Treatment of Children with Acute Myeloid Leukemia (AML)
The main treatment for children with AML is chemotherapy. Because of the intensity of treatment and the risk of serious complications, children with AML need to be treated in cancer centers or hospitals that have experience with this disease.
Phases of chemotherapy for AML
Treatment of most children with acute myeloid leukemia (AML) is divided into 2 main phases of chemotherapy:
The goal of induction chemotherapy is to achieve a remission. This means that leukemia cells are no longer found in bone marrow samples, the normal marrow cells return, and the blood counts improve. A remission is not necessarily a cure. More than 85% of children with AML enter remission after induction.
The chemo drugs most often used to treat AML early are cytarabine (ara-C), and anthracycline drugs (daunorubicin is used most often). Chemo treatments for AML are intense and cause very low blood counts. Children with AML often stay in the hospital during chemo and after, while the blood counts are low. Sometimes side effects from treatment can be serious enough to be life-threatening, but in recent years, advances in supportive care (nursing care, nutrition, antibiotics, red blood cell and platelet transfusions as needed, etc.) have made these much less common than in the past.
Some children with AML may get a dose of the targeted drug gemtuzumab ozogamicin (Mylotarg) along with chemo as part of their induction treatment. AML with FLT3 gene changes may get targeted therapy as well with drugs like sorafenib (Nexavar).
If the doctors think that the leukemia may not respond well to just 2 chemo drugs alone, they may add another chemo drug such as etoposide or 6-thioguanine.
Treatment with these chemo drugs is repeated until the bone marrow shows no more leukemia cells. This usually occurs after 2 or 3 cycles of treatment.
Preventing relapse in the central nervous system: Most children with AML will also get intrathecal chemotherapy (given directly into the cerebrospinal fluid, or CSF) to treat or help prevent leukemia from relapsing in the brain or spinal cord. Radiation therapy to the brain is used less often.
Consolidation (intensification) begins after the induction phase. The purpose is to kill any remaining leukemia cells by using more intensive treatment.
Some children have a sibling who would be a good stem cell donor. For these children, a stem cell transplant might be recommended once the leukemia is in remission, especially if the AML has some poorer prognostic factors. Most studies have found this improves the chance for long-term survival over chemo alone, but it is also more likely to cause serious complications. For children with good prognostic factors, some doctors may recommend more cycles of intensive chemotherapy, and reserve the stem cell transplant in case the AML relapses.
Chemotherapy drugs used in this treatment phase may include etoposide, cytarabine, or anthracycline drugs (such as daunorubicin or mitoxantrone).
Intrathecal chemo (into the CSF) often continues during this phase of treatment.
Unlike treatment for ALL, maintenance chemo is not needed for most children with AML.
Treating refractory or recurrent AML
In some cases, AML may not respond to the usual treatments. In this case, doctors may find leukemia on bone marrow or blood tests after treatment has started that does not go away. Sometimes, this is called minimal residual disease (MRD), which means special lab tests must be used to find small amounts of leukemia. Other times, leukemia cells can be seen under the microscope on standard lab tests.
When leukemia cells never completely go away, this is called refractory AML. When the leukemia goes away during treatment and then recurs (comes back), this is called recurrent (or relapsed) AML.
Less than 10% of children have refractory AML (leukemia that does not respond to initial treatment). These leukemias are often very hard to cure, and doctors may recommend a stem cell transplant, if it can be done.
Generally, the outlook for a child whose AML relapses (comes back) after treatment is slightly better than if the AML never went into remission, but this depends on how long the initial remission was. In more than half of cases, relapsed leukemia can be put into a second remission with more chemo. The chance of getting a second remission is better if the first remission lasted for at least a year, but long-term second remissions are rare without a stem cell transplant. Many different combinations of standard chemo drugs have been used in these situations, but the results have been mixed.
Another option for some children with refractory or recurrent AML is treatment with targeted drugs such as gemtuzumab ozogamicin (Mylotarg) or venetoclax (Venclexta). If the leukemia cells have a KMT2A gene change, treatment with a targeted drug called a menin inhibitor, such as revumenib (Revuforj), might be an option.
Most children whose leukemia has relapsed are good candidates for clinical trials testing new treatment regimens. The hope is that remission can be achieved so that a stem cell transplant can be considered. Some doctors may advise a stem cell transplant even if there is no remission. This can sometimes be successful.
Treating children with acute promyelocytic leukemia (APL)
Treatments used for acute promyelocytic leukemia (APL), a subtype of acute myeloid leukemia (AML), differs from the usual AML treatment. Similar to other forms of AML, there are two main phases of treatment. These are called induction and consolidation.
During induction, children with APL typically respond very well to targeted drugs called arsenic trioxide (ATO) and all-trans retinoic acid (ATRA). This phase lasts at least 4 weeks, sometimes longer if the cancer cells are still detected in the blood. In children with white blood cell counts of less than 10,000 at diagnosis, called standard-risk APL, treatment with these drugs alone often results in remission. In high-risk APL, these drugs are used in combination with chemo (usually anthracycline drugs like daunorubicin or cytarabine).
Rarely, APL can spread to the cerebrospinal fluid (CSF). Lumbar punctures, when needed, are often delayed until any bleeding risk in the child is improved. Children with leukemia cells in the CSF will receive treatment with triple intrathecal therapy during this early phase of treatment. This treatment is a combination of cytarabine, hydrocortisone and methotrexate given into the CSF by a lumbar puncture. These treatments continue twice weekly for 2 to 3 weeks.
Consolidation is similar to induction, where ATO and ATRA are the main drugs used. The schedule of how these drugs are given is a little different than in the first phase of treatment. Because of how well this treatment works, stem cell transplant is not typically needed.
Treating recurrent APL
If the leukemia comes back after treatment, it can be treated. ATO, ATRA, and gemtuzumab may be options for treatment. A stem cell transplant may be considered once a second remission is achieved.
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- References
Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).
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Kutny MA, Alonzo TA, Abla O, et al. Assessment of Arsenic Trioxide and All-trans Retinoic Acid for the Treatment of Pediatric Acute Promyelocytic Leukemia: A Report From the Children's Oncology Group AAML1331 Trial. JAMA Oncol. 2022;8(1):79-87.
National Cancer Institute. Childhood Acute Promyelocytic Leukemia Treatment (PDQ). 2024. Accessed at https://www.cancer.gov/types/leukemia/hp/child-aml-treatment-pdq/childhood-apl-treatment-pdq on May 7, 2025.
Last Revised: July 22, 2025
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