Acute Myeloid Leukemia (AML)

Leukemia is the general term for some different types of blood cancer. AML is one of four main types of leukemia.

About Marrow, Blood and Blood Cells

The information on this page about normal blood and marrow may help you understand the AML information in the rest of this Guide.

Marrow is the spongy center inside of bones where blood cells are made.

Blood cells are made in the marrow. They begin as stem cells. Stem cells become red cells, white cells and platelets in the marrow. Then the red cells, white cells and platelets enter the blood.

Platelets form plugs that help stop bleeding at the site of an injury.

Red cells carry oxygen around the body. When the number of red cells is below normal, the condition is called anemia. Anemia may make you feel tired or short of breath. It may make the skin look pale.

White cells fight infection in the body. There are two major types of white cells: germ-eating cells (neutrophils and monocytes) and infection-fighting “lymphocytes” (B cells, T cells and natural killer [NK] cells).

Plasma is the liquid part of the blood. It is mostly water. It also has some vitamins, minerals, proteins, hormones and other natural chemicals in it.

About AML

AML is a type of cancer that begins in the bone marrow. The chance of getting AML increases with age. But a person can get AML at any age. About 8 in 10 adults with acute leukemia have AML. About 1 in 6 children with leukemia has AML.


Figure 1: The cells in panels A and B are shown much larger than actual size.The cells are also stained with a special dye so that they can be seen more clearly.

Panel A shows different types of normal marrow cells seen through a microscope. These normal cells are in various stages of development.

Panel B shows AML blast cells seen through a microscope. These cells have stopped developing.

Causes and Risk Factors of AML. AML starts with a change to a single cell in the bone marrow. Doctors do not know what causes most cases of AML. There is no way to prevent AML. You can’t catch AML from someone else.

Certain things may increase the risk of getting AML, such as

  • Some types of chemotherapy
  • Radiation used to treat cancer
  • Down syndrome
  • Tobacco smoke
  • Repeated contact with the chemical benzene.

Benzene can harm normal marrow cells. The majority of benzene in the environment comes from petroleum products, however, half of the personal exposure is from cigarette smoke. The average smoker is exposed to about 10 times the daily intake of benzene compared to nonsmokers. Benzene is also found in some work settings. But, strict rules have led to lowered amounts of benzene in the workplace.

Most people with the risks listed on page 8 do not get AML.

Signs and Symptoms. Many of the signs and symptoms of AML are also caused by other types of illness. Most people with these signs and symptoms do not have AML.

A sign is a change in the body that the doctor sees in an exam or a test result.

A symptom is a change in the body that the patient can see or feel.

Some Signs and Symptoms of AML

Not feeling well

Having fewer normal bone marrow cells may cause aches in the legs, arms or hips. Patients may have a mild fever, enlarged lymph nodes or swollen gums.

Tiring more easily, shortness of breath, pale skin color

Having fewer healthy red cells may lower energy levels. It may also make people feel short of breath while doing day-to-day activities. Some people with fewer red cells may have a pale skin color.

Weight loss

Some people with AML lose weight because they eat less and/or they use more energy.

Black-and-blue marks or pinhead-sized red spots

A low platelet count may cause patients to bruise more easily or to have tiny red spots called petechiae on the skin.

Bleeding for a long time from minor cuts

A low platelet count may lead to a longer bleeding time or slower healing of cuts.


It is important for patients to receive the right diagnosis. AML is diagnosed with blood and bone marrow tests.

Blood Cell Counts. The doctor orders a test called a CBC (complete blood count) to count the numbers of red cells, white cells and platelets. Usually, patients with AML have lower-than-expected numbers of red blood cells and platelets.

Blood Cell Examination. The cells are stained (dyed) and looked at through a microscope. This test is also called a blood smear. A person with AML usually has too many leukemic blast cells in the blood. These cells do not function like normal cells.

Bone Marrow Tests. Your doctor will also do other tests to make sure the diagnosis of AML is right. Tests called bone marrow aspiration and bone marrow biopsy are done to find out the percentage of AML cells in the marrow. A person with AML usually has at least 20 percent AML cells.

Types of AML

Knowing the patient’s AML type helps the doctor to plan treatment. Most people with AML have one of the eight AML types: MO, M1, M2, M3, M4, M5, M6 or M7. Most types of AML are treated in the same way. But patients with type M3 (acute promyelocytic leukemia, or APL for short) need a different treatment plan. More information about APL treatment is on page 21.

Flow Cytometry. Your doctor will order a test called flow cytometry to find out the type of AML you have.

Cytogenetic Analysis. Other tests are done to look for genetic changes in your AML cells. This examination of AML cells is called cytogenetic analysis. The results help your doctor to plan your treatment.


Treatment Planning

The goal of treatment for AML is to cure the disease.

  • Almost half of children with AML are cured.
  • Patients with acute promyelocytic leukemia (APL) have higher cure rates overall compared to adults with other AML types.
  • Some adults with other types of AML may be cured or have long periods of remission.
  • There has been improvement in treatment outcomes for people with all types of AML.

Some things that may affect the outcome of your AML treatment are

  • Your type of AML
  • The results of your lab tests
  •  Your age and general health
  •  Your medical history, including whether you Were treated with chemotherapy before for another type of cancer
  • Had a myelodysplastic syndrome (MDS)
  • Whether you have A serious infection at the time of diagnosis
  • AML in your central nervous system
  • AML that has not responded to treatment or has relapsed.

It is important to talk to your doctor about the results of your bone marrow examination. This test provides information about the marrow cells that is needed for treatment planning. It is also important to talk to your doctor about molecular and genetic tests.

Fast Facts About AML Treatment

  • Some patients who have AML are cured with treatment.
  • Many patients with AML need treatment as soon as possible after diagnosis.
  • An AML patient is usually treated by a hematologist/oncologist.
  • AML patients should be treated by doctors who are skilled in treating patients with AML.
  • The treatment plan for each patient is based on his or her AML test results.
  • The initial goal of treatment is usually to get the patient into remission.
  • A remission means: There is no sign of AML cells in the blood or marrow; blood cell counts are back to normal.
  • Many older patients can be treated for AML and enter remission.
  • Most patients need chemotherapy to achieve remission. At least two drugs are combined to treat patients. This is called induction therapy.
  • More treatment is needed once a remission is achieved to help prevent a relapse. This is called post-remission therapy.
  • Post-remission therapy may consist of chemotherapy, stem cell transplantation or low-dose maintenance chemotherapy.
  • Patients may have a return of AML after treatment. This is called a relapse.

About AML Treatments

Treatment for patients with AML (or relapsed AML) may include

  • Chemotherapy
  • Stem cell transplantation
  • New approaches under study (clinical trials).

A patient may receive different drugs from those described in this Guide and still be receiving proper treatment.

Speak to your doctor to find out what treatment is best for you. Our Information Specialists can help you plan questions to ask your doctor about treatment.

Induction Therapy. Induction therapy is the first part of treatment with chemotherapy. Most AML patients need to start induction chemotherapy right away. Induction therapy is done in the hospital. Patients are often in the hospital for 4 to 6 weeks for this first part of treatment.

The aims of induction therapy are to

  • Kill as many AML cells as possible
  • Get blood counts back to normal
  • Get rid of all signs of the disease for an extended period of time.

Chemotherapy and other drugs. Chemotherapy drugs and other types of drugs kill or damage cancer cells. Several types of drugs are used to kill AML cells. Each drug type works in a different way. Combining the drugs can make the treatment work better.

The first round of chemotherapy usually does not get rid of all the AML cells. Most patients will need more rounds of therapy. Usually the same drugs are used for the added rounds needed to complete induction therapy.

When there is no sign of AML, this is called a remission.

Some drugs are given by mouth. Other drugs are given through a central line, port or PICC. Medications, nutrition and blood products can all be administered via central lines or PICCs. Blood for lab tests can also be withdrawn via these lines. Central lines, ports and PICCs can stay in place for weeks or months. You can talk to your doctor about the best one for you or your child to use.

Central Lines, Ports and PICCs

Central Line

This is a thin tube that is put under the skin and into a large vein in the chest. The central line stays firmly in place. Catheter is another word for central line.


This is a small device that is used with a central line. The port is placed under the skin of the chest. After the site heals, no dressings are needed and no special home care is needed. To give medicines or nutrition or to take blood samples, the doctor or nurse puts a needle through the skin into the port. A numbing cream can be put on the skin before the port is used.

PICC or PIC Line

PICC or PIC line is short for percutaneously inserted central venous catheter. A PICC is inserted through a vein in the arm.

Some of the drugs used to treat AML are listed below. These are some of the standard drugs used and some of the drugs under study in AML clinical trials.

Table 1:Some Drugs Used to Treat AML

Drug Types

Drug Name

Anthracyclines ( Antitumor Antibiotics)

daunorubicin (Cerubidine®); doxorubicin (Adriamycin®); idarubicin (Idamycin®); mitoxantrone (Novantrone®)


cladribine (2-CdA; Leustatin®); clofarabine (Clolar®); cytarabine (cytosine arabinoside, ara-C; Cytosar-U®); fludarabine (Fludara®); hydroxyurea (Hydrea®); methotrexate; 6-mercaptopurine (Purinethol®); 6-thioguanine (Thioguanine Tabloid®)
Topoisomerase Inhibitors etoposide (VP-16; VePesid®, Etopophos®); topotecan (Hycamtin®)
DNA Damaging (Alkylating) Agents

cyclophosphamide (Cytoxan®); carboplatin (Paraplatin®); temozolomide (Temodar®)

Cell-Maturing Agents

all-trans retinoic acid (ATRA, tretinoin; Vesanoid®); arsenic trioxide (Trisenox®)

Hypomethylating Agents

azacitidine (Vidaza®); decitabine (Dacogen®)

Radiation Therapy

Sometimes radiation therapy may be used to treat a large mass of AML cells in the spine or brain called a chloroma.

Postremission Therapy. More treatment is usually needed even after an AML patient is in remission. Some AML cells may remain that are not found by common blood or marrow tests. This part of AML treatment is called postremission therapy or consolidation therapy.

Chemotherapy is part of postremission therapy for AML. Stem cell transplantation may be part of postremission therapy for some AML patients.

The treatment setting (hospital or outpatient) and the length of hospital stay depend on the postremission treatment. The length of time in the hospital also depends on any side effects of treatment. Patients are often in the hospital for 4 to 6 weeks. Some patients may need to be in the hospital longer.

Relapsed or Refractory AML

Some patients have a remission after treatment, but then AML cells return later (a relapse). Patients may have AML cells in the marrow even after treatment (refractory AML).

Patients who relapse may be treated with the same drugs as newly diagnosed patients—or different drugs may be given. Patients who have a matched donor may be given an allogeneic stem cell transplant. Information about allogeneic stem cell transplantation begins on page 19.

With refractory AML, drugs that were not used in the first round of treatment may be given. An allogeneic transplant may also be a part of treatment.

Stem Cell Transplantation

Your doctor will talk with you about whether stem cell transplantation is a treatment option for you.

Allogeneic Stem Cell Transplantation. An allogeneic transplant uses stem cells from a donor. The donor may be a brother or sister. Or, the donor can be an unrelated person with stem cells that “match” the patient’s. Stem cells may also come from a cord blood unit (the blood in the umbilical cord after a baby’s birth).

The goals of an allogeneic transplant are to:

  • Restore the body’s ability to make normal blood cells after high-dose chemotherapy
  • Cure the patient of his or her AML by killing any remaining AML cells.

Allogeneic transplants may be done in the hospital. First, the patient is given high-dose chemotherapy and/or radiation therapy. Stem cells are collected from a donor. The donor stem cells are given to the patient through an IV (intravenous) line or central line. The donor stem cells go from the patient’s blood to the marrow and help start a new supply of red cells, white cells and platelets.

Your doctor will talk with you about whether an allogeneic transplant is a treatment option for you.

Allogeneic stem cell transplantation is a high-risk procedure. Doctors are working to make allogeneic transplants safer. An allogeneic transplant may be a choice for an AML patient if

  • He or she has a type of AML that is hard to treat.
  • The expected benefits of an allogeneic transplant exceed the risks.
  • There is a stem cell donor.

The upper age limit for an allogeneic transplant depends on the treatment center. Many centers use age 60 or 65 years as the upper age limit for an allogeneic transplant.

Your doctor will talk to you about whether a reduced-intensity allogeneic stem cell transplant is a treatment option for you. The goal of a reduced-intensity transplant is to cure the patient of his or her AML. A reduced-intensity allogeneic transplant uses lower doses of chemotherapy than a standard allogeneic transplant. Some older and sicker patients may be helped by this treatment. Many centers use 70 years or older as the upper age limit for a reduced-intensity allogeneic transplant.

A reduced-intensity allogeneic transplant may be a choice for an AML patient if

  • He or she has a type of AML that is hard to treat.
  • A standard allogeneic transplant is not a choice because of the patient’s age or overall health.
  •  The expected benefits of a reduced-intensity allogeneic transplant exceed the risks.
  •   There is a stem cell donor.

Autologous Stem Cell Transplantation. An autologous transplant uses the patient’s own stem cells.

The goal of an autologous transplant is to restore the body’s ability to make normal blood cells after high-dose chemotherapy.

The patient’s own blood or marrow stem cells are stored before chemotherapy begins. They are infused back into the patient’s blood after chemotherapy ends. The stem cells are given back to the patient through an IV (intravenous) line or central line. The stem cells go from the patient’s blood to the marrow and help start a new supply of red cells, white cells and platelets.


AML Treatment in Children

There are about 3,811 new cases of leukemia each year in the United States in children younger than 15 years. About 4 out of 5 children with AML go into remission. Over half of children with AML have no signs of disease after 5 years. More than half of these children are considered cured.

Induction therapy for children with AML starts with two or three drugs. More treatment is needed after a child with AML is in remission (postremission therapy). It is given because some AML cells may remain after induction therapy. These AML cells do not show up in standard blood or marrow tests. Postremission therapy in children includes a number of chemotherapy drugs.

AML treatment is less likely to bring about a remission or cure in children

  • With very high white cell counts
  • Younger than 2 years old
  • With certain chromosome changes in their AML cells.

 Allogeneic stem cell transplantation may be used in children who are not doing well or whose AML returns after high-dose chemotherapy. Doctors will discuss the benefits and risks of allogeneic transplantation with parents and older children.

AML Treatment in Older Patients

AML is more common in older patients. At least half of patients are over 65 years old when their disease is diagnosed.

Today, remissions are possible for some older people with AML, including those who may have other serious health problems. But treatment results in adults are not as good as treatment results in children.

Some healthy older patients can be treated with the same doses of chemotherapy as younger adults. Sometimes older patients have other medical problems, such as heart disease, kidney or lung disease or diabetes. The doctor takes these other medical problems into account to decide which drugs to use and in what dosage.

The doctor will also consider

  • The type of AML
  • The patient’s physical ability to handle the treatment
  • The patient’s feelings about the treatment approach.

 Some older patients may have a reduced-intensity allogeneic stem cell transplant. See page 20 for more information.

Source:  The AML Guide, Leukemia and Lymphoma Society

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