Childhood Leukemia Overview

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Treating Leukemia in Children TOPICS

High-dose chemotherapy and stem cell transplant for childhood leukemia

Stem cell transplant (SCT) can sometimes be used for children whose chances of cure are poor with standard or even intense chemotherapy. SCT lets doctors use very high doses of chemo. The high doses of these drugs destroy the bone marrow, which keeps new blood cells from being made. This poses a threat to the child’s life. But after treatment is finished, the child gets a transplant of blood-forming stem cells to replace the bone marrow.

Allogeneic stem cell transplant

The type of SCT used for childhood leukemia is known as an allogeneic stem cell transplant. In this type of transplant, the blood-forming stem cells come from the blood or bone marrow of another person.

Usually the donor is a brother or sister, but rarely the donor is an unrelated volunteer who has the same tissue type as the child. Stored stem cells from umbilical cord blood can also be used. These stem cells come from blood drained from the umbilical cord and placenta after a baby is born and the cord is cut.

To learn about how a stem cell transplant is done, see Stem Cell Transplant (Peripheral Blood, Bone Marrow, and Cord Blood Transplants).

When SCT might be used

Acute lymphocytic (lymphoblastic) leukemia (ALL): SCT could be used for children whose ALL doesn’t respond well to the first treatment or comes back soon after going into remission. SCT may also be advised for children with some less common forms of ALL.

Acute myeloid leukemia (AML): Because AML comes back more often than ALL, many doctors advise SCT for children with AML right after they have gone into remission, especially if the child has a brother or sister who can donate stem cells. If the cancer comes back after the first round of chemo, most doctors will suggest SCT as soon as the child goes into remission again. In either case, it is important that the leukemia is in remission before the transplant. Otherwise, it 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).

Practical points

A stem cell transplant is a complex treatment that can cause life-threatening side effects. If the doctors think your child might be helped by this treatment, it’s important that it be done at a hospital where the staff has a lot of experience with the procedure.

Stem cell transplant often requires a long hospital stay and can be very expensive (often costing well over $100,000). Be sure to get a written approval from your insurer if your child is to have this treatment. Even if the transplant is covered by your insurance, your co-pays or other costs could easily amount to 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.

Side effects of SCT

Side effects from a stem cell transplant can be short-term or long-term.

Short-term side effects: Early side effects are those of high-dose chemotherapy, and can be severe. They can include:

  • Low blood cell counts (with fatigue and an increased risk of infection and bleeding)
  • Nausea and vomiting
  • Loss of appetite
  • Mouth sores
  • Diarrhea
  • Hair loss

One of the most common and serious short-term effects is the increased risk of severe infection. Antibiotics are often given to try to prevent this. Other side effects, like low red blood cell and platelet counts, may mean that the patient will need blood product transfusions or other treatments.

Long-term and late side effects: Some side effects can last a long time. Sometimes they don’t show up until months or even years after the transplant. Long-term side effects could include:

  • Graft-versus-host disease (see below)
  • Radiation damage to the lungs
  • Problems with the thyroid or other hormone-making glands
  • Problems with fertility
  • Damage to bones or problems with bone growth
  • Development of another cancer (including leukemia) years later

Graft-versus-host disease (GVHD) is a major concern of an allogeneic SCT. This happens when the donor immune system cells attacks the patient’s own cells, mainly in the skin, liver, and digestive tract.

Symptoms can include severe skin rashes with itching and severe diarrhea. The liver and lungs can also be damaged. The patient may also become tired and have aching muscles. If severe enough, the disease can be fatal. Drugs that weaken the immune system are often given to try to keep GVHD under control.

You can find out more about long-term effects in the section “Long-term effects of treatment for childhood leukemia.”

To learn more about stem cell transplants, see Stem Cell Transplant (Peripheral Blood, Bone Marrow, and Cord Blood Transplants).

Last Medical Review: 05/13/2015
Last Revised: 02/03/2016