Version July 2025
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ACUTE MYELOID LEUKEMIA OVERVIEW —
Acute myeloid leukemia (also called AML) is a cancer of blood and bone marrow cells. AML affects a category of cells called myeloid cells, and it is called acute because it develops and advances quickly, and requires prompt treatment.
Normally, myeloid and other blood cells are produced in the bone marrow (the spongy area in the middle of bones) in a carefully regulated fashion. In someone with AML, this process is abnormal. Large numbers of immature and abnormal myeloid cells (called myeloblasts, or just "blasts") are produced, fill the bone marrow space, and are released into the bloodstream. In their immature state, these cells cannot perform their usual functions. The overgrowth of these cells leads to an inadequate number of normal, healthy blood cells, including white blood cells, red blood cells, and platelets. This can result in:
●Neutropenia (low numbers of neutrophils) – Neutrophils are a type of white blood cell that helps to fight infection. People with neutropenia are more likely to get infections.
●Anemia (low numbers of red blood cells) – Red blood cells circulate in the blood vessels and carry oxygen to our tissues. People without enough red cells may be pale and are often tired and short of breath.
●Thrombocytopenia (low numbers of platelets) – Platelets are small cells in the blood that help to prevent and stop bleeding. People with low platelets have bleeding and spontaneous bruising.
More detailed information about AML, written for healthcare providers, is available by subscription. (See 'Professional level information' below.)
GENERAL INFORMATION ABOUT ACUTE MYELOID LEUKEMIA TREATMENT —
A number of chemotherapy medications are effective against AML. The goal of treatment is to kill the malignant cells while limiting the effects on the residual normal bone marrow cells. Studies are underway to find the best medicines, doses, and treatment schedules for AML.
Researchers have discovered that AML is caused by mutations in bone marrow stem cells. The mutations are found in all the AML cells that these malignant stem cells produce. The cause of the mutations is not known for most people with AML, but some cases occur in individuals who previously received chemotherapy or radiation therapy. Mutations may also occur due to toxins in the environment, such as smoking or benzene. Rarely, people may inherit genes that increase the possibility of developing AML.
These mutations are used to classify the type of AML that you have, and they may guide the type of treatment that you are given. Your age and other active medical problems may also affect the choice of treatment.
The usual treatment of AML is divided into two phases: induction of remission and post-remission therapy.
REMISSION INDUCTION IN AML —
The initial phase of treatment is called remission induction (or induction therapy). The goal of induction therapy is to decrease the number of leukemia cells to an undetectable level, restore the production of normal blood cells, and relieve AML-related symptoms. The type and intensity of remission induction therapy varies with the AML-related mutations, age, and fitness.
Chemotherapy refers to medicines that interfere with their ability of cells to multiply and grow. Because most of an adult's normal body cells are not actively growing, they are not as affected by chemotherapy as cancer cells. However, the normal cells in the bone marrow (where the blood cells are produced), hair follicles, and the lining of the gastrointestinal (GI) tract are all actively reproducing and growing. Effects of chemotherapy on these and other normal tissues cause side effects during treatment, including hair loss, nausea, diarrhea, anemia (lowered red blood cell count), an increased risk of infection (lowered white blood cell count), and bleeding (lowered platelet count).
Intensive remission induction therapy for AML generally includes a drug called cytarabine (usually given as a continuous intravenous infusion for seven days) plus either daunorubicin or idarubicin (given as intravenous injections on the first three days of treatment); this combination is sometimes called the "7+3" regimen. A third drug may be added to the 7+3 regimen for leukemia that has particular mutations. In some circumstances, cytarabine and daunorubicin are given as a combination medication (called CPX-351) that is infused on three of the first five days of treatment. In certain circumstances, other types of induction therapy may be used. (See 'AML in older or less-fit people' below.)
Chemotherapy kills AML cells over the first 7 to 14 days, but it takes several weeks for the bone marrow to produce enough normal blood cells again. A second course of chemotherapy may be needed if the leukemia did not respond well enough to the first induction treatment. As a result, 7+3 induction therapy usually requires hospitalization for four or more weeks for supportive care with IV antibiotics and frequent transfusions.
Induction therapy frequently results in a complete remission of the AML, meaning that there are no visible leukemia cells in the blood or bone marrow when examined under a microscope, and the bone marrow is functioning normally. However, such remissions are usually short-lived unless additional post-remission therapy is given. (See 'Post-remission therapy of AML' below.)
Complete remission — The first goal of AML treatment is to achieve a complete remission (CR). A bone marrow biopsy and blood testing are done to determine if you have achieved a CR.
A person is described as having a CR when there are no visible leukemia cells in the blood or bone marrow when examined under a microscope, and the bone marrow is functioning normally. However, such remissions are usually short-lived unless additional post-remission therapy is given. (See 'Post-remission therapy of AML' below.)
Sensitive laboratory methods can sometimes detect leukemia that is not readily observed by a microscope; this is called measurable residual disease (MRD), and the persistence of this low level of disease may impact additional treatment plans.
POST-REMISSION THERAPY OF AML —
Post-remission therapy is given with the intention of killing leukemia cells that may remain in the bone marrow or blood, but are undetectable under the microscope.
There are three basic treatment choices for post-remission therapy:
●Additional chemotherapy, sometimes called remission consolidation therapy
●Stem cell transplantation from a healthy donor (allogeneic stem cell transplantation)
●Stem cell transplantation using your own stem cells (autologous stem cell transplantation).
The "best" post-remission treatment depends upon several factors, including how aggressive or resistant to treatment the AML is. People with AML can be classified by risk based on genetic testing of their leukemia cells:
●People with favorable-risk disease are usually advised to continue with chemotherapy. This postremission treatment is called consolidation therapy or post-remission chemotherapy. Many patients with favorable-risk AML can be cured with consolidation chemotherapy.
●People with unfavorable-risk or adverse-risk disease are usually advised to have an allogeneic stem cell transplantation, if possible.
●The best treatment for intermediate-risk disease is not clear. Most cases of AML are intermediate-risk disease. Participation in a clinical trial is recommended, when possible. (See 'Clinical trials' below.)
Consolidation chemotherapy — Consolidation therapy often includes "high-dose cytarabine." Cytarabine is given again by intravenous infusion but at a higher dose than was used during the remission induction phase. Consolidation chemotherapy is usually given in the hospital over several days and repeated every four to six weeks. Most patients are able to recuperate at home between courses of chemotherapy, although transfusions may be needed as an outpatient until the normal blood counts recover. Consolidation chemotherapy is given for approximately two to six months.
Stem cell transplantation — Stem cell transplantation is also called bone marrow transplantation or hematopoietic cell transplantation (HCT).
For HCT, you will be given high doses of chemotherapy or total body irradiation (TBI) that are referred to as conditioning therapy (also called preparative therapy). Conditioning therapy is intended to kill cancer cells, but it also destroys nearly all the normal cells developing in the bone marrow. This means that your body's normal source of critical blood components (ie, the bone marrow) is no longer functional. Conditioning therapy can cause side effects that may not be tolerable for some patients. In some cases, reduced-intensity or non-myeloablative conditioning may be used, which causes fewer and less severe side effects.
After receiving conditioning therapy, you must receive a healthy supply of blood or bone marrow stem cells from a healthy donor to re-establish the blood cell production in the bone marrow and also generate a new immune system. The transplanted cells (the "graft") are given by an intravenous transfusion. (See "Patient education: Hematopoietic cell transplantation (bone marrow transplantation) (Beyond the Basics)".)
Transplantation may be recommended for some patients with adverse prognoses, those who have had a relapse following a period of remission, and those who do not achieve complete remission after initial induction therapy. However, HCT is not recommended for all patients with AML. Serious, and sometimes even fatal, complications are more common after donor stem cell transplantation than with chemotherapy. In certain groups of people, there is no clear benefit of stem cell transplantation over chemotherapy.
There are two main types of stem cell transplantation:
●Allogeneic HCT – Allogeneic transplantation, which uses a stem cell graft from a healthy donor, is generally preferred over autologous transplantation for people with AML, but there are more side effects.
Ideally, the stem cell graft is donated by a sibling with a similar genetic makeup (called a matched related donor). If you do not have a sibling with similar genetic characteristics, an unrelated person with a similar genetic makeup may be used (called a matched unrelated donor). If neither type of matched graft is available, other possibilities include a relative who shares some genetic make-up (a so-called haploidentical donor) or cord blood stem cells collected at birth from a newborn's umbilical cord, but these "alternative" donors are less proven in this setting.
Allogeneic transplantation treats AML in two ways. First, the conditioning chemotherapy or radiation therapy that is given immediately before transplantation kills leukemia cells in the blood and bone marrow that might have been resistant to lower doses of chemotherapy. Second, when stem cells from another person are transfused, some of the donor stem cells mature into immune cells, and these donor immune cells can cause an immune response that helps destroy any remaining leukemia cells. This is called the "graft-versus-leukemia" or "graft-versus-tumor" effect.
Unfortunately, this response is closely associated with a complication called "graft-versus-host disease," in which the immune response includes an attack on some of the patient's own healthy organs. Symptoms can include severe skin rash, diarrhea, liver damage, and other problems.
●Autologous HCT – Autologous transplantation uses your own normal stem cells, which are collected when you are in complete remission. Shortly afterward, high-dose chemotherapy or radiation is given and the harvested cells are returned by intravenous infusion.
Because the transplanted stem cells do not come from another person, there is no "graft-versus-host" disease. This helps avoid most of the side effects of treatment. However, autologous transplantation is also somewhat less effective than allogeneic transplantation in fighting the leukemia, because of the lack of a "graft-versus-leukemia" effect. Autologous transplantation is less often recommended for treatment of AML for this reason.
AML IN OLDER OR LESS-FIT PEOPLE —
In general, older people or those with other significant medical conditions do not respond as well to treatment for AML as younger, fit individuals. There is no specific age cut-off, but people older than 60 or 65 years are often considered "older" in this setting, although good medical fitness can enable older patients to receive intensive treatments.
Outcomes in older people may be related to the following factors:
●Leukemia cells that are harder to treat with standard chemotherapy drugs ("chemotherapy-resistant" cells) are more common in older people.
●Other serious medical conditions, such as diabetes, kidney, lung, or heart disease, are more common as we age and may increase the risk of treatment-related complications.
Treatment decisions for older people with AML are best made on a case-by-case basis. Less-intensive treatments, such as azacitidine or decitabine, with or without venetoclax, can be effective against AML and are generally better tolerated than intensive induction therapy in older patients. Some older people with slowly progressing AML, severe underlying health problems, or genetically high-risk and unfavorable AML, may decide that the expected benefit of chemotherapy is not worth the anticipated discomfort, hospitalization, and potentially toxic side effects. For some, supportive care using blood transfusions for anemia or bleeding and antibiotics as needed for infections may be chosen, even though this does not treat the underlying AML.
Which treatment is right for me? — You and your family should get information from your health care provider about your type of AML, the expected benefits of various treatments, possible side effects and toxicities, and your long-term outlook. These discussions are critical to determining the best course of action for you. Many new drugs or drug combinations are currently being studied for patients with AML. The best treatment for you may include a clinical trial so that you can have access to the latest new drugs. (See 'Clinical trials' below.)
TREATMENT OF RELAPSED OR RESISTANT ACUTE MYELOID LEUKEMIA —
A limited number of treatments are effective in the treatment of AML that does not respond to induction therapy or AML that relapses after initial chemotherapy:
●About half of people with a remission that lasted more than one year can benefit from a second treatment with remission induction therapy. This may include drugs like those that were used for the earlier remission induction treatment, or similar drug combinations. For relapsed AML that has a mutation in an IDH gene, the KMT2A gene, or the FLT3 gene, treatments that specifically target those mutations can be combined with induction chemotherapy or used alone. Stem cell transplantation should be considered for patients with relapsed AML after a second complete or good partial remission is obtained.
●People who relapse within 12 months of their initial diagnosis usually have AML with a high degree of drug resistance and therefore have a lower likelihood of achieving a second complete remission. Medicines specifically approved for use in patients with relapsed AML or experimental agents may be useful in this setting, followed by stem cell transplantation, if a remission occurs.
More detailed information is available by subscription. (See "Treatment of relapsed or refractory acute myeloid leukemia".)
LONG-TERM MONITORING OF ACUTE MYELOID LEUKEMIA —
Once you are in complete remission and have completed post-remission therapy, you will need long-term monitoring so that any relapse can be detected quickly and treated. Relapse is most likely to occur within the first two years after completion of induction chemotherapy, and it becomes less common later.
Prognosis — Your chances of being cured of AML depend upon your age, other health conditions, how aggressive your AML is, and whether your AML was caused by chemotherapy or radiation therapy that was given to treat another disorder before you were diagnosed with AML. In one study, approximately 65, 40, and 15 percent of people with favorable, intermediate, and unfavorable risk disease, respectively, were alive five years after diagnosis.
However, when discussing chances of cure, it is important to remember that these numbers represent averages and do not necessarily predict what will happen to you.
CLINICAL TRIALS —
Many patients with leukemia will be asked to enroll in a clinical (research) trial. A clinical trial is a carefully controlled way to study the effectiveness of new treatments or new combinations of known therapies. Ask your doctor for more information, or read about clinical trials at:
●www.cancer.gov/research/participate/clinical-trials
Videos addressing common questions about clinical trials are available from the American Society of Clinical Oncology (www.cancer.net/research-and-advocacy/clinical-trials/welcome-pre-act).
WHERE TO GET MORE INFORMATION —
Your healthcare provider is the best source of information for questions and concerns related to your medical problem.
This article will be updated as needed on our website (www.uptodate.com/patients). Related topics for patients, as well as selected articles written for healthcare professionals, are also available. Some of the most relevant are listed below.
Patient level information — UpToDate offers two types of patient education materials.
The Basics — The Basics patient education pieces answer the four or five key questions a patient might have about a given condition. These articles are best for patients who want a general overview and who prefer short, easy-to-read materials.
Patient education: Acute myeloid leukemia (AML) (The Basics)
Patient education: Leukemia in adults (The Basics)
Patient education: Neutropenia and fever in people being treated for cancer (The Basics)
Patient education: Bone marrow aspiration and biopsy (The Basics)
Beyond the Basics — Beyond the Basics patient education pieces are longer, more sophisticated, and more detailed. These articles are best for patients who want in-depth information and are comfortable with some medical jargon.
Professional level information — Professional level articles are designed to keep doctors and other health professionals up-to-date on the latest medical findings. These articles are thorough, long, and complex, and they contain multiple references to the research on which they are based. Professional level articles are best for people who are comfortable with a lot of medical terminology and who want to read the same materials their doctors are reading.
Acute myeloid leukemia: Clinical manifestations, pathologic features, and diagnosis
Clinical manifestations, pathologic features, and diagnosis of acute promyelocytic leukemia in adults
Acute myeloid leukemia: Cytogenetic abnormalities
Acute myeloid leukemia: Induction therapy in medically fit adults
Initial treatment of acute promyelocytic leukemia in adults
Molecular biology of acute promyelocytic leukemia
Acute myeloid leukemia: Molecular genetics
Acute myeloid leukemia: Overview of complications
Acute myeloid leukemia: Pathogenesis
Acute myeloid leukemia in younger adults: Post-remission therapy
Acute myeloid leukemia: Risk factors and prognosis
Therapy-related myeloid neoplasms: Epidemiology, causes, evaluation, and diagnosis
Acute myeloid leukemia: Management of medically unfit adults
Treatment of relapsed or refractory acute myeloid leukemia
Treatment of relapsed or refractory acute promyelocytic leukemia in adults
Acute myeloid leukemia: Induction therapy in medically fit adults, section on 'Introduction'
The following organizations also provide reliable health information.
●National Library of Medicine (medlineplus.gov/healthtopics.html)
●National Cancer Institute (www.cancer.gov/about-cancer)
●American Cancer Society (www.cancer.org)
●The Leukemia & Lymphoma Society (www.lls.org)
●National Marrow Donor Program (bethematch.org/)
●The American Society of Clinical Oncology (https://www.cancer.net/cancer-types/leukemia-acute-myeloid-aml)
●The American Society of Hematology (www.hematology.org/education/patients)
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