Bone Marrow (Stem Cell) Transplant for Sickle Cell Disease

Bone marrow (stem cell) transplants have been used for the treatment and cure of a variety of cancers, immune system diseases, and blood diseases for many years. Doctors in the United States and other countries have developed studies to treat children who have severe sickle cell disease with bone marrow (stem cell) transplants. In a person with sickle cell disease, the bone marrow produces red blood cells that contain hemoglobin S. This leads to the complications of sickle cell disease. • To prepare for a bone marrow (stem cell) transplant, strong medicines, called chemotherapy, are used to weaken or destroy the patient’s own bone marrow, stem cells, and infection fighting system. – This is done so the patient does not reject the new blood cells coming from the donor. • The patient’s bone marrow then is replaced with blood-forming stem cells from a donor who does not have sickle cell disease. – This can be a donor with normal hemoglobin or sickle cell trait. – The actual transplant is given like a blood transfusion through an IV tube.   Abstract source