The first phase of treatment is induction therapy. The goal of induction is to destroy as many cancer cells as possible in order to achieve (induce) a complete remission. Typically, initial therapy requires a hospital stay of four to six weeks.
The most common induction regimen for AML includes cytarabine and an anthracycline drug, such as daunorubicin or idarubicin. This is called the “7+3” regimen, because cytarabine is most often given by continuous intravenous (IV) infusion over seven days, while the anthracycline drug is given by an IV infusion in a single dose for three days during the first week of treatment.
Other drugs may be added or substituted for higher-risk patients, such as:
- Midostaurin (Rydapt®) for AML with an FLT3 mutation
- Gemtuzumab ozogamicin (Mylotarg™) for CD33-positive AML
Other drugs may be used as a substiture for the 7+3 regimen including:
- CPX-351 (Vyxeos®)
- High-dose cytarabine with idarubicin or daunorubicin and etoposide
- High-dose cytarabine with mitoxantrone
- Fludarabine with high-dose cytarabine, idarubicin and a granulocyte colony-stimulating factor (G-CSF)
Some drugs are given by mouth (orally). Other drugs are inserted directly into the patient’s bloodstream through a central line, a port, or a PICC.
At the end of induction therapy, blood and bone marrow tests are done to see how well your treatment is working. A remission is achieved when you no longer have signs and symptoms on AML.
If the initial treatment does not induce a remission, induction therapy can be repeated, either with the same drugs or with a new chemotherapy regimen.
Most patients develop dangerously low blood cell counts and may become very ill, needing supportive (palliative) care with IV antibiotics and frequent blood transfusions during this time.
Even when a complete remission is achieved, some leukemia cells that cannot be seen with a microscope may still remain in the bone marrow. This is referred to as “minimal residual disease (MRD),” also called “measurable residual disease.” Patients who achieve remission after initial treatment but have MRD are at increased risk of disease relapse.
Testing for MRD can help doctors identify patients who may benefit from further treatment with intensified therapies, such as allogeneic stem cell transplantation. It is important to get tested for MRD after achieving remission. The tests used most commonly to detect MRD are flow cytometry, polymerase chain reaction (PCR) and DNA sequencing.
Learn more about MRD in our free fact sheet, Minimal/Measurable Residual Disease (MRD).
"Consolidation therapy" is treatment that is given after cancer is in remission following induction therapy. The goal of consolidation therapy is to lower the number of residual leukemia cells in the body or eliminate them entirely to help prevent the leukemia from returning.
There are two basic treatment choices for consolidation therapy:
- Additional intensive chemotherapy
- Allogeneic stem cell transplantation
Patients with favorable risk factors are often given intensive chemotherapy with high-dose cytarabine and other drugs for their consolidation therapy. Patients with high-risk AML, based on their prognostic factors, receive more aggressive therapy, such as allogeneic stem cell transplantation.
Not all patients can tolerate intensive therapies or even want them. Patients whose comorbidities and performance status make them poor candidates for intensive chemotherapy may still be able to participate in clinical trials. Or they may benefit from lower-intensity therapies which may relieve symptoms, improve quality of life and potentially extend survival.
The third phase of treatment is called “maintenance.” The main objective of maintenance therapy is to deliver a less toxic therapy to prevent relapse after intensive chemotherapy. Maintenance therapy is often an extended course of treatment. Not everyone with AML will receive maintenance therapy. It depends on the subtype, consolidation treatment and risk of relapse.
For some adult patients, the doctor may prescribe azacitidine (Onureg®) as maintenance therapy.
AML cells can spread to the cerebrospinal fluid, the fluid around the brain and spinal cord. This is uncommon, occurring in less than 5 percent of AML patients. Because CNS involvement is rare in cases of AML, doctors often do not test for it at the time of diagnosis unless the patient is experiencing neurologic symptoms, such as headache or confusion. If neurologic symptoms are present, the doctor may order imaging tests and/or a lumbar puncture to determine if there are leukemia cell in the spinal fluid.
Treatment for CNS involvement
If leukemia cells are found in the spinal fluid, intrathecal chemotherapy is administered, a treatment in which chemotherapy drugs are injected directly into the spinal fluid.
Find a list of standard drugs and drugs under clinical study to treat AML in the booklet on this page.
