Transplantation of blood-forming stem cells for children with sickle cell disease

Sickle cell disease is a genetic disorder mainly characterized by the presence of deformed, sickle-shaped red blood cells in the blood stream. These cells deprive tissues of blood and oxygen resulting in periodic and recurrent painful attacks. Complications include acute chest syndrome and stroke. Although sickle cell disease is responsive to preventive and supportive measures such as the use of prophylactic antibodies and periodic blood transfusion, these do not provide a cure. The use of hematopoietic (blood forming) stem cell transplantation involves replacing the deformed red blood cells and the cells that produced these hematopoietic stem cells with normal cells from a healthy donor thereby producing normal red blood cells. These stem cells can be derived from either the bone marrow or blood (umbilical cord blood or peripheral blood) of a healthy individual. There are no randomized controlled trials assessing the benefits and risks; the most appropriate source of stem cells; or the most eligible participants (those who have experience severe complication or those who have not) of the procedure in children with sickle cell disease.