Sickle cell disease (SCD) is an inherited disorder that affects the production of red blood cells. Normal bone marrow produces red blood cells that are round, made to tumble through blood vessels as they carry oxygen to organs and tissue. The bone marrow of a child with SCD produces red blood cells that are sickle, or crescent, shaped. Instead of tumbling, they stack up in the small vessels of the body. If they stack up in the small vessels of the head and neck, your child may have a stroke. If they stack up in the small vessels of the chest, the blockage may result in lung damage. Regularly and often, the malformed cells stack up in the small vessels of the joints and cause intense, debilitating pain. Blocked blood vessels can also cause damage to most organs of the body, including spleen, liver and kidneys.
Normal red blood cells live for about 120 days in the bloodstream, flowing through vessels to deliver oxygen and remove carbon dioxide. Sickle cells live only 10 to 20 days, dying too quickly for the bone marrow to replace them at a healthy rate.
SCD is diagnosed in infancy or very early childhood. In the United States, SCD predominately affects children in the African American community, but it is an international health problem. It exists in all areas of Africa and in much of the Middle East and Asia. It also affects a large number of children in India, Italy, Greece, Spain, Portugal and a number of other countries.
Nearly 2.5 million people in the U.S. carry the sickle cell trait, which means they have inherited the gene for SCD. Both parents must carry the trait for a child to be born with sickle cell disease. It is estimated that between 70,000 and 100,000 people in the U.S. are currently living with SCD. Another 1,000 babies are born with sickle cell disease each year.
The number of people, nearly all children or young adults, living with SCD worldwide is almost impossible to calculate. In some parts of Africa, as much as 25% of the population carry the trait and 1-2% of all babies are born with the disease.
In technologically advanced countries, people living with SCD can expect to live into their mid 40’s with comprehensive treatment. In less developed countries, those born with SCD die overwhelmingly in infancy or early childhood.
Current routine treatment for SCD involves a monthly blood transfusion to replace some portion of the circulating blood with healthy red blood cells capable of carrying oxygen to organs and tissue. Long term transfusion therapy, however, creates an iron overload that will eventually damage or destroy the child’s organs. In addition to transfusion therapy, there are a number of oral medications that are used routinely to help control symptoms and protect against infection.
How Can These Children Be Helped?
A successful bone marrow stem cell transplant will cure sickle cell disease
A successful bone marrow transplant can cure SCD. The NFCTR is supporting a research collaboration between the University of Louisville and Duke University that would allow small amounts of healthy bone marrow to be safely transplanted into a child with SCD. The transplanted healthy bone marrow would produce an ongoing supply of normal red blood cells large enough to eliminate the symptoms and crisis of SCD. The research being funded easily translates into rural areas or less developed countries where a level of treatment this sophisticated is rare. This “mini” bone marrow transplant does not require a perfect donor match and can be performed as an outpatient procedure.
See what Collaborators Have to Say
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Dr. Suzanne Ildstad |
Dr. Joanne Kurtzberg |
Dr. Suhag Parikh |
Professor of Surgery, University of Louisville |
Professor of Pediatrics and Pathology, Duke University |
Assistant Professor of Pediatrics, Duke University |
Director, Institute of Cellular Therapeutics |
Director, Pediatric Blood and Marrow Transplant Program |
Pediatric Blood and Marrow Transplant Program Hematologist/Oncologist |
Jewish Hospital Distinguished Professor of Transplantation |
Director, Carolinas Cord Blood Bank |
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Igwe Family |
In the news on Wave 3 |
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Louisville, Kentucky |
Wave 3 news April 7, 2008 Louisville, Kentucky |
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Access Clinical Trial Information
Useful / Helpful links
National Heart, Lung and Blood Institute
http://www.nhlbi.nih.gov/health/
Medline Plus
http://www.nlm.nih.gov/medlineplus/
Sickle Cell Disease Association of America, Inc.
http://www.sicklecelldisease.org/
American Sickle Cell Anemia Association
http://www.ascaa.org