Pediatric acute myeloid leukemia (AML) overview

Although acute myeloid leukemia (AML) is more common in adults, children can also develop the disease. Pediatric AML affects blood-forming cells in the bone marrow and may progress quickly, which is why treatment often begins soon after diagnosis.

Children with AML are cared for by specialized pediatric cancer teams that understand the unique medical, emotional and developmental needs of children and families navigating treatment. NMDPSM offers educational resources, transplant support information and guidance to help support families throughout their AML journey.

Signs and symptoms of pediatric AML

Many symptoms of pediatric AML occur because the body is unable to make enough healthy blood cells. Symptoms can appear quickly and may vary from child to child.

Symptoms of pediatric AML may include:

Fatigue or weakness: Low red blood cell counts can make children feel unusually tired or weak.
Frequent infections: AML can affect healthy white blood cells, making it harder for the body to fight infections.
Fever: Persistent or unexplained fevers are common in children with AML.
Easy bruising or bleeding: Low platelet counts may cause bruising, nosebleeds or bleeding gums.
Bone or joint pain: Leukemia cells can build up inside the bone marrow and cause discomfort or pain.

Pale skin: Anemia caused by low red blood cell counts may cause children to appear pale.
Shortness of breath: Reduced oxygen-carrying blood cells may make physical activity more difficult.
Petechiae: Tiny red, purple, or brown spots (depending on skin tone), under the skin caused by bleeding from small blood vessels. This is often referred to as “leukemia rash.”
Swollen gums: In some cases, abnormal blood cells may build up in the gums and cause swelling or tenderness.
Loss of appetite or weight loss: Some children may eat less or lose weight during illness.

Because many AML symptoms can resemble common childhood illnesses, it’s important to speak with a doctor if symptoms persist, worsen or seem unusual.

Testing for and confirming a pediatric AML diagnosis

Doctors use several tests to diagnose AML in children and better understand the specific subtype of leukemia. These tests help guide treatment planning and determine whether additional treatments, including blood or marrow transplant, may be recommended.

Testing can include:

Blood tests
Bone marrow biopsy or aspiration
Genetic and molecular testing
Imaging tests
Lumbar puncture (spinal tap)
Human leukocyte antigen (HLA) typing for transplant planning

Your child’s health care team will explain which tests are needed and what the results may mean for treatment.

Treating pediatric AML

Treatment for pediatric AML is based on factors such as the child’s age, AML subtype and response to treatment. Children with AML are treated by specialized pediatric cancer teams that closely monitor growth, development and long-term health throughout treatment.

Treatment options for pediatric AML may include:

Chemotherapy
Blood or marrow transplant (BMT)
Clinical trials

Chemotherapy uses powerful medicines to destroy leukemia cells and help the body begin producing healthy blood cells again. It is often one of the first treatments used for pediatric AML and may begin soon after diagnosis.

Children receiving chemotherapy are closely monitored by their health care team to manage side effects, monitor blood counts and support recovery throughout treatment.

Chemotherapy may be used:
- As the first treatment after diagnosis
- Before a blood or marrow transplant
- After transplant to help reduce the risk of relapse
- Alongside targeted therapies or clinical trials
Side effects vary depending on the medicines used and how a child’s body responds to treatment.

Common side effects of chemotherapy include:
- Fatigue
- Nausea or vomiting
- Hair loss
- Increased risk of infection
- Easy bruising or bleeding
- Mouth sores
- Loss of appetite
The health care team can help manage side effects and provide supportive care throughout treatment.
A blood or marrow transplant (BMT), also known as a bone marrow transplant, blood stem cell transplant or SCT, may be recommended for some children with AML as part of their treatment plan.

For pediatric AML, doctors most commonly use an allogeneic transplant, which uses healthy blood stem cells from another person. A transplant replaces unhealthy blood-forming cells with healthy donor cells and reduces the risk of leukemia returning.

If your child needs blood stem cells from an unrelated donor, their medical team works with NMDP to access a network of donor registries worldwide to find the right donor for your child.

NMDP provides educational resources, support services and guidance to help patients, families and caregivers navigate AML treatment and transplant. Research initiatives such as NMDP Donor for All are also helping expand access to BMT for more children.

Learn more about BMT for AML
Clinical trials help researchers study new treatments and improve existing therapies for pediatric AML. Some clinical trials explore new medicines, while others focus on improving treatment outcomes or reducing side effects.

Participation in a clinical trial is always voluntary, and patient safety is closely monitored throughout the process. You can talk with your child’s health care team to better understand whether a clinical trial may be appropriate.

NMDP’s Patient Support Center can help families better understand available clinical trial options and eligibility requirements.

Explore clinical trial support

How treatment decisions are made for pediatric AML

Treatment plans for pediatric AML are based on several factors related to a child’s diagnosis and overall health.

Doctors may consider:

The AML subtype
Genetic and molecular testing results
The child’s age and overall health
Response to initial treatment
Risk of relapse
Whether a suitable donor is available for transplant

Your child’s health care team can help explain treatment options, possible side effects and long-term considerations so your family can make informed decisions about care.

Long-term effects and recovery after treatment

Some AML treatments may cause long-term side effects that can affect a child’s health and development over time. Follow-up care helps doctors monitor recovery and manage possible complications.

Long-term considerations may include:

Growth and development changes
Fertility concerns
Bone or joint health issues
Organ function monitoring
Learning or concentration difficulties
Emotional and mental health support
Graft-versus-host disease (GVHD) after transplant

Every child’s recovery journey is different, and health care teams work closely with families to support long-term health after treatment.

Support for children and families facing AML

NMDP offers educational resources, transplant support information and guidance to help children, parents, caregivers and families navigate pediatric AML treatment and recovery.

NMDP can help by: