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Side Effects

Both cancer therapy and acute myeloid leukemia (AML) can produce side effects. For most patients, side effects are temporary and subside once the body adjusts to therapy or when therapy is completed. For other patients, side effects can be more severe, sometimes requiring hospitalization. The side effects of chemotherapy may vary, depending on the drugs used and the overall health of the patient. 

Before you undergo treatment, talk with your doctor about potential side effects. Medication and other therapies can prevent or manage many side effects.

Side Effects and Complications

Low blood cell counts. AML decreases the production of normal blood cells. In addition, chemotherapy is toxic to both normal blood cells and AML cells. The normal blood cells are eliminated from the marrow along with AML cells. For the patient, this can lead to:

  • Anemia: Low red blood cell count
  • Thrombocytopenia: Low platelet count
  • Neutropenia: Low neutrophil (a type of white blood cell) count

Patients may need red blood cell and platelet  transfusions for a period of several weeks during treatment. After that, blood cell counts usually return to normal.

Infection. During treatment for AML, the deficiency of neutrophils and monocytes (types of white blood cells) can lead to infection. The risk of infection may be increased because chemotherapy damages the lining of the mouth and intestines, making it easier for bacteria to enter the blood. When the white blood cell count is low and infection risk is increased, antibiotics are given to prevent or treat infection. White blood cell transfusions are generally not used for AML patients, so doctors sometimes use growth factors to help increase a patient’s white blood cell count. However, growth factors are used only in special circumstances, and routine use of these agents is not recommended.

Patients and families should practice frequent and vigorous handwashing and take other precautions to avoid exposure to bacteria, viruses and other infection-causing agents. Caregivers of patients who have central lines or ports need to carefully clean the sites, as instructed by the medical team.

Seek medical attention immediately if you show any signs of infections such as:

  • A temperature of 100.4°F or higher
  • Chills
  • Coughing
  • Sore throat
  • Pain during urination
  • Diarrhea

Patients with AML Patients with AML are advised to receive certain vaccinations. Speak to your doctor for more information. 

Graft-versus-Host Disease. If you undergo an allogeneic stem cell transplantation, you're at high risk of developing graft versus host disease (GVHD). The older you are, the higher your risk for GVHD. GVHD develops when the donor's immune cells mistakenly attack the patient's normal cells. GVHD can be mild, moderate or severe - even life threatening.

Tumor Lysis Syndrome. This condition occurs when a large number of cancer cells die within a short period of time, releasing their contents into the blood. TLS can be severe during the early phases of treatment, especially for patients who have very high white blood cell counts before they start induction therapy. As the leukemia cells die, they break apart and release their contents into the bloodstream, which changes the normal balance of chemicals in the blood. This can overwhelm the kidneys because they cannot get rid of the substances all at once. Uric acid is one of the chemicals released by dying cancer cells. Very high levels of uric acid and other chemicals can cause severe damage to the kidneys and heart. If untreated, TLS can lead to heart arrhythmias, seizures, loss of muscle control, acute kidney failure and even death.Patients with leukemia who are at high risk for or have developed laboratory or clinical evidence of TLS are given drugs, such as allopurinol (Zyloprim®) or rasburicase (Elitek®), which prevent or lessen TLS.

Differentiation Syndrome. This is a potentially life-threatening complication of treatment with differentiating agents, such as all-trans retinoic acid (ATRA), enasidenib (Idhifa®) and ivosidenib (Tibsovo®). Symptoms include fever, swelling in the limbs and trouble breathing. Patients may also experience a drop in blood pressure and have fluid build-up around the lungs or heart. Treatment must begin at the first signs or symptoms. Treatment consists of steroid therapy or the administration of the antimetabolite drug hydroxyurea and other chemotherapy drugs to decrease the number of white blood cells, which are the source of differentiation effects. In severe cases, use of differentiating agents is stopped.

Other Side Effects. The following side effects are also common:

  • Mouth ulcers
  • Diarrhea
  • Hair loss
  • Rashes
  • Nausea and vomiting
  • Fatigue
  • Loss of appetite
  • Neuropathy


Chemotherapy may also affect fertility (the ability to have a biological child in the future). Patients concerned about this potential side effect should talk with a fertility specialist before beginning treatment. Click here to learn more about fertility. 

Long-Term and Late Effects of Treatment

Treatment for individuals who have AML sometimes causes effects that continue after treatment ends (long-term effects) or develop much later in life (late effects). Various factors can influence the risk of developing long-term or late effects, including the following:

  • Type and duration of treatment
  • Age at the time of treatment
  • Sex and overall health

Most AML patients are treated with an anthracycline, such as daunorubicin. Anthracyclines have been associated with increased risk for heart muscle injury or chronic heart failure. Heart disease may not become apparent until many years after therapy ends.

Stem cell transplantation is used to treat some patients with AML. It has been associated with long-term or late effects, including infertility, thyroid dysfunction, chronic fatigue and risk for developing a second cancer (lymphoma; melanoma of the skin; or cancer of the tongue and salivary glands, central nervous system, bone, soft tissue and/or thyroid gland). The number of patients who develop secondary cancers is small. These and other possible long-term and late effects can be managed.


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