High-dose Chemotherapy and Stem Cell Transplant for Childhood Leukemia

A stem cell transplant (SCT), also known as a bone marrow transplant or hematopoietic stem cell transplant), can sometimes be used to help improve the chances of curing childhood leukemia.

How SCT is different from solid organ transplants

Compared to solid organ transplants that work immediately, SCT instead “plants the seeds” that allow new bone marrow to grow and make blood cells. First, high-dose chemotherapy destroys the bone marrow (where new blood cells are formed) and the leukemia. Then, SCT replaces the blood-forming stem cells and immune system in the bone marrow that were killed by the chemo. It can take weeks to months after new blood stem cells are given before the bone marrow is able to make all of the blood cells on its own.

To learn about how a SCT is done, see Stem Cell Transplant for Cancer.

Allogeneic stem cell transplant

For childhood leukemias, the type of transplant used is known as an allogeneic SCT. In this type of transplant, the blood-forming stem cells are donated from another person.

The donor’s tissue type (also known as the HLA type) should match the patient’s tissue type as closely as possible to help prevent the risk of major problems with the transplant. Tissue type is based on certain substances on the surface of cells in the body. The closer the tissue match between the donor and the recipient, the better the chance the transplanted cells will “take” and begin making new blood cells. This is different from a person’s blood type (also known as ABO or Rh).

The donor may be a sibling with the same tissue type as the patient. Other times, it can be an HLA-matched, unrelated donor—a stranger who has volunteered to donate blood-forming stem cells. Sometimes umbilical cord blood stem cells are used. These stem cells come from blood drained from the umbilical cord and placenta after a baby is born and the umbilical cord is cut. (This blood is rich in stem cells.) Whatever their source, the stem cells are then frozen and stored until they are needed for the transplant.

When a SCT might be used

Whether a SCT may be used in treatment depends on the type of leukemia.

Practical points

A SCT is a complex treatment that can cause life-threatening side effects. If the doctors think your child can benefit from a transplant, the best place to have this done is at a cancer center where the staff has experience with the procedure and with managing the recovery period.

A SCT often requires a long hospital stay and can be very expensive. Even if the transplant is covered by your insurance, your co-pays or other costs could easily amount to many thousands of dollars. It is important to find out what your insurer will cover before the transplant to get an idea of what you might have to pay.

Be sure to talk to your child’s doctor before the transplant to learn about possible long-term side effects your child might have. More information on long-term effects can be found in After Treatment for Childhood Leukemia.