A bone marrow transplant (BMT) can be a lifesaving treatment for children who have blood cancers, such as leukemia, Hodgkin's lymphoma and non-Hodgkin's lymphoma; inherited blood disorders, such as sickle cell anemia and thalassemia; or bone marrow diseases, such as anaplastic anemia.
The goal of a BMT is to replace the diseased or damaged tissue inside bones with healthy, functional tissue. This soft, spongy tissue – bone marrow – contains stem cells that can produce red blood cells, which carry oxygen; white blood cells, which fight infection; and platelets, which help blood clot. In a BMT procedure, the patient is infused with healthy stem cells that will migrate to the bones and start producing new blood cells. The transplanted cells may come from a healthy marrow donor or, if possible, may be healthy stem cells collected from the patient's own blood. The long-term survival rate for children varies depending on the disease being treated. For leukemia, there is a wide range, from 30 percent to 70 percent; for genetic diseases, the rate is 80 percent to 95 percent.
Before the transplant, the patient undergoes a conditioning regimen designed to help the transplant succeed. Patients may receive chemotherapy, other drugs, radiation therapy or some combination of these, with the goals of preventing the immune system from rejecting the transplanted cells, making room in the bone marrow for the new stem cells, and killing any cancer cells remaining in the body.
The BMT process
The process can be broken down into six phases:
- The transplant decision
- Before admission
- Admission to the BMT unit
- Day of the transplant
- After the transplant
- Leaving the hospital
Our BMT program uses donor cells from several types of donors, including the patient. If a matched relative can’t be found, we may use partially matched relatives, unrelated donors or other alternative donors.
Although BMT is the best and sometimes only treatment option for some diseases, it carries certain risks, especially with repeated marrow transplants. Risks include damage to healthy tissues, such as the brain, lungs, liver and kidneys. To minimize this damage, we have developed novel protocols, such as the following:
- Reduced-intensity conditioning
- Busulfan pharmacokinetic studies (to optimize dosing)
- Umbilical cord blood transplants
The post-transplant period is a critical stage in which patients may develop complications of chemotherapy or radiation therapy as well as a reaction known as graft-versus-host disease. After a BMT, the increased risk of infections may last six to 18 months. Your child’s doctor and the UCSF transplant team will monitor your child’s health carefully.
If your child is a prospective patient, you can take an online tour of the BMT unit and follow-up clinic to see where your child will stay and receive treatments. You’ll also need to learn about the special dietary concerns of BMT recipients (following certain guidelines will help make the transplant successful), and it’s wise to prepare yourself, your child and your family for the emotional aspects of the transplant journey.
Family members and friends who plan to visit a BMT patient in the hospital should be aware of the special rules they'll need to follow.