•  
  •  

Bone Marrow Transplant for Sickle Cell Disease

Treatment Overview

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 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 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, 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

Bone marrow transplants offer a potential cure for a child's sickle cell disease. They are usually 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 (someone who has closely matching bone marrow).
  • No significant damage to major organs.

The risks of bone marrow 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% of children who had transplants.1 But the risk of dying after a transplant is about 5% to 10%.2

Risks

  • 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.
  • In some cases, this disease can cause long-term sickness or death (about 5% to 10% of bone marrow transplant recipients die).2
  • A person may be unable to have children (infertile) after a bone marrow transplant.

What To Think About

  • Transplanted bone marrow doesn't always work properly. This can cause life-threatening problems.
  • Bone marrow transplant is still an experimental treatment.
  • 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.3
  • Very few hospitals offer this procedure.

Complete the special treatment information form (PDF)(What is a PDF document?) to help you understand this treatment.

References

Citations

  1. 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/.
  2. 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.
  3. Steinberg MH (2008). Sickle cell disease and associated hemoglobinopathies. In L Goldman, D Ausiello, eds., Cecil Medicine, 23rd ed., pp. 1217–1226. Philadelphia: Saunders Elsevier.

Credits

By Healthwise Staff
Primary Medical Reviewer E. Gregory Thompson, MD - Internal Medicine
Specialist Medical Reviewer Martin Steinberg, MD - Hematology
Last Revised October 7, 2010

Last Revised: October 7, 2010

Author: Healthwise Staff

Medical Review: E. Gregory Thompson, MD - Internal Medicine & Martin Steinberg, MD - Hematology

This information does not replace the advice of a doctor. Healthwise, Incorporated disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use. How this information was developed to help you make better health decisions.

To learn more visit Healthwise.org

© 1995-2012 Healthwise, Incorporated. Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Healthwise, Incorporated.