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Chemotherapy (chemo) is the use of anti-cancer drugs that are injected into a vein, under the skin, or into the cerebrospinal fluid (CSF), or drugs that are taken by mouth to destroy or control cancer cells. Except when given into the CSF, these drugs enter the bloodstream and reach all areas of the body, making this treatment useful for cancers such as leukemia that spread throughout the body.
Chemotherapy is the main treatment for most people with acute myeloid leukemia (AML), although sometimes other treatments might be used as well.
Less intense chemo might be recommended for people with other serious health issues, but advanced age by itself is not a barrier to getting chemo.
Treatment of AML is usually divided into phases:
In a third phase, called maintenance (or post-consolidation), low doses of chemo (or other treatments) are given for months or years after consolidation is finished. This is often used to treat acute promyelocytic leukemia (APL), and it is sometimes used for other types of AML as well.
Most chemo drugs used to treat AML are given into a vein in the arm (IV), although some can be injected under the skin or taken by mouth as pills. If there are signs that the leukemia has reached the brain or spinal cord (which is not common with AML), chemo might also be given into the CSF (known as intrathecal chemo). This can be done with a thin tube (catheter) that is put in through a small hole in the skull (such as an Ommaya reservoir), or during a lumbar puncture (spinal tap).
Most chemo regimens used to treat AML are intensive and can cause serious side effects, so treatment is typically given in the hospital.
The chemo drugs used most often to treat AML are a combination of:
Other chemo drugs that may be used to treat AML include:
For more on how chemo is used to treat AML, see Typical Treatment of Most Types of Acute Myeloid Leukemia (AML), Except APL.
Chemo drugs can affect some normal cells in the body, which can lead to side effects. The side effects of chemo depend on the type and dose of drugs given and how long they are taken. Side effects can include:
Chemo drugs also affect the normal cells in bone marrow, which can lower blood cell counts. This can lead to:
Most side effects from chemo go away once treatment is finished. Low blood cell counts can last weeks, but then should return to normal. There are often ways to lessen these side effects. For example, drugs can be given to help prevent or reduce nausea and vomiting. Be sure to ask about medicines to help reduce side effects, and let your doctor or nurse know when you do have side effects so they can be managed effectively.
Low white blood cell counts: Some of the most serious side effects of chemo are caused by low white blood cell counts.
If your white blood cell counts are very low during treatment, you can help lower your risk of infection by carefully avoiding exposure to germs. During this time, your doctor or nurse may tell you to:
You may get antibiotics before you have signs of infection or at the earliest sign that an infection may be developing (such as a fever). You may also get drugs that help prevent viral and fungal infections.
Drugs known as growth factors, such as filgrastim (Neupogen, other names), pegfilgrastim (Neulasta, other names), and sargramostim (Leukine), are sometimes given to increase the white blood cell counts after chemo, to help lower the chance of infection. However, it’s not clear if they have an effect on treatment success.
Low platelet counts: If your platelet counts are low, you may be given drugs or platelet transfusions to help prevent bleeding.
Low red blood cell counts: Shortness of breath and extreme fatigue caused by low red blood cell counts (anemia) may be treated with drugs or with red blood cell transfusions.
Decisions about when a patient can leave the hospital are often influenced by their blood counts. Some people find it helpful to keep track of their counts. If you are interested in this, ask your doctor or nurse about your blood cell counts and what these numbers mean.
Side effects of specific drugs: Certain drugs have some specific possible side effects. For example:
Other organs that could be damaged by chemo drugs include the kidneys, liver, testicles, ovaries, and lungs. Doctors and nurses carefully monitor treatment to limit the risk of these side effects as much as possible.
If serious side effects occur, the chemo may have to be reduced or stopped, at least for a short time. Careful monitoring and adjustment of drug doses are important because some side effects can last a long time.
Tumor lysis syndrome: This side effect of chemo can occur in patients who have large numbers of leukemia cells in the body, mainly during the induction phase of treatment. When chemo kills these cells, they break open and release their contents into the bloodstream. This can overwhelm the kidneys, which aren’t able to get rid of all of these substances at once. Excess amounts of certain minerals can also affect the heart and nervous system. This can be prevented by giving extra fluids during treatment and by giving certain drugs, such as bicarbonate, allopurinol, and rasburicase, which help the body get rid of these substances.
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Last Revised: February 3, 2023