Bone marrow transplantation works to cure sickle cell disease by replacing unhealthy blood-forming cells with healthy cells. There are a number of steps involved in this process:
1. The Pre-Transplant Phase: During this phase, we evaluate your child to see if he or she is eligible for transplantation by conducting a physical exam and a series of tests. We'll also provide you with information about the process and what to expect. As a result of this evaluation (which we conduct on an outpatient basis), we'll choose the best treatment plan for your child.
2. Finding a Donor: Bone marrow transplantation requires a close match between the HLA tissue type of the patient and the donor. HLA stands for "human leukocyte antigen," a marker that the immune system uses to recognize which cells belong in the body and which do not. During the pre-transplant phase, we'll try to identify a donor for your child. A sibling without sickle cell disease typically offers the best chance for a match. For children without a matched sibling donor, we can conduct a search for an unrelated donor through the National Marrow Donor Program (also known as the Be The Match Registry) and other worldwide registries.
3. The Conditioning/Transplant Phase: Your child will stay in the hospital for an average of 8 to 12 weeks during this phase. For the first week of this hospital stay, we will give chemotherapy once or twice a day for seven to eight days to destroy your child's old bone marrow and promote acceptance of the donor's bone marrow, which we give one to two days after chemotherapy is done. We provide all the care your child needs during this time and support for you and your family as well.
4. The Recovery Phase: Your child's recovery begins in the hospital after receiving the healthy bone marrow. Your child may leave the hospital once we see that the bone marrow has been accepted and your child has recovered from the side effects of the chemotherapy. In the first months after transplant, the focus will be on recovery: eating well, avoiding infections, monitoring for complications, taking medications, and regular outpatient visits. We continue to monitor and support your child on an outpatient basis for another six to twelve months. While every child is different, for most children, the return to normal activity and school takes about one year.