Are most transplants successful and are they dangerous?

Transplants are definitely dangerous. Because the body’s immune system must essentially be shut down by high-dose chemotherapy and/or radiation to prevent rejection of the “foreign” transplanted cells, the child is at risk from even the smallest cold or other infection. In addition, neurotoxic medications are needed to prevent Graft vs. Host Disease, in which infection-fighting cells from the donor recognize the patient’s body as foreign, and then it just as if they were attacking an infection. Statistics show that the mortality rate after such transplants can be as high as 40%. The disease also seems to briefly progress more rapidly after transplant—for about six months—meaning that the child will lose some additional brain function before improving, even if the transplant is successful.

However, for boys who are candidates for a transplant, it is currently the only option to halt the disease’s destruction of brain cells. Transplantation must be considered very carefully; there are no easy answers.