Oncologist-approved cancer information from the American Society of Clinical Oncology

Leukemia - Acute Myeloid - AML


Last Updated: February 06, 2012

This section has been reviewed and approved by the Cancer.Net Editorial Board,  11/10

Overview

Leukemia is a cancer of the blood. Leukemia begins when normal blood cells change and grow uncontrollably. Acute myeloid leukemia (AML) is a disorder of the process that normally produces neutrophils, a type of white blood cell. AML may sometimes be called acute myelogenous leukemia, acute myelocytic leukemia, or acute nonlymphocytic leukemia. Unlike chronic leukemia, acute leukemia develops quickly and generally needs immediate treatment. AML occurs in people of all ages but is most common in adults older than 65.

About neutrophils

Neutrophils fight infections caused by bacteria. Mature neutrophils develop from immature (undeveloped) white blood cells in a process called differentiation. The production of mature neutrophils usually is highly regulated. For example, the body rapidly makes more neutrophils during an infection and returns to a regular level of production when the infection is controlled.

About AML

In AML, acquired mutations (damage to the genetic material or DNA) in the blood-forming cells cause problems with the normal process of differentiation, causing many immature cells called myeloblasts or blasts. Blasts do not act like fully developed, healthy blood cells. The large number of blasts also reduces the production of healthy red blood cells and platelets. Therefore, people with AML are usually anemic (because they do not have enough red blood cells), more likely to get infections (because they do not have enough mature neutrophils), and bruise or bleed easily (because of a low level of platelets).

AML is usually found in the blood and bone marrow (the spongy, red tissue in the inner part of the large bones), but it can sometimes also invade other parts of the body, such as the brain, skin, and gums. Occasionally, AML cells can form a solid tumor called a myeloid sarcoma or chloroma that may be located nearly anywhere in the body.

This section is about AML in adults. Read about childhood AML.

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Find out more about basic cancer terms used in this section.

Statistics

This year, an estimated 13,780 people of all ages (7,350 men and boys and 6,430 women and girls) in the United States will be diagnosed with AML. AML is the second most common type of leukemia diagnosed in adults. An estimated 10,200 deaths (5,790 men and boys and 4,410 women and girls) from AML will occur this year.

The five-year survival rate (percentage of people who survive at least five years after the cancer is detected, excluding those who die from other diseases) of people with AML is 24%. However, it is important to note that survival depends on several factors, including biologic features of the disease and, in particular, a patient’s age (see Subtypes for more information). Although AML is a serious disease, it is treatable and often curable with chemotherapy.

Cancer survival statistics should be interpreted with caution. These estimates are based on data from thousands of people with this type of cancer in the United States each year, but the actual risk for a particular individual may differ. It is not possible to tell a person how long he or she will live with AML. Because the survival statistics are measured in five-year intervals, they may not represent advances made in the treatment or diagnosis of this cancer. Learn more about understanding statistics.

Statistics adapted from the American Cancer Society's publication, Cancer Facts and Figures 2012.

Medical Illustrations

Adult Marrow Anatomy

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Blood Anatomy

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Risk Factors

A risk factor is anything that increases a person’s chance of developing cancer. Although risk factors can influence the development of cancer, most do not directly cause cancer. Some people with several risk factors never develop cancer, while others with no known risk factors do. However, knowing your risk factors and communicating them to your doctor may help you make more informed lifestyle and health care choices.

Although the cause of AML is not known, several factors are associated with an increased risk of the disease. The following factors may raise a person’s risk of developing AML:

Age. AML is more common in older adults, and most people are around age 65 when diagnosed.

Smoking. The risk of AML has been linked to exposure to tobacco smoke, probably in association with other causes.

Genetic disorders. AML occurs more frequently in people with inherited disorders such as Down syndrome, ataxia telangiectasia, Li-Fraumeni syndrome, Klinefelter syndrome, Fanconi anemia, Wiskott-Aldrich syndrome, and Bloom syndrome.

High doses of radiation. People who have been exposed to high levels of radiation, such as long-term survivors of atomic bombs, may be more likely to develop AML.

Previous cancer treatment. People who have received chemotherapy and/or radiation therapy for other types of cancer, such as breast cancer, ovarian cancer, and lymphoma, have a higher risk of AML in the years following such treatment.

Chemicals. Prolonged contact with products containing the chemical benzene raises the risk of AML. However, exposure to industrial solvents and hair dyes has not been proven to increase a person’s risk of AML.

Symptoms

People with AML may experience the following symptoms or signs. Sometimes, people with AML do not show any of these symptoms. Or, these symptoms may be caused by a medical condition that is not cancer. If you are concerned about a symptom or sign on this list, please talk with your doctor.

  • Fatigue

  • Weakness

  • Easy bruising or bleeding

  • Weight loss

  • Fever

  • Bone or abdominal pain

  • Difficulty breathing; dyspnea (shortness of breath)

  • Frequent infections

  • Swollen lymph nodes (glands)

  • Swollen or bleeding gums

Diagnosis

Doctors use many tests to diagnose leukemia and determine the subtype (see Subtypes). Some tests may also determine which treatments may be the most effective. For most types of cancer, a biopsy is the only way to make a definitive diagnosis of cancer. If a biopsy is not possible, the doctor may suggest other tests that will help make a diagnosis. Imaging tests may be used to find out whether the cancer has metastasized. Your doctor may consider these factors when choosing a diagnostic test:

  • Age and medical condition

  • The type of cancer suspected

  • Severity of symptoms

  • Previous test results

The following tests may be used to diagnose AML:

Blood tests. To diagnose AML, a doctor will do blood tests to count the number of white blood cells and to see if they look abnormal under the microscope. Special tests called flow cytometry (immunophenotyping) and cytochemistry are sometimes used to distinguish AML from other types of leukemia and to determine the exact subtype of AML. Chromosome studies called cytogenetics are used to identify genetic changes in the AML blasts, and these changes often help doctors decide on the best treatment plan.

Genetic testing. Several genetic mutations in the AML cells have been found that can help determine prognosis (chance of recovery), and these molecular analyses are now being done more often during diagnosis.

Bone marrow biopsy. In a bone marrow biopsy, a doctor takes a sample of marrow, usually from the back of the hipbone, with a needle. The patient is given medication to numb the area beforehand. The cells from the marrow, along with the cells from the blood, are analyzed by a pathologist (a doctor who specializes in interpreting laboratory tests and evaluating cells, tissues, and organs to diagnose disease).

Lumbar puncture (spinal tap). A lumbar puncture is a procedure in which a doctor takes a sample of cerebral spinal fluid (CSF) to look for cancer cells, blood, or tumor markers (substances found in higher than normal amounts in the blood, urine, or body tissues of people with certain types of cancer). CSF is the fluid that flows around the brain and the spinal cord. Doctors generally give an anesthetic to numb the lower back before the procedure. The CSF is then examined under the microscope for the presence of AML cells.

Imaging tests. A computed tomography (CT or CAT) scan (test that creates a three-dimensional picture of the inside of the body) or magnetic resonance imaging (MRI, test that uses magnetic fields, not x-rays, to produce detailed images of the body) may be used to learn more about the cause of symptoms or to help diagnose infections in patients with AML. They are not regularly used for staging of AML since the disease has often spread throughout the bone marrow and before diagnosis. These tests may not be done for every patient.

Learn more about what to expect when having common tests, procedures, and scans.

Find out more about common terms used during a diagnosis of cancer.

Subtypes

There are different subtypes of AML. Although all subtypes cause decreases in normal blood counts, different types of AML are associated with specific symptoms and problems and can behave differently after treatment.

Morphology

AML is initially defined according to its morphology (what the cancerous cells look like under the microscope). The classification of AML is named according to the type of normal, immature white blood cell it most closely resembles.

Most patients with AML are classified as having myeloblastic leukemia, which means the cancer is in the cells that normally produce neutrophils. Other patients have a type of AML called monocytic. In monocytic leukemia, the cells resemble other white blood cells called monocytes. Mixtures of myeloblastic and monocytic leukemia can occur.

Sometimes the AML seems to come from cells that produce red blood cells (erythroid) or platelets (megakaryocytic). Promyelocytic leukemia (APL) is a unique subtype of AML where the cancer cell stops maturing when the cell is at a stage called the promyelocyte or progranulocyte.

Flow cytometry is a test that can detect the presence of particular proteins on the surface of abnormal cells and is sometimes used to find the difference among these subtypes.

The classification system from the World Health Organization (WHO) includes:

  • AML with recurrent genetic abnormalities (that is, with specific chromosomal changes)

  • AML with multilineage dysplasia (abnormalities in the appearance of other blood cells)

  • AML with myelodysplastic syndrome, related to therapy

  • AML that is not otherwise categorized

The French-American-British (FAB) classification is an older system for describing AML morphology, but it is still commonly used and is listed below for reference.

M0: Myeloblastic without differentiation
M1: Myeloblastic without maturation
M2: Myeloblastic with maturation
M3: Promyelocytic
M4: Myelomonocytic
M5a: Monocytic without differentiation (monoblastic)
M5b: Monocytic with differentiation
M6: Erythroleukemic
M7: Megakaryocytic

Cytogenetics

AML is also classified according to the cytogenetic (chromosome) changes found in the leukemia cells. Sometimes the doctor can find these changes by looking at the chromosomes in dividing cells under the microscope, while other changes can be found only with molecular tests that can find very small changes in the DNA.

Certain chromosomal changes are closely matched with particular morphologic subtypes of AML. More importantly, the chromosomal changes help doctors determine the best treatment options because these changes can usually predict how effective treatment will be. Chromosomal changes are commonly grouped according to the likelihood that the subtype will respond to treatment. (Note: all chromosomes are numbered from one to 22; sex chromosomes are called “X” or “Y.” The letters “p” and “q” refer to the “arms” or specific areas of the chromosome.)

Some of the most common chromosomal changes are grouped as follows:

Favorable. Chromosomal changes associated with successful treatment include abnormalities of chromosome 16 at bands p13 and q22, a translocation (exchange of genetic material) between chromosomes 8 and 21, and a translocation between chromosomes 15 and 17 (found in progranulocytic leukemia).

Intermediate. Findings suggestive of a less favorable prognosis include normal chromosomes (no abnormality detected) and a translocation between chromosomes 9 and 11.

Unfavorable. Examples of chromosomal changes that are associated with less successful treatment and low cure rates include extra copies of chromosomes 8 or 13, deletion of all or part of chromosomes 5 or 7, complex abnormalities involving many chromosomes, and abnormalities of chromosome 3 at band q26.

In general, the more favorable changes occur in younger patients, while the unfavorable changes are more common in patients older than 60. In each of these groups, there is still a wide range of how the cancer responds to treatment, with average expected long-term rates of successful AML treatment ranging from 50% to 60% in the favorable group to less than 10% in the unfavorable group. How well treatment works also depends on other factors, including the patient’s age and the white blood cell count. It is not possible to predict exactly the likelihood of successful treatment for an individual person with AML.

Molecular Genetics

Mutations that are too small to be seen with a microscope and cannot be found with cytogenetic tests have been found using tests called molecular assays. For example, patients with mutations in the NPM1 gene have a better prognosis (chance of recovery), and chemotherapy (see Treatment) is not as effective for patients with mutations in the FLT3 gene. Therefore, finding these changes during diagnosis helps determine a patient’s treatment options.

Recurrent. Recurrent AML is cancer that comes back after treatment.

Treatment

The treatment of AML depends on the subtype, morphology, and cytogenetics of AML (see Subtypes) and the patient’s overall health. In many cases, a team of doctors will work with the patient to determine the best treatment plan.

The most successful treatment for AML depends on the results of the first treatment, so it is important for patients to have the initial treatment at a center experienced with AML.

This section outlines treatments that are the standard of care (the best treatments available) for this specific type of cancer. Patients are also encouraged to consider clinical trials as a treatment option when making treatment plan decisions. A clinical trial is a research study to test a new treatment to prove it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options. For more information, see the Clinical Trials section.

Descriptions of the most common treatment options for AML are listed below.

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells. The drugs travel through the bloodstream to cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist a doctor who specializes in treating blood disorders. Some people may receive chemotherapy in their doctor's office or outpatient clinic; others may go to the hospital. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a specific time.

Chemotherapy is the primary treatment for AML. Chemotherapy may be given by mouth in a pill, injected into a vein, or injected into the CSF. Several drugs are used to treat AML.

Chemotherapy for AML can be divided into three phases: remission induction, post-remission consolidation, and maintenance. However, maintenance refers to the long-term use of chemotherapy for patients in remission and is not commonly used in AML.

Induction therapy. This stage of therapy refers to the initial period of treatment after the diagnosis is made. The goal of induction therapy is a complete remission (CR), which means that the blood counts have returned to normal, the leukemia is not visible when a bone marrow sample is examined under the microscope, and the signs and symptoms of AML are gone.

The combination of cytarabine (Cytosar-U, Tarabine PFS) given over seven days and an anthracycline drug, such as daunorubicin (Cerubidine, Rubidomycin) or idarubicin (Idamycin), given for three days is used most often. In addition to killing leukemia cells, these drugs also destroy healthy cells, increasing the risk of infection and bleeding (see below). Most patients require hospitalization for three to five weeks during induction therapy. Sometimes, two courses of therapy are needed to achieve a CR. Approximately 75% of younger adults with AML and about 50% of patients older than 50 achieve a CR after treatment.

Some older adults may not be able to have induction therapy with the standard drugs, and the drugs decitabine (Dacogen) and clofarabine (Clolar, Clofarex) may be used instead.

Consolidation or intensification therapy. This stage of chemotherapy refers to the use of a variety of different drugs that are designed to kill AML cells that remain after successful induction therapy. Whether the AML will recur if no further treatment is given after a CR is not yet known. Younger adults in remission are commonly given two to four courses of high-dose cytarabine at monthly intervals, while several different regimens are used for older patients. Although chemotherapy is usually given in the hospital, most of the recovery time is spent as an outpatient.

For some patients, stem cell transplantation (see below) is recommended instead of consolidation therapy.

Learn more about chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Acute promyelocytic leukemia (APL) treatment

The treatment of the APL subtype of AML is somewhat different. This subtype is very sensitive to the effects of all-trans retinoic acid (ATRA), a drug that is similar to vitamin A and is given by mouth. People with the APL subtype who receive a combination of ATRA and chemotherapy (see above) with idarubicin or daunorubicin are very likely to have a CR.

Bleeding is a common symptom of APL, and patients with this subtype often need many platelet and blood transfusions during initial treatment. Compared with other subtypes of AML where maintenance therapy is not used, patients with APL benefit from the long-term (one to two years) use of ATRA. Arsenic trioxide (Trisenox) is effective when given intravenously (through a vein) for the treatment of APL that has come back after treatment.

Side effects of chemotherapy

Chemotherapy for AML attacks rapidly dividing cells, including those in normal tissues, such as the hair, lining of the mouth, intestines, and bone marrow. People with AML receiving chemotherapy may lose their hair, develop mouth sores, or have nausea and vomiting. Hair will regrow after treatment is finished, and effective drugs help prevent and control nausea and vomiting.

Patients are encouraged to talk with their doctors about short-term and long-term side effects before treatment begins.

Because of the effect on the bone marrow, chemotherapy used for AML will lower the body’s resistance to infection for a short time, leading to increased bruising and bleeding and fatigue. People with AML will receive antibiotics to prevent and treat infections and will need transfusions of red blood cells and platelets throughout chemotherapy. Chemotherapy may also affect the patient’s fertility (ability to have a child in the future).

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation therapy is called external-beam radiation therapy, which is radiation given from a machine outside the body. A radiation therapy regimen usually consists of a specific number of treatments given over a specific time. Because AML is found throughout in the blood throughout the body, radiation therapy is generally used only when the cancer has spread to the brain or to shrink a chloroma (a localized mass of tissue).

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about radiation therapy.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because blood stem cells are typically what is being transplanted, not the actual bone marrow tissue.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO).

In an ALLO transplant, stem cells are obtained from a donor whose tissue matches the patient’s on a genetic level; this testing is called HLA-typing. Most often, a patient’s brother or sister serves as the donor, although unrelated donors can serve as the donor too. Millions of people worldwide have volunteered to donate stem cells for patients who do not have matched family members; your health care team will search a computer registry to look for a match. In addition, stem cells derived from umbilical cord blood are sometimes considered if family donors are not available. Often, umbilical cord blood from two donors is needed for larger adults.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed, usually after the high-dose treatment (explained below) is completed.

In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and have replacement blood stem cells create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system so that, in an ALLO transplant, the donor cells are not rejected by the body. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells. Sometimes, ALLO transplants can also be performed after giving lower doses of chemotherapy and/or radiation therapy that are still sufficient to suppress the immune system and allow growth of the donor cells. (These transplants, sometimes termed “mini-transplants” or “reduced intensity transplants” have less immediate side effects, allowing the procedure to be used for older patients.)

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and patient’s age and general health.

For both ALLO and AUTO transplant types, the replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions.

In an ALLO transplant, another major risk is that the donor’s cells will recognize the patient’s body as foreign, causing graft-versus-host disease (GVHD). GVHD may be a serious complication of allogeneic transplants and can be fatal. Other side effects may include liver problems, diarrhea, infections, and rashes. However, GVHD can also be a benefit, in that the donor cells can recognize the cancer cells as foreign and destroy these cells, a mechanism that is one of the major reasons why ALLO transplantation generally works so well over the long term. The risk of GVHD can be reduced with exact HLA-type matching and the use of preventive drugs.

In an AUTO transplant, there is little risk of GVHD because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous. Learn more about bone marrow and stem cell transplantation.

Recurrent/refractory AML

If leukemia is still present after initial treatment, the disease is called refractory AML. Patients with refractory disease may be offered new drugs being tested in clinical trials. Patients should also be considered for an ALLO stem cell transplant as part of their treatment program.

The treatment for recurrent AML often depends on the length of the initial remission. If AML comes back after a long remission, it may respond again to the original treatment. If the remission was short, then other drugs are used, often in the form of new drugs being tested in clinical trials. An ALLO stem cell transplant is often offered to patients whose leukemia has come back after initial treatment. However, the best treatment after a recurrence is not yet known, and many drugs and other approaches are being evaluated in clinical trials.

Find out more about common terms used during cancer treatment.

Clinical Trials Resources

Doctors and scientists are always looking for better ways to treat patients with AML. A clinical trial is a way to test a new treatment to prove that it is safe, effective, and possibly better than a standard treatment. The clinical trial may be evaluating a new drug, a new combination of existing treatments, a new approach to radiation therapy or surgery, or a new method of treatment or prevention. Patients who participate in clinical trials are among the first to receive new treatments, such as new chemotherapy before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.

Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that finding new drugs and other therapies is the only way to make progress in treating AML. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with AML.

Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill”. The use of placebos in cancer clinical trials is rare. When a placebo is used in a study, it is done with the full knowledge of the participants. Find out more about placebos in cancer clinical trials.

To join a clinical trial, patients must participate in a process known as informed consent . During informed consent, the doctor should list all of the patient’s options, so the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment. Learn more about clinical trials, including patient safety, phases of a clinical trial, deciding to participate in a clinical trial, questions to ask the research team, and links to find cancer clinical trials.

For specific topics being studied for AML, learn more in the Current Research section.

Side Effects

Cancer and its treatment can cause a variety of side effects. However, doctors have made major strides in recent years in reducing pain, nausea and vomiting, and other physical side effects of cancer treatments. Many treatments used today are less intensive but as effective as treatments used in the past. Doctors also have many ways to provide relief to patients when such side effects do occur.

Fear of treatment side effects is common after a diagnosis of cancer, but it may be helpful to know that preventing and controlling side effects is a major focus of your health care team. Before treatment begins, talk with your doctor about possible side effects of the specific treatments you will be receiving. The specific side effects that can occur depend on a variety of factors, including the type of cancer, its location, the individual treatment plan (including the length and dosage of treatment), and the person’s overall health.

Ask your doctor which side effects are most likely to happen (and which are not), when side effects are likely to occur, and how they will be addressed by the health care team if they do happen. Also, be sure to communicate with the doctor about side effects you experience during and after treatment. Learn more about the most common side effects of cancer and different treatments, along with ways to prevent or control them.

In addition to physical side effects, you may experience psychosocial (emotional and social) effects as well. Learn more about the importance of addressing such needs, including concerns about managing the cost of your cancer care.

Learn more about late effects or long-term side effects by reading the After Treatment section or talking with your doctor.

After Treatment

After treatment for AML ends, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your recovery for the coming months and years.

People in remission should receive regular follow-up examinations for several years to detect evidence of recurrence or late effects (side effects that occur years after treatment) of chemotherapy.

ASCO offers cancer treatment summary forms to help keep track of the cancer treatment you received and develop a survivorship care plan once treatment is completed.

People recovering from AML are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, not smoking, eating a balanced diet, and having recommended cancer screening tests. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level. Learn more about healthy living after cancer.

Find out more about common terms used after cancer treatment is complete.

Current Research

AML research is ongoing. The following advances may still be under investigation in clinical trials and may not be approved or available at this time. Most cancer centers are actively involved in clinical trials aimed at increasing the rate of cure from AML. Always discuss all diagnostic and treatment options with your doctor.

Understanding AML biology. Research designed to learn more about the biology of AML is ongoing to improve its treatment, particularly for older patients.

New drugs and treatment regimens. Researchers are looking at the use of existing drugs given in different doses and schedules, as well as new drugs. Specific research includes the use of drugs called hypomethylating therapy, such as azacitidine (Mylosar, Vidaza), and the new drug laromustine (Cloretazine, Onrigin).

Targeted therapy. Targeted therapy is a treatment that targets specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. Researchers are studying ways to block the products of specific mutations found in AML cells. For example, about 30% of patients with AML have mutations in a gene that makes a protein called FLT3, which can increase the growth of AML cells. Drugs that block FLT3 are being tested in clinical trials. Other targeted therapies are being researched that could block the proteins in AML cells that cause resistance to chemotherapy (keeps the chemotherapy from working). Specific targeted therapy drugs being researched include gemtuzumab ozogamicin (Mylotarg), sorafenib (Nexavar), and tipifarnib (Zarnestra).

Immunotherapy. Immunotherapy (also called biologic therapy) is designed to boost the body’s natural defenses to fight the cancer. It uses materials either made by the body or in a laboratory to bolster, target, or restore immune system function. Researchers are specifically looking at the use of antibodies directed against the AML cells.

Stem cell/bone marrow transplantation. Different ways to make stem cell transplantation (see Treatment) safer, easier, and more effective are also being studied.

Looking for More about Current Research?

If you would like additional information about the latest areas of research regarding leukemia, explore these related items:

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Questions to Ask the Doctor

Regular communication with your doctor is important in making informed decisions about your health care. Consider asking the following questions of your doctor:

  • What is my diagnosis? What does it mean?

  • Can you explain my pathology report (laboratory test results) to me?

  • Do I need to start treatment right away?

  • What subtype of AML do I have?

  • Can you recommend a leukemia specialist?

  • Where is the best place for me to be treated?

  • What are my options for treatment?

  • What clinical trials are open to me?

  • What treatment do you recommend? Why?

  • How likely is it that my AML will go into remission?

  • Who will be part of my health care team, and what does each member do?

  • Who will be coordinating my overall treatment and follow-up care?

  • What are the possible side effects of this treatment, both in the short term and the long term?

  • Will this treatment affect my fertility? If so, can you recommend a fertility specialist before treatment begins?

  • How will the treatment affect my daily life? Will I be able to work, exercise, and perform my usual activities?

  • If I’m worried about managing the costs related to my cancer care, who can help me with these concerns?

  • What follow-up tests will I need, and how often will I need them?

  • What support services are available to me? To my family?

Patient Information Resources

In addition to Cancer.Net, there are other sources of information about this type of cancer available online. Cancer.Net maintains a list of national, not-for-profit organizations that may be helpful in finding additional information, services, and support. As always, be sure to talk with your doctor about questions you may have about information you find about this disease.

View organizations that offer information on this specific type of cancer.