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High-dose Chemotherapy and Stem Cell Transplant for Childhood Leukemia

A stem cell transplant (SCT) (also known as a bone marrow transplant) can sometimes be used to help improve the chances of curing childhood leukemia. SCT lets doctors use even higher doses of chemotherapy than a child could normally tolerate.

High-dose chemotherapy destroys the bone marrow, which is where leukemia starts, but it's also where new blood cells are formed. This could lead to life-threatening infections, bleeding, and other problems caused by low blood cell counts. A stem cell transplant is given after the chemo to restore the blood-forming stem cells in the bone marrow.

The blood-forming stem cells used for a transplant can come either from the blood or from the bone marrow. Sometimes stem cells from a baby’s umbilical cord blood are used.

Allogeneic stem cell transplant

For childhood leukemias, the type of transplant used is known as an allogeneic stem cell transplant. 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.

The donor is usually a brother or sister with the same tissue type as the patient. Rarely, it can be an HLA-matched, unrelated donor – a stranger who has volunteered to donate blood-forming stem cells. Sometimes umbilical cord 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.

To learn about how a stem cell transplant is done, see Stem Cell Transplant for Cancer.

When a stem cell transplant might be used

Acute lymphocytic leukemia (ALL): In ALL, SCT might be used in children in some high-risk groups, whose leukemia is more likely to come back after the initial (induction) chemo. In this case, the transplant is done after the induction chemo puts the leukemia into remission.

SCT might also be an option if the leukemia doesn’t respond well to initial treatment, or if it relapses (comes back) soon after going into remission. It’s less clear if SCT should be used for children whose ALL relapses later (such as more than 6 months or a year) after finishing the initial chemo. These children will often do well with another round of standard dose chemo.

SCT may also be recommended for children with some less common forms of ALL, such as those whose leukemias have the Philadelphia chromosome or those with T-cell ALL that don’t respond well to initial treatment.

Acute myelogenous leukemia (AML): Because AML relapses more often than ALL, SCT might be recommended right after the AML has gone into remission (after the initial chemo treatment), if the child has a brother or sister with the same tissue type who can donate stem cells for the transplant. This is especially true if there is a very high risk of relapse (as with some subtypes of AML or when there are certain gene or chromosome changes in the leukemia cells). There is still some debate about which children with AML need this type of intensive treatment.

If a child with AML relapses after their first round of standard chemo, most doctors will recommend SCT as soon as the child goes into remission again.

In either case, it is important that the leukemia is in remission before getting a stem cell transplant. Otherwise, the leukemia is more likely to return.

Other leukemias: SCT might also offer the best chance to cure some less common types of childhood leukemia, such as juvenile myelomonocytic leukemia (JMML) and chronic myelogenous leukemia (CML). For CML, newer targeted therapy drugs are likely to be used first for most children, but a transplant might still be needed at some point.

Practical points

A stem cell transplant 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 stem cell transplant 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’s 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 Living as a Childhood Leukemia Survivor.

More information about stem cell transplant

To learn more about stem cell transplants, including how they are done and their potential side effects, see Stem Cell Transplant for Cancer.

For more general information about side effects and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as editors and translators with extensive experience in medical writing.

Horton TM, Steuber CP. Overview of the treatment of acute lymphoblastic leukemia in children and adolescents. UpToDate. 2018. Accessed at on December 29, 2018.

National Cancer Institute. Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®)–Health Professional Version. Accessed at on December 29, 2018.

National Cancer Institute. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Health Professional Version. Accessed at on December 29, 2018.

Tarlock K, Cooper TM. Acute myeloid leukemia in children and adolescents. UpToDate. 2018. Accessed at on December 29, 2018.

Last Revised: February 12, 2019

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