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While AML is primarily a disease of adult-onset, it can rarely occur in children. About 4.5% of all AML cases in the United States from 2013-2017 occurred in children less than 20 years of age. AML in children presents similarly as it does in adult. Click here to learn more about AML. The tests done to obtain an AML diagnosis is similar as well. Once diagnosed, AML needs to be treated right away with chemotherapy. Your child may be hospitalized for treatment soon after diagnosis. 

Risk Factors for Developing Childhood AML

It is currently unknown why children develop AML but there are some factors that put children at higher risk for the development of this cancer. These risk factors include:

  • Having a brother or sister, especially a twin, with leukemia
  • Being Hispanic
  • Being exposed to cigarette smoke or alcohol before birth
  • Having a personal history of aplastic anemia
  • Having a personal or family history of MDS
  • Having a family history of AML
  • Past treatment with chemotherapy or radiation therapy
  • Being exposed to ionizing radiation or chemicals such as benzene
  • Having certain syndromes or inherited disorders, such as:
  • Down syndrome
  • Aplastic anemia
  • Fanconi anemia
  • Neurofibromatosis type 1
  • Noonan syndrome
  • Shwachman-Diamond syndrome
  • Li-Fraumeni syndrome

AML Treatment in Children

The treatment for childhood AML usually has two phases: induction therapy and consolidation therapy. Induction therapy is the first phase of treatment, and it aims to get rid of as many leukemia cells as possible. This ideally puts the AML into remission. The second phase, consolidation therapy, aims to get rid of any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse. Consolidation therapy may also consist of an allogeneic stem cell transplant. 

Induction therapy in children is often the same as in adults: cytarabine and an anthracycline (either daunorubicin or idarubicin). Heart and kidney function will be monitored closely as this regimen is given. If the child’s AML is CD33 positive, a drug called gemtuzumab ozogamicin (Mylotarg), may also be given alongside the other two drugs. 

One treatment intervention that is different in children than in adults is that children often receive central nervous system (CNS) prophylaxis in order to prevent the spread of leukemia cells to the central nervous system. To do this, intrathecal chemotherapy is used. This process administers chemotherapy drugs directly into the spinal fluid. 

There are two types of childhood AML that are treated differently:

  1. AML in children with Down Syndrome: Having Down Syndrome puts children at a 10-20 times higher risk for AML, but it’s been determined that they have better outcomes with a less intensive chemotherapy regimen. A lower-intensity regimen is often better tolerated by the child and has shown to produce good outcomes in this population.
  2. Acute promyelocytic leukemia (APL): Just like in adults, APL is treated with different drugs. APL has a specific genetic change that allows a drug called All-trans retinoic acid (ATRA) to be very effective. 


Childhood cancer can have long-lasting physical and emotional effects on both the child and the family. While children who survive AML can go on to have very productive and enjoyable lives, some additional, ongoing care is still beneficial. It is important for the child to visit a pediatrician or doctor at least once per year for a physical examination and bloodwork. The child should also be visiting an oncology clinic on a schedule that is recommended by his or her oncologist. The child and family should be informed of signs and symptoms to look out for that warrant a prompt visit to either their doctor or oncologist.

Some symptoms that your doctor may ask you to look out for and schedule a visit if they occur include:

  • Prolonged bleeding
  • Poor healing cuts 
  • Frequent infections
  • Mild fever
  • Swollen gums
  • Pale complexion
  • Abnormal fatigue

Long term effects of AML

There can be some lingering effects from treatment for months or even years after treatment is completed. The severity of these symptoms varies widely and some children might not have any long-term side effects at all. Due to the effects of anthracyclines, it’s important to have the child’s heart and kidney function monitored periodically.

Some examples of possible long-term effects include:

  • Thyroid dysfunction
  • Fatigue
  • Infertility
  • Hearing loss
  • A secondary cancer
  • Cognitive effects like difficulty learning, concentrating and fine motor coordination

It is also important to promote a healthy lifestyle by encouraging children to eat a wide variety of foods like fruit, vegetables, whole grains, high-quality proteins and healthy fats. Exercise, avoidance of smoking and regular doctor’s visits are also a part of a healthy lifestyle.   

Click here for a great resource discussing long-term follow-up recommendations.

Cancer Support

It may be helpful to seek out support groups or professional counseling if your child is struggling emotionally with their cancer diagnosis. Because of the rare nature of AML, especially in children, your child may never meet another person who has been through the same experience that they have. Connecting with others who they can relate to can help children cope with their past experiences and allow them to improve their overall mental fitness.  

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