Stem Cell Transplant for Sickle Cell Disease
Stem cell transplantation is a potential cure for sickle cell 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, usually a healthy brother or sister. The child who has sickle cell disease is then treated with drugs that destroy his or her 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 begins to replace the recipient's bone marrow. These new cells restore the immune system and make normal red blood cells.
What To Expect After Treatment
After stem cell transplant, recovery takes 1 to 2 months in the hospital. The child's natural defense system needs this time to start working again.
During recovery, doctors watch closely for signs that the immune system is rejecting the new bone marrow and for signs of infection. If a problem occurs, recovery can take longer or the transplant may fail.
Why It Is Done
Stem cell transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for children younger than 16 who have:1
- Severe sickle cell disease complications, including repeat strokes, episodes of acute chest syndrome, and painful events.
- An available donor (someone who has closely matching bone marrow).
The risks of stem cell transplant become greater as a person gets older and/or develops damage to major organs. For these reasons, a bone marrow transplant is not a treatment option for most adults who have sickle cell disease. But research on bone marrow transplants in adults is ongoing.
How Well It Works
If successful, a bone marrow transplant can cure sickle cell disease. This treatment has been successful in about 85 out of 100 children who had transplants.1, 2 But the risk of dying after a transplant is about 5% to 10%.3
- Any bone marrow transplant is risky, because
complications, such as severe infections and immune system problems, can occur.
- If the recipient's natural defense (immune) system isn't weakened enough by the medicines given before the transplant, it can attack the new stem cells and cause the transplant to fail.
- If the donor's stem cells don't match the recipient's closely enough, the donor's new immune system cells may attack certain organs in the recipient (graft-versus-host disease).
- Other complications can include seizures and bleeding in the brain.
- Life-threatening problems can happen (about 5 to 10 out of 100 bone marrow transplant recipients die).3
- A person may be unable to have children (infertile) after a bone marrow transplant.
What To Think About
- Bone marrow transplants are seldom used. Few children meet the criteria. And donors are almost always siblings, so finding a matching bone marrow donor may not be possible. The cost of the procedure is also a barrier.
- About 1 out of 10 people with sickle cell disease have a matching donor.4
- Very few hospitals offer this procedure.
- National Heart, Lung, and Blood Institute, National Institutes of Health (2002). The Management of Sickle Cell Disease (NIH Publication No. 02-2117). Available online: http://www.nhlbi.nih.gov/health/prof/blood/sickle/.
- Ratko TA, et al. (2012). Hematopoietic Stem-Cell Transplantation in the Pediatric Population. Comparative Effectiveness Review No. 48 (AHRQ Publication No. 12-EHC018-EF). Rockville, MD: Agency for Health Care Research and Quality.
- Wang WC (2009). Sickle cell anemia and other sickling syndromes. In JP Greer et al., eds., Wintrobe's Clinical Hematology, 12th ed., pp. 1038–1082. Philadelphia: Lippincott Williams and Wilkins.
- Steinberg MH (2012). Sickle cell disease and other hemoglobinopathies. In L Goldman, A Shafer, eds., Goldman's Cecil Medicine, 24th ed., pp. 1066–1075. Philadelphia: Saunders.
Last Revised: October 1, 2012
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