Treating Sickle Cell Disease with Bone Marrow Transplantation

November 08, 2009

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Treating Sickle Cell Disease with Bone Marrow Transplantation

by Lewis Hsu, M.D., Ph.D.
reviewed by Laura Jana, M.D., F.A.A.P.

If the family of a child with sickle cell disease is considering bone marrow transplantation, it must first meet three requirements: The bone marrow donor must a) be a sibling; b) be an immunologic match (HLA type match); and c) not have sickle cell disease. (The donor can have sickle cell trait, however). Statistically, the odds that a child with sickle cell disease will have a suitable donor seem to be around one in five (18.75 percent).

These low odds explain why so few children with sickle cell disease become eligible for bone marrow transplantation. On top of that, the procedure is risky: Between 5 and 10 percent of children who undergo the process don't survive because of a major infection, uncontrollable bleeding, or organ failure involving the liver, kidney, lungs. Another 8 to 12 percent reject the graft (the term used to describe the transplanted bone marrow). These dangers restrict bone marrow transplants to children in relatively good physical condition and who suffer such serious complications from sickle cell disease that their families feel the risks are worth taking.

Siblings hold the key
A full HLA (or immunologic) match between brothers or sisters offer the best chances for a successful bone marrow transplant (BMT). Lesser degrees of a match increase the likelihood of two negative side effects:

Graft rejection, in which your child goes through the transplantation process, only to have her own bone marrow grow back.

Graft vs. Host Disease, in which the transplanted marrow perceives the rest of your child's body as foreign tissue and attacks it. This condition can be mild and treated with a brief course of immune-suppressant medications, or it can be quite severe and cause life-threatening damage to the body. Occasionally, it can be a chronic problem and require long-term treatment.

Parents and half-siblings seldom present a full HLA match, unless the family tree is very inbred (for example, everyone is from the same isolated village and all are related to each other's cousins). Therefore, your child's half-brother or -sister stands a slim chance of being an HLA-matched donor.

Future possibilities
A number of other transplantation options are now being explored, including transplantations that involve less chemotherapy, mid-trimester transplants for pregnant patients, and seeking out unrelated for HLA-matched umbilical cord blood. All of these approaches are extremely new, involving just one or two cases, and some results have yet to be published. None of the options involves use of fetal tissue, and, to date, no gene therapy is available for clinical use. More information is expected over the next few years.


RELATED INFORMATION

			General Medical Care for Children With Sickle Cell Disease
			Hydroxyurea Treatment for Sickle Cell Disease
			Sickle Cell Disease

Created February 04, 2001
Reviewed February 04, 2001

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