NIH to Hold Consensus
Development Conference on Hydroxyurea Treatment for Sickle
Cell Disease, February 25 – 27
Panel will assess hydroxyurea’s impact on this painful
condition
For many patients, the pain and complications associated
with sickle cell disease can have a profound impact on
their quality of life, ability to work, and long-term health
and well-being. Unfortunately, these challenges are often
coupled with significant barriers to care. Hydroxyurea
is an FDA-approved therapy for adults with certain forms
of sickle cell disease, and acts to increase the percentage
of non-sickled red blood cells in circulation. However,
concerns remain for both physicians and patients. The conference
panel will examine these issues in detail at the upcoming
conference, resulting in a summary of what we know and
what we need to learn about hydroxyurea treatment for sickle
cell disease. |
What: |
Experts will describe the available evidence
on hydroxyurea treatment for sickle cell disease, including
efficacy, effectiveness, harms, barriers to treatment,
and future research needs. Following a series of scientific
presentations and open public discussions, an impartial,
independent panel will issue a statement of its findings
on the final day of the conference, and will hold a press
conference at 2:00 p.m. on Wednesday, February 27. Convened
by the Office of Medical Applications of Research (OMAR)
and the National Heart, Lung, and Blood Institute (NHLBI)
of the NIH, this conference is free and open to the public
and the media.
The conference presentations, open discussions, and the
panel's statement will focus on these questions:
- What is the efficacy (results from clinical studies)
of hydroxyurea treatment for patients who have sickle
cell disease in three groups: infants, preadolescents,
and adolescents/adults?
- What is the effectiveness (in everyday practice) of
hydroxyurea treatment for patients who have sickle cell
disease?
- What are the short- and long-term harms of hydroxyurea
treatment?
- What are the barriers to hydroxyurea treatment for
patients who have sickle cell disease and what are the
potential solutions?
- What are the future research needs?
|
When: |
Monday, February 25, 2008 - 8:30 am - 5:30
pm
Tuesday, February 26, 2008 - 8:30 am - 12:00 pm
Wednesday, February 27, 2008 - 9:00 am - 11:00 am
Press Conference: Wednesday, February 27,
2:00 p.m. |
Where: |
Natcher Conference Center
NIH Main Campus - Building 45
9000 Rockville Pike
Bethesda, Maryland 20892
Campus visitor information: http://www.nih.gov/about/visitor/index.htm
The conference will also be webcast live at http://videocast.nih.gov/.
(Reporters viewing the press conference via webcast will
be able to submit questions on-line through the videocast
website.) |
Why: |
Sickle cell disease is an inherited blood
disorder that affects between 50,000 and 75,000 people
in the United States, and is most common among people whose
ancestors come from sub-Saharan Africa, South and Central
America, the Middle East, India, and the Mediterranean
basin. Sickle cell disease occurs when an infant inherits
the gene for sickle hemoglobin from both parents (Hb SS,
or sickle cell anemia), or the gene for sickle hemoglobin
from one parent and another abnormal hemoglobin gene from
the other parent. Each year, approximately 2,000 babies
with sickle cell disease are born in the United States. The
condition is chronic and lifelong, and is associated with
a decreased lifespan and diminished quality of life. Infections
and lung problems are the leading cause of death. In
addition, approximately 2 million Americans carry the sickle
cell trait, which increases the public health burden as
this disorder is passed on to future generations.
The red blood cells in people with sickle cell disease
become deoxygenated (or depleted of oxygen), and crescent-shaped
or "sickled." The sickled blood cells
become sticky, and adhere to blood vessel walls, thereby
blocking blood flow within limbs and organs. These
changes lead to acute painful episodes, chronic pain, and
chronic damage to the brain, heart, lungs, kidneys, liver,
joints, eyes, and spleen. Pain crises are responsible
for most emergency room visits and hospitalizations of
people with sickle cell disease. Standard treatments
for acute pain crises include painkilling medications,
fluid replacement, and oxygen.
In the mid-1990s, researchers began investigating the
potential of hydroxyurea to reduce the number and severity
of pain crises in sickle cell patients. Hydroxyurea
is in a class of anti-cancer drugs and it acts to increase
the overall percentage of normally structured red blood
cells in the circulation. By diluting the number
of cells that "sickle," it may, if taken on
a daily basis, reduce their damaging effects. Hydroxyurea
was approved by the FDA for use in adults with certain
forms of sickle cell anemia in 1998. However, there
are a number of unresolved issues about the use of hydroxyurea,
including a lack of knowledgeable providers who treat sickle
cell disease, and patient and practitioner questions about
safety and effectiveness, including concerns regarding
potential long-term carcinogenesis. |
Background: |
The conference is presented through the NIH
Consensus Development Program. A fact sheet describing the
conference process is available at http://consensus.nih.gov/forthemedia.htm. |
For More Information: |
Conference agenda, speakers, logistics,
and online registration are available at http://consensus.nih.gov.
To schedule interviews, please contact Lisa Ahramjian
at AhramjianL@od.nih.gov or
(301) 496-4999. |
Note to Reporters: |
Reporters viewing the press conference via
webcast at http://videocast.nih.gov will
be able to submit questions on-line through the videocast
Web site beginning at 1:30 p.m. on Wednesday, February 27. |
Note to TV Editors: |
The press conference on Wednesday, February
27 will be broadcast live via satellite at the following
coordinates:
Satellite C-Band
G3 transponder 23
Orbital location: 95 degrees west
Downlink frequency: 4160H
Audio: 6.2/6.8
Test Time: 1:30 - 2:00 p.m.
Broadcast: 2:00 - 3:00 p.m. |
The Office of the Director, the central office at NIH, is responsible
for setting policy for NIH, which includes 27 Institutes and Centers.
This involves planning, managing, and coordinating the programs
and activities of all NIH components. The Office of the Director
also includes program offices which are responsible for stimulating
specific areas of research throughout NIH. Additional information
is available at http://www.nih.gov/icd/od/.
The National Institutes of Health (NIH) — The Nation's
Medical Research Agency — includes 27 Institutes and
Centers and is a component of the U.S. Department of Health and
Human Services. It is the primary federal agency for conducting
and supporting basic, clinical and translational medical research,
and it investigates the causes, treatments, and cures for both
common and rare diseases. For more information about NIH and
its programs, visit www.nih.gov. |