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Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a type of cancer that begins in your bone marrow, where all blood cells are formed. The disease is called “acute” because of its aggressive nature, and “myeloid” because it affects the myeloid cells, which develop into red blood cells, white blood cells or platelets. You may have also heard AML referred to as acute myelocytic leukemia, acute myelogenous leukemia, acute granulocytic leukemia or acute non-lymphocytic leukemia, as the disease has several names.

There are many effective treatments available for AML, including chemotherapy, targeted therapy, drug therapy and bone marrow transplants. In addition, there is ongoing research leading to promising new therapies. This has led to the ability to treat specific subtypes of AML with tailored treatments. Researchers are also on the path to discovering how different gene mutations lead to specific forms of the disease. There are many innovative treatment options for acute myeloid leukemia, with more on the way from dedicated doctors, experts and researchers.

What Is Acute Myeloid Leukemia (AML)?

Acute myeloid leukemia, or AML for short, is a type of cancer that affects the blood and bone marrow. This disease is called “acute” because of its aggressive nature, and “myeloid” because it affects the myeloid cells, which develop into red blood cells, white blood cells or platelets.

AML is known by several other names, including acute myelogenous leukemia, acute myelocytic leukemia, acute granulocytic leukemia and acute non-lymphocytic leukemia.

AML Symptoms 

The symptoms of AML often include one or more of the following:

What Are the Symptoms of AML?

What Are the Symptoms of AML?

  • weight loss
  • fever
  • fatigue
  • bone pain
  • unusual bleeding (such as regular nosebleeds or bleeding gums)

Because the disease lowers the number of healthy red blood cells in your body, it tends to create anemia in some patients, which encapsulates some of the symptoms listed above. Anemia can also cause:

  • headaches
  • pale skin
  • difficulty breathing
  • feeling dizzy

As white blood cell counts are lowered, your body has difficulty fighting off other diseases and frequent infections may occur. And, in addition to the aforementioned frequent bleeding, a loss of healthy platelets in your blood can also cause bruising and heavy menstrual bleeding (in women).

What Causes AML?

The exact cause for AML is oftentimes unknown. In some, it may be due to familial AML predisposition syndromes. In others, it may be secondary to treatment for another cancer (either chemotherapy or radiotherapy), or it may evolve from another hematological cancer like myeloproliferative neoplasms or myelodysplastic syndrome. Chromosomal or gene abnormalities that are acquired during life may affect the normal functioning of bone marrow to produce immature cells called blasts that cause AML. 

AML Risk Factors

There are several known risk factors for developing AML.


While the reasons for this are not yet clear, men are more likely than women to develop AML.


The risk of developing AML increases with age. Most people diagnosed with AML are age 65 or older.


According to the American Cancer Society, smoking is the only lifestyle factor associated with an increased risk for AML.

Environmental Exposure

Occupational exposure to certain chemicals such as benzene and potentially formaldehyde may increase the risk of AML.

Previous Chemotherapy Treatment

Certain chemotherapy drugs known as alkylating agents and topoisomerase II inhibitors have been known to increase your chances of developing AML.

Examples of alkylating agents include cyclophosphamide, mechlorethamine, procarbazine, chlorambucil, melphalan, busulfan, carmustine, cisplatin and carboplatin.

Examples of topoisomerase II inhibitors include etoposide, teniposide, mitoxantrone, epirubicin and doxorubicin.

Exposure to Radiation

This includes having been exposed to a nuclear incident as well as previous radiation treatments for cancer.

Genetic Syndromes

If you have one of a number of genetic diseases, your chances of getting AML may be higher.

These include Fanconi anemia, Bloom syndrome, Ataxia-telangiectasia, Diamond-Blackfan anemia, Down syndrome, Shwachman-Diamond syndrome, Li-Fraumeni syndrome, neurofibromatosis type 1, severe congenital neutropenia (Kostmann syndrome) and trisomy 8.

Family History

If someone in your immediate family has been diagnosed with AML, your risk is higher.

AML Diagnosis

If you develop AML symptoms, your doctor will likely recommend one or more of these tests to confirm or rule out the disease.

Here is a list of what you might experience.

Physical Exam

Your doctor will likely start with a physical exam, which usually includes questions about your family medical history, pre-existing conditions and overall health.

Blood Tests

Patients with AML have elevated white blood cell counts and lower than normal numbers of red blood cells and platelets. Blast cells — immature cells circulating in the blood that are normally in bone marrow — may also be present. So after a physical exam, your doctor is likely to order a complete blood count (CBC) and flow cytometry.

Bone Marrow Biopsy

While blood tests may raise the suspicion for leukemia, bone marrow biopsy is the definitive method to diagnose AML. There are two parts to the procedure: bone marrow aspiration and biopsy. The procedure itself is an office procedure lasting for 15-20 minutes. A local anesthetic is injected to numb the skin and covering of the bone called the periosteum. During the biopsy procedure, a small amount of bone marrow tissue will be withdrawn with a needle (aspiration) and a marrow core (biopsy) will be taken from the hip bone. The sample will then be examined under the microscope to confirm the diagnosis of AML.

Spinal Tap

In some cases, your doctor may want to check for leukemia cells in the fluid around your spinal cord. This is done by inserting a small needle between two vertebrae in your lower back and removing a sample of fluid from your spinal canal. As with a bone marrow biopsy, a local anesthetic is commonly used to numb the area and make you more comfortable.

If you’ve been diagnosed with AML, your doctor will likely recommend additional tests to diagnose and determine the presence of genetic mutations. These follow-up tests are important because knowing your AML subtype can help you and your medical team decide which treatment is right for you. 

I’ve Been Diagnosed with Acute Myeloid Leukemia, Now What?

It’s human nature to be scared after receiving a cancer diagnosis. Speak with your doctor regarding further management. By this time, most patients diagnosed with AML will be under the care of a doctor specialized in hematology/oncology. Discuss with your doctor regarding the exact diagnosis, subtype and if there are any genetic mutations that could be targeted, prognosis and the goals of treatment. This discussion will help guide you and your family in making an informed choice about the treatment in consultation with your doctor. According to the Leukemia & Lymphoma Society, several advances in AML treatment sound promising.

AML Treatment

The “acute” in acute myeloid leukemia means it is a fast-growing cancer, so most patients diagnosed with AML will need to start treatment right away. Your treating doctor will create a plan based on your AML subtype, the presence of genetic mutations, your age and general health, whether you have received chemotherapy in the past to treat other types of cancer, potential side effects and more.

Common AML treatments include:


This treatment uses strong drugs to kill or damage cancer cells. It is the most common treatment for AML and is often used in conjunction with other therapies. The chemotherapy, or “chemo,” drug most often used to treat AML is a cytosine arabinoside (Cytarabine) combined with an anthracycline drug, such as daunomycin (Daunorubicin) or idarubicin (Idamycin).

Almost always, patients will need to stay in the hospital while receiving chemotherapy for AML, as many normal blood cells are destroyed alongside the leukemic cells. You may need blood transfusions or develop a fever because of low immunity during chemotherapy. Staying in the hospital ensures your medical team can mitigate any side effects and keep you safe during this time.

Targeted Drug Therapy

Targeted drug therapy is the use of drugs that work by attacking a specific gene mutation or a cell protein. In AML, targeted therapy is often used when there is a drug available for a particular mutation. Discuss with your doctor if you have any targetable mutations that will permit the use of such drugs. If you have a targetable mutation, drugs include FLT3 inhibitors, IDH inhibitors, gemtuzumab ozogamicin (Mylotarg), Hedgehog pathway inhibitors and BCL-2 inhibitors.

Other Drug Therapy

If you were diagnosed with acute promyelocytic leukemia (APL), you will receive drugs called all-trans-retinoic acid (ATRA) and arsenic trioxide. They are differentiation agents that will promote the immature blasts to mature cells. While the second is a form of arsenic, used in small doses it can be helpful in treatment associated with side effects like fatigue, nausea, diarrhea and tingling in the hands or feet. You will be closely monitored in the hospital if you are diagnosed with APL.

Surgery and radiation therapy are not typically used to treat AML but may be used in very specific circumstances.

Here are a few questions to ask when discussing treatment options with your doctor:

  • How long does the treatment last?
  • How do I know if I am cured?
  • How often should I follow up with you?
  • How can I keep myself safe from getting an infection?
  • You say genetic mutations can cause AML — does that mean my kids will be affected?

When Is a Stem Cell Transplant Used to Treat AML?

A stem cell transplant is often suggested if patients are diagnosed with any subtype of leukemia that has a higher risk of relapse. Introducing a healthy bone marrow from a different person may decrease the risk of relapse in AML.

AML Stages

Because AML does not form tumors, it isn’t staged like other cancers. Instead, the subtype of AML and whether or not the leukemia has spread beyond the blood and marrow are the two pieces of information used to inform a patient’s treatment plan and prognosis. If you don’t know which subtype of AML you have, ask your doctor.

The World Health Organization (WHO) classification is often used to define the AML subtype.

AML Survival Rate

While AML is a type of cancer that can be difficult to cure, recent advancements have led to improved remission and cure rates. According to Surveillance, Epidemiology, and End Results (SEER) data, published by the National Cancer Institute, the relative five-year survival rate from 2010 to 2016 was 28.7%, meaning that approximately 29 of 100 people with AML were still alive five years after their diagnosis. In addition, age-adjusted death rates for AML improved on average 0.3% each year from 2009 to 2018.

It’s important to recognize that there is hope — regular medical advancements are making progress.


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