Treatments for relapsed or refractory acute myelogenous leukemia

Relapsed, or recurrent, acute myelogenous leukemia (AML) means the leukemia has come back after treatment and reaching remission.

Refractory AML means the leukemia did not respond to treatment. Complete remission has not been reached because the chemotherapy drugs did not kill enough leukemia cells.

Both relapsed and refractory AML need more treatment to reach complete remission.


Chemotherapy is usually given for relapsed or refractory AML. It may include repeating cycles of the same or similar drugs that were used in induction treatment if the complete remission was longer than one year. Similar or higher doses of the drugs may be used.

A repeat course of the 7-and-3 protocol may be given. In this protocol, cytarabine (Cytosar, Ara-C) is given continuously for 7 days with an anti-tumour antibiotic given for 3 days. The anti-tumour antibiotics used in this protocol include:

  • daunorubicin (Cerubidine)
  • doxorubicin (Adriamycin)
  • idarubicin (Idamycin)
  • mitoxantrone (Novantrone)

Other types of chemotherapy that may be offered for relapsed or refractory AML are:

  • high-dose cytarabine (HDAC) alone or in combination with an anti-tumour antibiotic
  • etoposide (Vepesid, VP-16), cytarabine and mitoxantrone (Novantrone)
  • high-dose etoposide and cyclophosphamide (Cytoxan, Procytox)
  • FLAG – fludarabine (Fludara), cytarabine and granulocyte colony-stimulating factor (G-CSF)

Central nervous system treatment

The central nervous system (CNS) is the brain and spinal cord. If the leukemia has spread to the CNS, the treatment may include chemotherapy given directly into the spinal fluid (called intrathecal chemotherapy). The drug used in intrathecal chemotherapy is methotrexate or cytarabine. It is given during a lumbar puncture or through an Ommaya reservoir.

Radiation therapy is sometimes given to the brain and spinal cord along with intrathecal chemotherapy.

Stem cell transplant

In some cases, a stem cell transplant may be offered for relapsed or refractory AML. An allogeneic transplant is the preferred type of stem cell transplant. If a matched donor is not available, autologous stem cell transplant may be an option.

A stem cell transplant may be offered to people who relapse soon after they reach a first complete remission or a second remission. People who relapse after stem cell transplant may be offered other treatments, including infusion of lymphocytes from their stem cell donor (called donor leukocyte infusion, or DLI).

Radiation therapy

Radiation therapy may be given as part of the conditioning treatment before stem cell transplant. It may also be used to treat AML that has spread to the central nervous system (CNS).

Supportive therapy

Supportive therapy is important during every phase of treatment for AML. It is used to treat the complications that usually happen with treatments for AML and the disease itself.

Supportive therapies given during treatment for relapsed or refractory AML may include:

  • antibiotics, antivirals or antifungals to prevent or fight infections.
  • growth factors to help the bone marrow recover from chemotherapy (chemotherapy can affect the bone marrow so it doesn’t make enough healthy blood cells, which can increase the risk for infection)
  • transfusions of red blood cells, platelets, fresh frozen plasma and cryoprecipitate (a product that replaces clotting factors) as needed.
  • drugs to bring down high levels of some chemicals in the blood that increase when many cancer cells die at the beginning of treatment (called tumour lysis syndrome)
  • leukapheresis to remove large numbers of white blood cells

Clinical trials

You may be asked if you want to join a clinical trial for AML. Find out more about clinical trials.

lumbar puncture

A procedure in which a needle is inserted into the lower part (lumbar region) of the spinal column.

Doctors may use lumbar puncture to give drugs or to remove cerebrospinal fluid (CSF) for examination under a microscope or to lower spinal fluid pressure.

Also called spinal tap.

Ommaya reservoir

A device surgically implanted beneath the scalp that is used to deliver chemotherapy drugs directly into the cerebrospinal fluid (CSF) around the brain and spinal cord.


A type of white blood cell that fights viruses, bacteria, foreign substances or abnormal cells (including cancer cells).

The 3 types of lymphocytes are B cells, T cells and natural killer (NK) cells.

growth factor

A substance that regulates the growth, division and survival of cells.

Growth factors are found naturally in the body. They can also be made in a lab.

Growth factors are used in biological therapy.


A procedure that uses a special machine (pheresis machine) to separate and collect specific white blood cells from withdrawn blood. The remaining blood is then returned to the body.

Leukapheresis is used to lower a very high white blood cell count in people with cancer (leukemia) or to remove white blood cells for transfusion.