

a doubling of patient care visits for DBA care and surveillance since their establishment; 

Whereas the DBA Public Health Outreach and Surveillance 

Program at the Centers for Disease Control and Prevention (‘‘CDC’’) has resulted in the completion of the first 

CDC brochure for the DBA patient population, the introduction of a DBA hotline and dedicated DBA nurse, and 

has resulted in a 25-percent increase of enrollment of 

DBA patients into the DBA Patient Registry in the first 

2 years of the program; 

Whereas the collaboration between the National Institutes of 

Health and the Centers for Disease Control and Prevention and their close collaboration with the Daniella Maria 

Arturi Foundation and the DBA Foundation have driven 

the many recent successes in the DBA field and serve as 

a model for addressing rare disease research efforts 

through close public and private collaboration to achieve 

the highest levels of success in the areas of improved patient care and disease research; 

Whereas the interagency collaboration achieved within the 

National Institutes of Health between the National 

Heart, Lung, and Blood Institute, the National Institute 

of Diabetes and Digestive and Kidney Diseases, the National Cancer Institute, and the Office of Rare Diseases 

to advance the research and understanding of DBA has 

resulted in significant advancements not only in the DBA 

scientific arena, but in understanding its many links to 

more prevalent disorders; and 
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Whereas the DBA research initiatives have already yielded 

tremendous success including the discovery of 2 

ribosomal protein (‘‘RP’’) genes and the identification 

that DBA is the first human disease linked to a 

ribosomal protein problem which, as a fundamental unit 

of cellular function, has been implicated in a wide range 

of human disorders including cancer, making this discovery a profound example of the additional benefits that 

may result from the study of DBA: Now, therefore, be 

it 

Resolved, That the House of Representatives— 

(1) recognizes that the identification of DiamondBlackfan Anemia (‘‘DBA’’) may advance the understanding of DBA, identify implications of cancer predisposition, and serve as an important model for understanding human development and the molecular basis for 

certain birth defects; 

(2) recognizes the importance of comprehensive care 

centers in providing complete care and treatment for 

each patient, leading to an increase in correct and early 

diagnosis; 

(3) commends Schneider Children’s Hospital for 

providing the first DBA Comprehensive Clinical Care 

Center for patients across the country, for developing 

the DBA Patient Registry which has proven a robust 

surveillance tool to understand the epidemiology, biology, 

and treatment of DBA, and for proving a valuable resource for investigators at a national level, working to 
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understand DBA’s link to more prevalent disorders facing Americans; 

(4) commends the Daniella Maria Arturi Foundation and the Diamond-Blackfan Anemia Foundation for 

their efforts to facilitate the successful collaboration 

among the National Institutes of Health and the Centers 

for Disease Control and Prevention to achieve a successful multidisciplinary approach between clinical and scientific DBA efforts with the goal of shortening the life 

cycle of success realized between the laboratory and applied patient care; and 

(5) encourages research efforts to further understand ribosomal protein deficiencies in rare inherited diseases and to advance the treatment options available to 

those with DBA. 

Attest: 

Clerk.