Stem Cell Transplantation for Leukemia in Children

When targeted chemotherapy drugs do not lead to remission in children with chronic myelogenous leukemia, doctors at Hassenfeld Children’s Hospital at NYU Langone may recommend a stem cell transplant, also called a bone marrow transplant. Stem cell transplantation is also sometimes recommended for children with acute leukemias, those with acute myeloid leukemia who are classified in a high-risk group at diagnosis, those whose cancer has returned after remission, or those whose cancer isn’t responding well to chemotherapy.

In stem cell transplantation, a high-dose chemotherapy drug is delivered with an intravenous infusion for a period of days to destroy cancer cells. This intense form of treatment also destroys healthy blood cells, including stem cells. These cells are then replaced with healthy stem cells provided by a donor—typically a sibling or other relative or a matched unrelated donor. Using stem cells from another person is called an allogeneic transplantation.

Including recovery time, your child may spend several weeks to months in the hospital during the transplantation process.

Finding a Donor

Stem cell transplantation requires a donation of bone marrow stem cells from a healthy person who is a “match,” meaning the person’s genetic makeup and tissue types are compatible with those of the child with leukemia. Siblings tend to make the best matches. If needed, our doctors can help to locate an unrelated match using national bone marrow registries.

Usually, it takes two to three months to find the appropriate donor or donors and to perform the necessary testing to make sure their stem cells are safe for your child. If it takes longer than expected to find a suitable donor, your doctors may recommend an additional cycle of chemotherapy until the search is complete.

Stem Cell Collection and Transplantation

After a donor is found to be a good match, stem cells are collected, or harvested, from the donor’s body in one of two ways. In a peripheral blood stem cell harvest, blood is collected from the donor, and stem cells are filtered out of it and stored for use by your child. This procedure takes place in the hospital or in our outpatient center, and the donor can go home the same day.

Another way to collect stem cells is called bone marrow retrieval surgery. In this method, stem cells are collected directly from the donor’s bone marrow during an inpatient procedure. The doctor inserts a needle into the donor’s hip bone and withdraws a sample of the stem cells. More than one insertion is required to harvest an adequate number of bone marrow cells. Usually, the donor can go home the same day.

These stem cells are given to your child through an IV infusion in the hospital. After the infusion, your child’s doctors watch for signs that the donated stem cells are making new, healthy blood cells in your child’s body.

Recovery from Stem Cell Transplantation

After a stem cell transplant, your child stays in the hospital in isolation to protect him or her from infection, often for several weeks. Usually, IV antibiotics are given to prevent or treat infections. A child may need periodic IV red blood cell and platelet transfusions until the donor’s stem cells start successfully dividing and multiplying in your child’s body.

Full recovery from a stem cell transplant can take several months. Medications are prescribed to prevent graft-versus-host disease, which occurs when the donated cells attack healthy tissues in the child’s body. 

Side effects of stem cell transplantation may include infection and irritation of the mouth and intestines or other organs. Long-term side effects may include infertility and growth delay.

Our doctors work quickly to manage any side effects your child may experience. Our specialists also monitor your child’s blood cell levels, looking to prevent side effects from worsening. Your child’s doctor can talk with you about all potential side effects and work with you to treat them, as needed.