[Pages H11524-H11526]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

EXPRESSING THE SENSE OF THE HOUSE OF REPRESENTATIVES WITH RESPECT TO
DIAMOND-BLACKFAN ANEMIA

Ms. BALDWIN. Madam Speaker, I move to suspend the rules and agree to
the resolution (H. Res. 524) expressing the sense of the House of
Representatives with respect to Diamond-Blackfan Anemia, as amended.
The Clerk read the title of the resolution.
The text of the resolution is as follows:

H. Res. 524

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;
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
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
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 Diamond-Blackfan
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 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.

The SPEAKER pro tempore. Pursuant to the rule, the gentlewoman from
Wisconsin (Ms. Baldwin) and the gentleman from New York (Mr. Fossella)
each will control 20 minutes.
The Chair recognizes the gentlewoman from Wisconsin.

General Leave

Ms. BALDWIN. Madam Speaker, I ask unanimous consent that all Members
have 5 legislative days to revise and extend their remarks and include
extraneous material on the resolution under consideration.
The SPEAKER pro tempore. Is there objection to the request of the
gentlewoman from Wisconsin?
There was no objection.
Ms. BALDWIN. Madam Speaker, I yield myself such time as may consume.
I rise in support of H. Res. 524, expressing the sense of the House
with respect to Diamond-Blackfan anemia, DBA.
DBA is a rare genetic bone marrow disorder affecting children and
adults, 90 percent of whom are younger than 1 year of age when they are
diagnosed. DBA results in severe anemia due to the failure to produce
red blood cells. The symptoms may vary greatly, from very mild to
severe and life-threatening. Unfortunately, because DBA is a rare
disease, there is limited research being done, and treatment options
are not optimal.
The resolution before us today as amended expresses the sense of the
House of Representatives that we should encourage further efforts to
clarify the natural history of DBA, continue efforts to raise awareness
and ease access of information about DBA, encourage research efforts
that will advance treatment options and seek a cure and encourage
cross-institutional research initiatives to study the intricacies
involved in this rare inherited disease.
This is an important piece of legislation, and I would like to
acknowledge and thank my colleague Representative Carolyn McCarthy for
her hard work and dedication on this issue. I urge all of my colleagues
to join me in support of this legislation.
Madam Speaker, I reserve the balance of my time.
Mr. FOSSELLA. Madam Speaker, I yield myself such time as I may
consume.

[[Page H11525]]

Let me join my colleague from Wisconsin in supporting H. Res. 524 and
also acknowledging again at the outset the work and efforts of my
colleague from New York (Mrs. McCarthy).
As was mentioned, the resolution recognizes the elements of the
Diamond-Blackfan anemia and the research being done on the disease.
DBA is a blood condition, as mentioned, present at birth which is
characterized by failure of the bone marrow to produce red blood cells,
and unlike other types of anemia, DBA relates to a bone marrow failure.
It's been the result of a genetic mutation and has generally been
diagnosed at birth.
The purpose of this resolution is to bring awareness to this disease
and the research and education surrounding Diamond-Blackfan anemia. As
is always the case, although the word is rare and operative, the point
is if somebody is suffering from DBA they're suffering, and just
because there may not be tens of thousands a year, the fact is that
suffering doesn't go away.
So I would urge the adoption.
Madam Speaker, I reserve the balance of my time.
Ms. BALDWIN. Madam Speaker, I am now proud to yield 5 minutes to my
colleague, the author of this resolution, the gentlelady from New York
(Mrs. McCarthy).
Mrs. McCARTHY of New York. Madam Speaker, I'd like to thank my
colleague Ms. Baldwin for her support, and I'd also like to thank my
colleague from New York, Vito Fossella, for taking a strong stance on
this issue.
I also want to say that this bill would not have made it to the floor
without the help of my good friend and colleague, Congressman Pallone
from New Jersey, for his support in bringing it up.
It was mentioned that Diamond-Blackfan anemia, or DBA, is a rare
genetic bone marrow failure disorder that affects children and adults,
stopping the body's ability to produce red blood cells.
A lot of our colleagues might remember, every year I go around and
ask all of my colleagues to sign a book so that I can have the
opportunity to teach my colleagues about DBA, so as we go down the road
mostly hopefully to get more research money.
Ninety percent of those suffering this disease were younger than 1
year old when they were diagnosed. Children living with DBA have an
increased risk of leukemia, solid tumors, and complete bone marrow
failure. Fifty percent of patients with DBA are also born with birth
defects, including abnormalities to the face, head, upper arm and hand,
and heart. Twenty-one percent of affected patients suffer from more
than one defect.
The individuals and families suffering from rare diseases such as DBA
not only face the challenges of their life-threatening diseases, but
they must also confront the limited treatment and the research options.
Researchers believe that the study of DBA will yield clues to several
other widespread diseases, providing valuable insights into the biology
of blood disorders, blood cell formation, leukemia, and serve as an
important model for understanding the genetics of birth defects.
Unfortunately, many of the long-term treatments for DBA have the
potential for serious side effects, including impaired growth,
diabetes, and iron overload.
The only cure for DBA is a bone marrow transplant, a procedure that
carries serious risks. And since most patients lack an acceptable
donor, it's an option available for only about 25 percent of the
patients.

{time} 1715

Rare diseases, such as DBA, where there are no regional or ethnic
trends and a small number of patients, make progress in treatment and
research difficult. Thankfully, there are centers across the Nation
that devote countless hours into understanding this disease. One such
center is based out of my district on Long Island. The DBA
Comprehensive Clinical Care Center at Schneider Children's Hospital in
New Hyde Park, New York, has become the hub for the care and treatment
of DBA patients across the country. The facility is also home of the
DBA Patient Registry, which has become a valuable national resource for
families and the researchers.
The success made at Schneider's Children's Hospital have prompted the
Centers for Disease Control and Prevention's DBA Public Health Outreach
and Surveillance Program to establish three additional DBA centers in
Texas, California, and Massachusetts to further patient access,
information, treatment, and care by DBA experts. This has resulted in a
doubling of patient care visits for DBA since their establishment.
The effects are also felt on a national level. The CDC has dedicated
resources and manpower to the study of DBA as well as patient outreach.
Because of these efforts, we have seen a 25 percent increase of
enrollment of DBA patients into the DBA Patient Registry in the first 2
years of the program. The collaboration achieved through Federal
programs such as NIH and the CDC and private groups such as the Daniela
Maria Arturi Foundation and the DBA Foundation have driven the many
recent successes in the DBA field. This partnership should serve as a
model for addressing rare disease research efforts through close public
and private manners. I have been working with the Arturi family for
many years. Their daughter Daniela was affected by this rare disease,
and they have been the vocal voices for increased funding for research
and treatment. Today, we in Congress will give them and all families
suffering from this rare disease a chance of hope.
Let me say that we hear constantly of these very rare diseases, and
the families sometimes feel they have no hope. I would encourage them
to reach out on the Internet to find the information they need to. The
foundation that was started 10 years ago has come such a long way where
researchers from across the world now come in for a conference every
year to find out more and what work has been done. And even though the
cure for DBA has not happened yet, the other research has helped many,
many other families. So, please, join me in supporting this resolution
and telling the families and the children with DBA that they are not
alone. I ask my colleagues to support H. Res. 524.
Mr. FOSSELLA. Madam Speaker, I reserve the balance of my time.
Ms. BALDWIN. Madam Speaker, I am pleased to yield 2 minutes to my
colleague from New York, Congressman Bishop.
Mr. BISHOP of New York. Madam Speaker, I rise in strong support and
as a proud cosponsor of this resolution, recognizing the importance of
the Federal Government's continued support for research into the rare
bone marrow failure disorder for which there is no known cure known as
Diamond-Blackfan anemia.
I am very proud to represent Manny and Maria Arturi of Remsenberg,
NY, located in my district. After the loss of their daughter Daniela
Maria nearly 12 years ago, the foundation they created and that bears
her name continues making great strides toward the ultimate goal of
finding a cure.
When a tragic disorder like this strikes infants within the first
year of their lives, it is all the more important for Congress to go on
record voicing our unwavering support to raise awareness and broaden
support for funding rare disease research. Accordingly, this resolution
demonstrates we support giving experienced doctors the resources for
the most complete care for those patients. And by encouraging the
National Institutes of Health and Centers for Disease Control to
coordinate a multidisciplinary approach toward a cure, this legislation
brings hope that other parents will be spared from the kind of
devastation felt by the Arturis once they learned of their child's
diagnosis.
Therefore, Madam Speaker, I encourage my colleagues to support this
resolution as well as other measures that will ultimately bring about a
cure for Diamond-Blackfan anemia.
Mr. FOSSELLA. Madam Speaker, let me again congratulate Mrs. McCarthy
for bringing this to the floor, and constantly, not just here but
constantly bringing attention to DBA.
Whenever a parent gets bad news on a child and an illness, you know
it takes to the heart. And there are so many innovative, wonderful,
compassionate health care professionals who try to bring a level of
comfort to those families, and I know that here in Congress we do the
same and try to bring awareness. And although rare, or rarer

[[Page H11526]]

than many illnesses, nevertheless, the pain and suffering remains the
same.
I yield back the balance of my time.
Ms. BALDWIN. Madam Speaker, Diamond-Blackfan anemia is such a serious
condition; and because it is such a rare disease, there is a real need
for increased awareness and research. I commend my colleague Mrs.
McCarthy for her advocacy on this issue, and I urge my colleagues to
support the resolution.
I yield back the balance of my time.
The SPEAKER pro tempore. The question is on the motion offered by the
gentlewoman from Wisconsin (Ms. Baldwin) that the House suspend the
rules and agree to the resolution, H. Res. 524, as amended.
The question was taken; and (two-thirds being in the affirmative) the
rules were suspended and the resolution, as amended, was agreed to.
A motion to reconsider was laid on the table.

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