What is sickle cell disease?

Sickle cell disease is an inherited blood disorder that primarily affects the red blood cells. People with sickle cell disease can suffer many problems from the disease including stroke, invasive bacterial infection, and lung disease. One of the most common problems is pain. This pain, which can occur throughout the body, is often extremely severe. People with sickle cell disease have described sickle cell pain as “like a hammer, beating and beating,” and “like being hit by a truck and getting run over.”

Who has sickle cell disease?

Millions of people from all around the world have sickle cell disease. In the United States, the disease is most common among people of African descent. The Center for Disease Control estimates that 1 out of every 500 African American newborns has sickle cell disease.

 

Is there a cure for sickle cell disease?

Yes.

Blood and marrow transplant is currently the only cure for sickle cell disease.

 

How does a blood and marrow transplant work?

A blood and marrow transplant is medical procedure, not a surgery.  It involves giving medicines to a patient to shut down his or her own production of blood cells so that he or she can accept new stem cells from a donor.  The new stem cells are given like a blood transfusion.  The patient must then continue to take medicines for some time after the transplant to ensure that the donor stem cells and the patient “accept” each other, allowing for the healthy growth of  new blood cells.

Why don’t all children with sickle cell disease get a blood and marrow transplant?

Blood and marrow transplants have serious risks that include death.  Blood and marrow transplants also work best with a healthy “matched” sibling donor, but most children with sickle cell disease do not have a sibling donor.

Through research led by STAR investigators, we can refine the transplant process to decrease its risks and safely perform transplants using alternative donors so that one day all children with sickle cell disease can look forward to a long, healthy life.