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NIH Office of the Director

Office of Medical Applications
of Research (OMAR)

For Immediate Release:
Tuesday, February 19, 2008

Lisa Ahramjian

NHLBI Communications

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.


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:

  1. 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?
  2. What is the effectiveness (in everyday practice) of hydroxyurea treatment for patients who have sickle cell disease?
  3. What are the short- and long-term harms of hydroxyurea treatment?
  4. What are the barriers to hydroxyurea treatment for patients who have sickle cell disease and what are the potential solutions?
  5. 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.

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.)


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.

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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.

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