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Stem Cell Transplantation

For some patients who are in remission and can tolerate intensive chemotherapy, the doctor may recommend stem cell transplantation during the consolidation phase of chemotherapy.

First the patient receives intensive chemotherapy, with or without radiation, 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.

The two main types of stem cell transplantation are 

  • Allogeneic stem cell transplantation, using stem cells from a matched or partially matched healthy donor
  • Autologous stem cell transplantation, using stem cells from the patient collected before chemotherapy 

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 patients with high-risk and intermediate-risk AML up to age 75 years and who have an HLA-matched donor.. 

Allogeneic Stem Cell Transplantation

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 cytogenetic and molecular test results and other prognostic factors. The decision to perform an allogeneic transplant also depends on other factors, including

  • Age
  • Physical fitness
  • Comorbidities (other medical conditions)
  • Social support (from family members, caregivers, friends, etc)
  • Patient’s understanding of the potential benefits and risks

Reduced-Intensity Allogeneic Stem Cell Transplantation

This type of transplantation may be a treatment option for older patients who cannot tolerate the high doses of chemotherapy used in preparation for a standard allogeneic stem cell transplant. The conditioning therapy used for a reduced-intensity transplant is of lower intensity than that for a standard stem cell transplant. It does not completely destroy the patient’s immune system or treat the AML as intensively. The goal is to have the donor stem cells become established in the patient’s bone marrow and produce white blood cells that will attack the patient’s remaining cancer cells.

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.

Autologous Stem Cell Transplantation

This is a procedure in which bone marrow is removed from a patient after achieving a remission during induction therapy, and then frozen and stored. The cells are returned to the patient’s body after receiving intensive chemotherapy.

Autologous transplantation is sometimes used for patients who do not have an HLA-matched donor. Autologous transplants are usually easier for patients to tolerate than allogeneic transplants. This is because patients receive their own stem cells, so the risk of some complications, such as graft-versus-host disease, is lower.

Autologous transplants are used less frequently than allogeneic transplants for AML patients mainly because of the lack of a graft-versus-leukemia effect and the risk of returning some leukemia cells back to the patient.


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