Bone marrow transplant for sickle cell disease

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. Bone marrow transplants require bone marrow from another person (donor) and carry a high degree of risk. They are used only in cases of severe sickle cell disease for children who have minimal organ damage due to the disease. Experts consider the procedure experimental.

Before the transplant, bone marrow stem cells are taken from a healthy brother or sister who has closely matching bone marrow. 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.

Once 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.

After bone marrow 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, health professionals 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.

Bone marrow transplants offer a potential cure for a child's sickle cell disease. They are considered only for children younger than 16 who have:

• Severe sickle cell disease complications, including repeat strokes, episodes of acute chest syndrome, and painful events.
• An available donor (a healthy brother or sister who has matching bone marrow).
• No significant damage to major organs.

About 1% of people with sickle cell disease meet the criteria for bone marrow transplant.¹

If successful, a bone marrow transplant can cure sickle cell disease. Although only used in select cases, it is successful in nearly 85% of transplant recipients. Another 10% survive, despite transplant complications and/or failure. About 5% percent of bone marrow transplant recipients die following the procedure.²

• 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 medications 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.
• In some cases, this disease can cause long-term sickness or death (5% of bone marrow transplant recipients die).²
• A person may be unable to have children (infertile) after a bone marrow transplant.

• Transplanted bone marrow doesn't always work properly. This can cause life-threatening problems.
• Bone marrow transplantation is still an experimental treatment, and only about 200 people with sickle cell disease have had this procedure.³
• Very few hospitals offer this procedure.
• The risks of bone marrow transplantation 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 adults with sickle cell disease.

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Citations

1. Steinberg MH (1999). Management of sickle cell disease. New England Journal of Medicine, 340(13): 1021–1030.

2. 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/.

3. Beutler E (2006). Disorders of hemoglobin structure: Sickle cell anemia and related abnormalities. In MA Lichtman et al., eds., Williams Hematology, 7th ed., pp. 667–700. New York: McGraw-Hill.