Chemotherapy and Drug Therapy

About Chemotherapy

Chemotherapy drugs kill fast-growing cells throughout the body, including both cancer cells and normal, healthy cells.

Chemotherapy is typically given in cycles. Each cycle is made up of a certain number of days of treatment, followed by a certain number of days of rest. The rest days allow the body time to recover before the next treatment cycle begins.

Some chemotherapy drugs are given intravenously, meaning they are injected into a vein. During an intravenous (IV) infusion, the drugs are injected slowly over the course of a few hours, or several days in the case of a continuous infusion.

Phases of Treatment

Acute myeloid leukemia (AML) is often treated in two phases

  • Induction therapy
  • Consolidation (intensification) therapy

Induction Therapy

The goal of induction therapy is to destroy as many cancer cells as possible to achieve a remission. In patients with AML, remission means that there are less than 5 percent blasts in the bone marrow and that blood counts have returned to normal.

Children with AML often receive two rounds of chemotherapy. During the first round, children often stay in the hospital for 4 weeks to receive supportive care with IV antibiotics and frequent blood transfusions. During this time the doctor will order blood and bone marrow tests to see how well the treatment is working.

After blood cell counts recover, children may go home for a few days or a week and then return to the hospital for the second round of induction, followed by another 4 weeks of recovery in the hospital. The second round of induction therapy may contain the same drugs that were used in the first round, or it may be a new chemotherapy regimen.

The chemotherapy regimen used most during the induction phase in children with AML includes cytarabine and an anthracycline. Daunorubicin is the anthracycline most often used for this regimen, although idarubicin and mitoxantrone are sometimes used. If an anthracycline is given, your doctor may administer another drug, dexrazoxane (Totect®, Zinecard®), around the same time as the anthracycline. This drug is not a chemotherapy agent, but it helps to minimize cardiac side effects that are associated with anthracyclines. Other chemotherapy drugs may be added to the cytarabine and anthracycline, such as etoposide or thioguanine.

In addition to the chemotherapy, children may receive targeted therapies during induction. This may include:

  • Gemtuzumab ozogamicin (Mylotarg™) along with chemotherapy as part of their induction treatment.
  • An FLT3 inhibitor (for patients with FLT3 mutations) such as sorafenib (Nexavar®), gilteritinib (Xospata®) or midostaurin (Rydapt®)

Treatment for patients with acute promyelocytic leukemia (APL), the M3 subtype of AML, differs from other AML treatments. Click here to read about treatment for APL.

AML patients whose leukemia cells have certain genetic mutations are assigned a specific risk status. Talk to your doctor about treatments available to target specific genetic mutations. See the LLS fact sheet, Cancer Molecular Profiling.

For information about the drugs listed on this page, visit Drug Listings.

Treatment Response and Minimal Residual Disease (MRD)

Approximately 75 to 80 percent of children with AML achieve a remission by the end of induction therapy. However, even when a complete remission is achieved, some leukemia cells that cannot be seen with a microscope may remain in the body. This is referred to as minimal residual disease (MRD), also called measurable residual disease.

Patients who have achieved remission after initial treatment but have MRD are at increased risk of disease relapse. Testing for MRD can help the doctor re-evaluate your child’s AML risk category and determine whether they may benefit from receiving further treatment with more intensive therapies.

For patients who do not achieve remission after the first course of induction chemotherapy, additional courses of chemotherapy may be given, either with the same drugs or with a new chemotherapy regimen.

Central Nervous System (CNS) Prophylaxis

Pediatric treatment regimens typically include treatment to prevent the spread of leukemia cells to the brain and spinal cord, as well as kill any leukemia cells that may already be there. The CNS-directed therapy begins during the induction phase and continues throughout the rest of treatment.

Some form of intrathecal chemotherapy is now incorporated into most protocols for the treatment of childhood AML. “Intrathecal” means that the chemotherapy drugs are injected into the fluid-filled space between the thin layers of tissue that cover the brain and spinal cord.

Intrathecal chemotherapy can be combined with the other types of chemotherapy that are given during the induction phase of treatment. The most common intrathecal chemotherapy drug used in children with AML is cytarabine.

If AML cells are found in the CNS at the time of diagnosis, a more intensive CNS-directed therapy is used. In these cases, additional drugs are included in the intrathecal therapy, such as methotrexate and a corticosteroid.

Consolidation (Intensification) Therapy

Consolidation therapy refers to treatments given to patients after their disease is in complete remission. It is given to children with AML after they complete induction therapy. The goal of consolidation therapy is to eliminate the residual leukemia cells in the body.

There are two basic treatment options for consolidation therapy:

Patients with favorable risk factors are often given 2-3 cycles of intensive chemotherapy with high-dose cytarabine and other drugs for consolidation therapy. The number of chemotherapy cycles varies from patient to patient. They are often hospitalized during consolidation therapy. They are often hospitalized during consolidation therapy. They may go home for a few days or a week between cycles. Additionally, CNS prophylaxis usually continues during this phase. 

Based on their prognostic factors, patients with high-risk AML receive more aggressive therapy that may include allogeneic stem cell transplantation.

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