French physicians say they have successfully treated children with
severe sickle cell disease using stem cell transplants. The researchers have performed 69 such transplants
since 1988. They report an 85 percent disease-free survival rate.
The researchers also believe that their approach does not involve any risk of serious complications for patients.
The findings were presented recently at the annual meeting of the American Society of Hematology in Philadelphia.
If they prove to be true, they will change the standard approach to treatment for people with severe sickle cell
disease.
Sickle cell disease is a rare inherited blood disorder affecting approximately 70,000 Americans. It is characterized
by the presence of sickle or crescent shaped red blood cells in the bloodstream. These abnormally shaped cells become
rigid and lodge themselves in the very tiny blood vessels known as capillaries. The capillaries become clogged,
preventing the normal flow of oxygen to tissues.
Check here to read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.