Questions and Answers on Bone Marrow and Cord Blood Stem Cell Transplants

Question: How is the Cord Blood Stem Cell transplant like Bone Marrow transplant?

Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar. However, the risks of graft versus host disease and graft rejection are higher, because the immunologic matching for an unrelated donor is never quite as perfect as with a matched relative donor.

Question: What are the cost and the steps to consider for bone marrow transplant?

Answer: Evaluation for Bone Marrow Transplantation is a long journey, because the risks and expenses are large. Here is an outline of what we would need to do to evaluate a child for possible bone marrow transplantation. We would certainly plan for many hours of discussion if you are interested in proceeding, but I wanted to make sure that you have a rough idea of the stepwise process:

1. SICKLE CELL ELIGIBILITY -- determine whether your son has had severe enough sickle cell disease to make the risks of bone marrow transplant worthwhile. A summary of his medical history would be very helpful, focusing on whether he has had stroke and chronic transfusion, or very frequent hospital stays for pain or for lung problems, or other major sickle cell problems. This can be done by fax or e-mail.

2. OTHER MEDICAL ISSUES -- You should make your doctor aware that your family is considering the bone marrow transplant (BMT) option. Ask the doctor to let us know whether there are any hidden medical problems that would influence the decision, such as chronic viral or other infections, problems with transfusion reactions, other medical problems unrelated to sickle cell, or anything else unusual. This does not have to be a lengthy or formal statement from the doctor at this point, more like a safety check to make sure that we are not missing any huge medical issues. A more complete medical record would be required later.

3. HLA-TYPING BLOOD TESTS -- Do immunologic typing (HLA typing) of your child and the relative with the highest probability of matching him - only brothers or sisters from the same parents are really potential donors, parents and half-siblings are very unlikely to match unless there was an unusual family tree. The HLA typing will cost several thousand dollars. If the sibling is not a full HLA match, then it is very unlikely that BMT can be done.

4. PRE-BMT EVALUATION -- after all of the above steps, then a formal evaluation by the BMT team can begin. This will include a very detailed look at his medical history and current medical condition from head to toe, plus your family's ability to cope with the BMT process. Parents will need to plan to stay in the Atlanta with the patient for a minimum of 8 months for the pre-transplant evaluation, transplant stay, and post-transplant follow-up. A financial arrangement will need to be made, with an estimated cost for the BMT process of $150,000 to $250,000. An outside expert panel will review the case on ethical grounds.

5. BMT & early follow-up period - This is a risky process, and the statistics are that there is a 5% to 8% chance of death. Death can be caused by infection, bleeding, toxic effects of the treatment, or the new bone marrow engrafting and then attacking the rest of the body. There is also a 10% to 12% chance that the child could go through the BMT process but reject the new bone marrow, ending up still having sickle cell disease. Therefore, the overall success rate of BMT for sickle cell disease is approximately 80% to 85%, of being cured of sickle cell. These require close medical follow-up, often in and out of the hospital and office daily, and many medications daily.

Question: How is the Cord Blood Stem Cell transplant like Bone Marrow Transplant?

Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar.

Question: I have a daughter that has sickle cell. If she would have a step sibling could she receive a bone narrow transplant if they match? What risk would it be if they do match and her body rejects the transplant?

Answer: The main concerns with sickle cell bone marrow transplantation are
a. the immunologic match between donor and recipient (HLA types) and
b. the health status of your daughter.

1. A full HLA match between brothers or sisters will have the very best chances for successful bone marrow transplant (BMT). Lesser degrees of match means greater chances of two bad outcomes:
a. Graft Rejection (your child goes through the BMT process but at the end her own bone marrow grows back and she still has sickle cell disease) or
b. Graft Versus Host Disease (GVHD - the transplanted marrow attacks the rest of your child's body as foreign tissue and can cause great damage.
c. very seldom will a person have a full HLA match with half-siblings or parents, unless the family tree is very inbred (for example, everyone is from the same isolated village or clan and all are related to each other's cousins. Therefore, your child's step-sister would not have a high chance of being an HLA-matched donor for BMT.

2. In addition to the chances of the two types of problems listed above (Graft Rejection and GVHD), there is a third set of bad problems that are side effects of the harsh BMT treatment process. Death may occur due to overwhelming infection, uncontrollable bleeding, and failure of organs such as liver or kidney or lungs. The chances that these bad side effects will occur are probably greater if your child is in worse health going into transplant. Therefore, the general feeling among sickle cell doctors in North and South America is that the only patients with severe sickle cell complications should be offered BMT (because only then are the high risks worthwhile), but that they be in relatively good physical condition. Your child may or may not meet these eligibility criteria.