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
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
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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
stem cell transplant, recovery takes 1 to 2 months
in the hospital. The child's natural defense system needs this time to start
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
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
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).
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
person may be unable to have children (infertile) after a bone marrow
What To Think About
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
National Heart, Lung, and Blood Institute, National
Institutes of Health (2002). The Management of Sickle Cell Disease (NIH Publication No. 02-2117). Available online:
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
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.
Primary Medical Reviewer
E. Gregory Thompson, MD - Internal Medicine
Specialist Medical Reviewer
Martin Steinberg, MD - Hematology
October 1, 2012
WebMD Medical Reference from Healthwise
October 01, 2012
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