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Stem Cell Transplantation

For some high-risk patients who are in remission, the doctor may recommend stem cell transplantation during the consolidation phase of chemotherapy.

First the patient receives intensive chemotherapy to kill the remaining leukemic cells in the patient's body. This also destroys the normal stem cells in the patient's bone marrow. After the chemotherapy, the patient receives an infusion of stem cells to replace the stem cells destroyed by the intensive therapy. These new stem cells restore healthy stem cells in the bone marrow that can form new red blood cells, white blood cells and platelets.

There are two main types of stem cell transplantation:

  • Allogeneic stem cell transplantation, using stem cells from a healthy donor
  • Autologous stem cell transplantation, using stem cells from the patient
    • This type of transplant is typically not used for treating AML. 

Research to determine which patients are most likely to benefit from stem cell transplantation after their first complete remission is evolving. Studies show that allogeneic stem cell transplantation may benefit high-risk and intermediate-risk AML patients who have an HLA-matched sibling donor.

Allogeneic Stem Cell Transplantation for AML

This is the most common type of stem cell transplantation used to treat AML. Allogeneic transplantation uses healthy blood-forming cells from an HLA-matched family member, an unrelated donor, or from umbilical cord blood. The donated stem cells restore the bone marrow’s ability to form new blood cells. Ideally, an allogeneic stem cell transplant will generate a new immune system for the patient. The immune system helps the body fight infections and other diseases. The new immune system also has the potential to recognize and attack any remaining cancer cells. The transplanted immune cells (the graft) perceive the leukemia cells in the body as foreign and destroy them. This is called the “graft-versus-leukemia” (GVL) effect.

Allogeneic stem cell transplantation, compared to other treatment approaches, is associated with a higher rate of side effects and mortality in patients. However, it may be considered for patients with higher-risk AML, based on their AML subtype and response to induction therapy. The decision to perform an allogeneic stem cell transplantation also depends on the following:

  • Your child’s age
  • Your child’s overall health
  • Availability of an HLA-matched donor

Graft Versus Host Disease

A serious risk of allogeneic and reduced-intensity allogenic stem cell transplantation is graft versus host disease (GVHD), which develops if the donor's immune cells attack your normal tissue. GVHD's effects can range from minor to life threatening.


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