Treatment Overview
Stem cell transplant is a possible cure for sickle cell disease. It's usually considered only for children younger than 16 who have severe complications from the disease.
Stem cells can be found in bone marrow. Bone marrow is the substance in the center of your bones that produces red blood cells. A person with sickle cell disease has bone marrow that produces red blood cells with defective hemoglobin S. But if that bone marrow is replaced with healthy bone marrow, a person's body may start to produce normal hemoglobin.
Stem cell transplants require bone marrow from another person (donor). This is called an allogeneic stem cell transplant.
Before the transplant, bone marrow stem cells are taken from someone who has closely matching bone marrow. This is most often a healthy brother or sister. The child who has sickle cell disease is then treated with drugs that destroy the child's bone marrow cells. After that, the donated bone marrow stem cells are injected into a vein.
After the process is complete, the donor's bone marrow starts to replace the recipient's bone marrow. These new cells restore the immune system and make normal red blood cells.
Until recently this treatment was mostly done for children. That's because the risk for serious problems increases with a person's age. But new methods of doing stem cell transplant are making it possible to successfully treat adults.
