ON THIS PAGE: You will learn about the different treatments doctors use for adults with AML. Use the menu to see other pages.
This section tells you the treatments that are the standard of care
for this type of leukemia. “Standard of care” means the best treatments
known. When making treatment plan decisions, patients are encouraged to
consider clinical trials as an option. A clinical trial is a research
study that tests a new approach to treatment. Doctors want to learn
whether the new treatment is safe, effective, and possibly better than
the standard treatment. Clinical trials can test a new drug, a new
combination of standard treatments, or new doses of standard drugs or
other treatments. Your doctor can help you consider all your treatment
options. To learn more about clinical trials, see the About Clinical Trials and Latest Research sections.
Treatment overview
In cancer care, different types of doctors often work together to
create a patient’s overall treatment plan that combines different types
of treatments. This is called a multidisciplinary team.
Cancer care teams include a variety of other health care professionals,
such as physician assistants, oncology nurses, social workers,
pharmacists, counselors, dietitians, and others.
Descriptions of the most common treatment options for AML are listed
below. Treatment options and recommendations depend on several factors,
including the subtype, morphology, and cytogenetics of AML (see Subtypes),
possible side effects, and the patient’s preferences and overall
health. Your care plan may also include treatment for symptoms and side
effects, an important part of cancer care.
The most successful treatment for AML depends on the results of the
first treatment, so it is important for patients to have their first
treatments at a center experienced with treating AML.
Take time to learn about all of your treatment options and be sure to
ask questions about things that are unclear. Talk with your doctor
about the goals of each treatment and what you can expect while
receiving the treatment. Learn more about making treatment decisions.
Intensive chemotherapy
Chemotherapy is the use of drugs to destroy cancer cells,
usually by ending the cancer cells’ ability to grow and divide. The
drugs get into the bloodstream to reach cancer cells throughout the
body. Chemotherapy is given by a medical oncologist, a doctor who
specializes in treating cancer with medication. Or, it is given by a
hematologist, a doctor who specializes in treating blood disorders.
Chemotherapy is the primary treatment for AML. Systemic chemotherapy
is delivered through the bloodstream to reach cancer cells throughout
the body. Common ways to give chemotherapy include:
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An intravenous (IV) tube placed into a vein using a needle. When
chemotherapy is given by IV, it may be given into a larger vein or a
smaller vein, such as in the arm. When it is given into a larger vein, a
central venous catheter or port may need to be placed in the body.
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An injection into the cerebral spinal fluid
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In a pill or capsule that is swallowed (orally)
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An injection under the skin, called a subcutaneous injection
A chemotherapy regimen, or schedule, usually consists of a
specific number of cycles given over a set period of time. A patient may
receive 1 drug at a time or combinations of different drugs given at
the same time. Several drugs are used to treat AML, which are discussed
below.
Learn more about the basics of 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.
Chemotherapy by phase
Chemotherapy for AML can be divided into 3 phases: induction, post-remission, and consolidation.
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Induction therapy. This is the first period of treatment after
a person’s diagnosis. The goal of induction therapy is a complete
remission (CR). A person has a CR when:
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Blood counts have returned to normal
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Leukemia cannot be found in a bone marrow sample when examined under the microscope
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There are no longer any signs and symptoms of AML
The combination of cytarabine (Cytosar-U) given over 4 to 7 days
and an anthracycline drug, such as daunorubicin (Cerubidine) or
idarubicin (Idamycin), given for 3 days is used most often. Patients may
also be given hydoxyurea (Droxia, Hydrea) to help lower white blood
cell counts. In addition to killing leukemia cells, these drugs also
damage healthy cells, increasing the risk of infection and bleeding (see
below). Most patients will need to stay in the hospital for 3 to 5
weeks during induction therapy before their blood counts return to
normal. Sometimes, 2 rounds of therapy are needed to achieve a CR.
Approximately 75% of younger adults with AML and about 50% of patients
older than 60 achieve a CR after treatment.
Some older adults may not be able to have induction therapy with the
standard drugs. The drugs decitabine (Dacogen), azacitidine (Vidaza),
and low dose cytarabine may be used instead. A clinical trial is also an
option.
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Post-remission therapy. After induction therapy, a variety of
different drugs are used to destroy AML cells that remain but cannot be
detected by medical tests. AML will almost certainly recur if no further
treatment is given after a CR. For some patients, bone marrow/stem cell
transplantation (see below) is recommended as part of post-remission
therapy.
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Consolidation therapy. Chemotherapy or stem cell transplantation may be used for consolidation therapy.
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Younger adults in remission are commonly given 2 to 4 rounds of high-
or intermediate-dose cytarabine or other intensive chemotherapy at
monthly intervals. Several different regimens are used for older
patients. Although chemotherapy is usually given in the hospital, most
of the recovery time can be spent at home.
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A bone marrow/stem cell transplantation is often recommended as
consolidation therapy for younger patients in whom cytogenetic or
molecular studies predict a poorer prognosis with only chemotherapy.
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Stem cell transplantation/bone marrow transplantation. A stem
cell transplant is a medical procedure in which bone marrow that
contains leukemia is destroyed and then replaced by highly specialized
cells, called hematopoietic stem cells, that develop into healthy bone
marrow. Hematopoietic stem cells are blood-forming cells 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 it is the stem cells in the blood that are typically
being transplanted, not the actual bone marrow tissue.
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 leukemia, results of any previous
treatment, and patient’s age and general health.
There are 2 types of stem cell transplantation depending on the
source of the replacement blood stem cells: allogeneic (ALLO) and
autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the
patient’s own stem cells. ALLO transplants are generally used for AML.
The goal is to destroy all of the cancer cells in the marrow, blood,
and other parts of the body using high doses of chemotherapy and/or
radiation therapy and then allow replacement blood stem cells to create
healthy bone marrow.
Side effects depend on the type of transplant, your general health, and other factors. Learn more about the basics of stem cell and bone marrow transplantation.
Side effects of chemotherapy
Chemotherapy for AML attacks rapidly dividing cells, including those
in healthy 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 there are effective drugs to help prevent and control nausea and vomiting.
The side effects of chemotherapy may be different depending on the
drugs used. Patients are encouraged to talk with their doctors about
short-term and long-term side effects before treatment begins.
Because of the effect on healthy blood cells in the bone marrow,
chemotherapy used for AML will lower the body’s ability to fight
infection for a short time, and increased bruising, bleeding, and
fatigue may be common. People with AML often 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,
or ability to have a child in the future. Patients concerned about this
are encouraged to talk with a fertility specialist before treatment
begins.
Targeted therapy
Targeted therapy is a treatment that targets the leukemia’s specific
genes, proteins, or the tissue environment that contributes to the
growth and survival of the leukemia. This type of treatment blocks the
growth and spread of leukemia cells while limiting damage to healthy
cells.
Recent studies show that not all cancers have the same targets. To
find the most effective treatment, your doctor may run tests to identify
the genes, proteins, and other factors in your tumor. This helps
doctors better match each patient with the most effective treatment
whenever possible. In addition, many research studies are taking place
now to find out more about specific molecular targets and new treatments
directed at them. Learn more about the basics of targeted treatments.
The following targeted treatments may be used for AML, depending on the gene mutations found in the leukemia cells:
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Midostaurin (Rydapt) for people who have AML with a FLT3 gene mutation. About 25% to 30% of people with AML have AML with a FLT3 gene mutation.
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Enasidenib (IDHIFA) for people who have relapsed or refractory AML with an IDH2 mutation.
Talk with your doctor about possible side effects for a specific medication and how they can be managed
Acute promyelocytic leukemia (APL) treatment
The treatment of the APL subtype of AML is very different. This
subtype is very sensitive to the effects of all-trans retinoic acid
(ATRA). ATRA is 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
arsenic trioxide (Trisenox) are very likely to have a CR. Less
commonly, chemotherapy containing regimens (see above) with idarubicin,
daunorubicin, or cytarabine may also be used. Arsenic trioxide may be
used during induction therapy alone or in combination with ATRA during
post-remission therapy or if APL comes back after treatment.
Mild to severe bleeding is a common symptom of APL. 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, some patients with APL may benefit from use of
ATRA plus low dose oral chemotherapy for 1 to 2 years after the initial
treatment.
Radiation therapy
Radiation therapy is the use of high-energy x-rays or other particles
to destroy 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, or schedule, usually consists of a specific
number of treatments given over a set period of time. Because AML is
found throughout the blood, radiation therapy is generally used only
when leukemia cells have spread to the brain or to shrink a myeloid
sarcoma.
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 the basics of
radiation therapy.
Getting care for symptoms and side effects
Leukemia and its treatment often cause side effects. In addition to
treatments intended to slow, stop, or eliminate the disease, an
important part of care is relieving a person’s symptoms and the side
effects of treatment. This approach is called palliative or supportive
care, and it includes supporting the patient with his or her physical,
emotional, and social needs.
Palliative care is any treatment that focuses on reducing symptoms,
improving quality of life, and supporting patients and their families.
Any person, regardless of age or type and stage of cancer, may receive
palliative care. It works best when palliative care is started as early
as needed in the cancer treatment process. People often receive
treatment for the leukemia at the same time that they receive treatment
to ease side effects. In fact, patients who receive both at the same
time often have less severe symptoms, better quality of life, and report
they are more satisfied with treatment.
Palliative treatments vary widely and often include medication,
nutritional changes, relaxation techniques, emotional support, and other
therapies. You may also receive palliative treatments similar to those
meant to eliminate the leukemia, such as chemotherapy or radiation
therapy. Talk with your doctor about the goals of each treatment in the
treatment plan.
Before treatment begins, talk with your health care team about the
possible side effects of your specific treatment plan and palliative
care options. During and after treatment, be sure to tell your doctor or
another health care team member if you are experiencing a problem so it
can be addressed as quickly as possible. Learn more about palliative care.
Refractory AML
If leukemia is still present after initial treatment, the disease is
called refractory AML. If this happens, it is a good idea to talk with
doctors who have experience in treating it. Doctors can have different
opinions about the best standard treatment plan. Also, clinical trials
might be an option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan.
Your treatment plan may include new drugs being tested in clinical
trials. An ALLO stem cell transplant should also be considered.
Palliative care will also be important to help relieve symptoms and side
effects.
For most patients, a diagnosis of refractory leukemia is very
stressful and, at times, difficult to bear. Patients and their families
are encouraged to talk about the way they are feeling with doctors,
nurses, social workers, or other members of the health care team. It may
also be helpful to talk with other patients, including through a
support group.
Remission and the chance of recurrence
A remission is when the leukemia cannot be detected in the body,
there are no symptoms, and a patient’s blood counts are normal. This may
also be called having “no evidence of disease” or NED.
A remission may be temporary or permanent. This uncertainty causes
many people to worry that the cancer will come back. While many
remissions are permanent, it’s important to talk with your doctor about
the possibility of the leukemia returning. Understanding your risk of
recurrence and the treatment options may help you feel more prepared if
the disease does return. Learn more about coping with the fear of recurrence.
If the leukemia does return after the original treatment, it is
called recurrent or relapsed leukemia. When this occurs, a new cycle of
testing will begin again to learn as much as possible about the
recurrence. After this testing is done, you and your doctor will talk
about your treatment options. Often the treatment plan will include the
treatments described above, such as chemotherapy, stem cell
transplantation, targeted therapy, and radiation therapy, but they may
be used in a different combination or given at a different pace. Your
doctor may suggest clinical trials that are studying new ways to treat
this type of recurrent leukemia. Whichever treatment plan you choose,
palliative care will be important for relieving symptoms and side
effects.
The treatment for recurrent AML often depends on the length of the
initial remission. If the AML comes back after a long remission, the
original treatment may work again. If the remission was short, then
other drugs are used, often through a clinical trial. An ALLO stem cell
transplant may be the best option for patients whose leukemia has come
back after initial treatment. However, many drugs and other approaches
are being researched in clinical trials and these may provide other
treatment options.
People with recurrent leukemia often experience emotions such as
disbelief or fear. Patients are encouraged to talk with their health
care team about these feelings and ask about support services to help
them cope. Learn more about dealing with cancer recurrence.
If treatment doesn’t work
Recovery from leukemia is not always possible. If the leukemia cannot
be cured or controlled, the disease may be called advanced or terminal.
This diagnosis is stressful, and for many people, advanced leukemia
may be difficult to discuss because it is incurable. However, it is
important to have open and honest conversations with your doctor and
health care team to express your feelings, preferences, and concerns.
The health care team is there to help, and many team members have
special skills, experience, and knowledge to support patients and their
families. Making sure a person is physically comfortable and free from
pain is extremely important.
Patients with advanced leukemia who have no more effective treatment
options available may want to consider a type of palliative care called
hospice care. Hospice care is designed to provide the best possible
quality of life for people who are near the end of life. You and your
family are encouraged to talk with the health care team about hospice
care options, which include hospice care at home, a special hospice
center, or other health care locations. Nursing care and special
equipment can make staying at home a workable option for many families.
Learn more about advanced cancer care planning.
After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.
The next section in this guide is About Clinical Trials.
It offers more information about research studies that are focused on
finding better ways to care for people with cancer. You may use the menu
to choose a different section to read in this guide.