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Childhood Cancer Types — Parent’s Guide
Acute myeloid leukemia (AML) in children — what parents need to know
Medically reviewed by Dr. C. Raghavendra Reddy, DM (Medical Oncology, Gold Medal) · Last reviewed June 2026
If your child has just been told they have acute myeloid leukemia — or if a blood test has raised concern and you are trying to understand what AML means — this page is written for you. AML in children is a cancer that starts in the bone marrow, the tissue that makes all blood cells. It is less common than other childhood leukaemias, but specialist centres treat it every day. Understanding the diagnosis, what to expect during treatment, and how decisions are made is the first step in walking this journey with your child.
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Understanding childhood AML
AML in children — the subtypes and what they mean
Acute myeloid leukemia (AML) is not a single disease. Modern diagnosis looks at the specific genetic and molecular features of the leukaemia cells to classify it into subtypes. These subtypes guide the treatment plan and help the medical team understand how the disease is likely to behave. Here is what each major category means for your child.
Lower-risk group
AML with Favourable Genetic Changes
Some children with AML have specific chromosomal or genetic changes in their leukaemia cells that are associated with a better response to chemotherapy. The bone marrow test at diagnosis identifies these changes. When a favourable genetic profile is confirmed, the treatment team can sometimes achieve sustained remission with chemotherapy alone, without the need for a stem cell transplant in the first round of treatment. Identifying this group is one of the most important reasons why thorough genetic testing is done at diagnosis rather than starting treatment immediately based on the blood count alone.
- Specific genetic changes identified on bone marrow testing
- Chemotherapy alone may achieve durable remission
- Stem cell transplant may be deferred unless disease returns
Higher-risk group
AML with High-Risk Genetic Features
Other children with AML carry genetic changes that are associated with a higher likelihood that the disease will be harder to eliminate with standard chemotherapy, or that it may return after initial treatment. For children in this group, the CION tumour board typically recommends a more intensive treatment approach. This often includes consolidation with a stem cell transplant — using bone marrow or peripheral blood stem cells from a matched sibling or an unrelated matched donor — as the best way to reduce the risk of the disease coming back. The genetic profile does not predict the outcome for a specific child; it informs the treatment recommendation.
- Genetic changes suggest higher relapse risk with standard chemotherapy
- Stem cell transplant often recommended in consolidation
- Donor search begins early so there is no delay when the time comes
Special population
AML in Children with Down Syndrome
Children with Down syndrome (trisomy 21) have a substantially higher risk of developing AML, particularly before the age of four. Interestingly, AML arising in children with Down syndrome — particularly a form known as AMKL (acute megakaryoblastic leukaemia) — tends to be highly responsive to standard chemotherapy and is treated with a specific protocol that uses lower doses than those used in children without Down syndrome. This adjusted approach is important because children with Down syndrome are more sensitive to certain side effects of intensive chemotherapy. A specialist with experience in this specific population is essential.
- Higher incidence and different biology compared to AML in other children
- Highly responsive to lower-intensity chemotherapy protocols
- Requires specialist experience with this specific patient group
If disease returns
Relapsed or Refractory Childhood AML
When AML does not respond to initial treatment (refractory AML) or returns after a period of remission (relapsed AML), the medical team's approach shifts. Salvage chemotherapy using alternative drug combinations is given to try to achieve a second remission, and a stem cell transplant is almost always part of the plan for children who achieve remission again. Clinical trials may also be available at this stage, offering access to newer treatment approaches that are being studied. We discuss all available options honestly at the CION tumour board and we explain them clearly to the family — with time, not in a rushed corridor conversation.
- Salvage chemotherapy aims to achieve a second remission
- Stem cell transplant is the standard consolidation after second remission
- Clinical trial participation may be discussed at this stage
AML is one type of leukaemia in children. For a broader overview of all types of childhood leukaemia, see our childhood leukemia overview page. For information about disease monitoring after treatment, see our page on minimal residual disease (MRD) in leukemia.
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The diagnosis and treatment journey
How childhood AML is diagnosed and treated — step by step
Every family facing a childhood AML diagnosis wants to know what happens next. Here is an honest, plain-language account of the diagnostic and treatment steps — so you understand what is coming and why each step is done.
Blood count and blood film — the first alert
A diagnosis of AML in children almost always begins with a full blood count (CBC). The CBC shows how many red blood cells, white blood cells, and platelets are in the blood. In AML, one or more of these numbers is typically abnormal. The blood film — where a drop of blood is spread on a glass slide and examined under a microscope — may show immature myeloid cells called blasts, which do not belong in circulating blood. These findings do not confirm AML on their own, but they immediately trigger the next step. No child should wait weeks for this investigation once symptoms such as unexplained pallor, bruising, or prolonged fever are present.
Bone marrow aspirate and biopsy — the definitive test
The definitive test for AML is the bone marrow aspirate and biopsy, performed under sedation or general anaesthesia so the child is comfortable throughout. A small sample of bone marrow is taken from the back of the hip bone (iliac crest). This sample is examined under a microscope to count the percentage of blast cells (more than 20% confirms AML), typed using immunophenotyping (which identifies the specific cell line the leukaemia comes from), and tested for chromosomal and genetic changes using cytogenetics and molecular testing. The genetic results, which take several days, are the most important piece of information guiding the treatment plan. No treatment protocol is started without this information.
Lumbar puncture and additional imaging
A lumbar puncture (spinal tap) is performed at or around the time of the bone marrow test to examine the cerebrospinal fluid (CSF) that surrounds the brain and spinal cord. If leukaemia cells are found in the CSF, additional treatment directed to the central nervous system will be included in the protocol. Imaging — typically an ultrasound of the abdomen and sometimes a CT or MRI of the chest — is also done to look for leukaemia deposits outside the bone marrow (such as the spleen, liver, or lymph glands). Together, these investigations give the team a complete picture of the disease before any treatment starts.
Induction chemotherapy — the first and most intensive phase
Once the full diagnostic picture is available and the CION tumour board has reviewed the results, induction chemotherapy begins. The goal of induction is remission — meaning that leukaemia cells are no longer detectable on standard tests, and the bone marrow is regenerating normal blood cells. Induction for childhood AML is intensive, and children are admitted to hospital for most or all of this phase because their blood counts fall very low, making them vulnerable to infection and bleeding. The team supports the child with antibiotics, antifungal medicines, blood transfusions, and platelet transfusions as needed throughout induction. Most children with AML achieve remission after induction, though the disease is not yet fully eradicated at this point.
Consolidation — eliminating remaining disease
After induction achieves remission, one or more consolidation courses are given to eliminate the leukaemia cells that remain below the threshold of detection on standard tests. These courses are also intensive and require hospital admission, but they are generally shorter than induction. The number and nature of consolidation cycles depend on the child’s risk category (determined by the genetic profile identified at diagnosis) and on the result of a test called minimal residual disease (MRD) testing, which checks how much leukaemia is left at a very sensitive level after induction. For children in the high-risk category, consolidation usually includes a stem cell transplant from a matched donor. For lower-risk children, chemotherapy alone may be sufficient consolidation.
Follow-up, monitoring, and survivorship care
After consolidation is complete, the child is monitored closely in the follow-up period with regular blood counts and, in some cases, bone marrow tests to confirm that the disease remains in remission. Unlike ALL, AML does not have a long maintenance chemotherapy phase. The frequency of follow-up appointments decreases over time as the child remains in remission. The CION team also provides survivorship care — attention to the child’s growth, development, school reintegration, and psychological wellbeing after treatment ends. We believe care led by a team extends beyond the last chemotherapy dose. We walk this journey with you all the way through.
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Common questions
Your questions about AML in children — answered
What is acute myeloid leukemia (AML) in children?
Acute myeloid leukemia (AML) is a cancer of the blood-forming cells in the bone marrow. In a healthy child, the bone marrow produces myeloid cells that mature into red blood cells, platelets, and certain types of white blood cells (including neutrophils and monocytes). In childhood AML, one abnormal myeloid cell begins multiplying out of control, producing immature cells called blasts that do not mature and cannot perform the functions of normal blood cells. These blasts crowd out healthy marrow, which is why children with AML often have anaemia, a higher risk of bleeding, and a weakened ability to fight infection. AML is less common than ALL (Acute Lymphoblastic Leukaemia) in children, but it is generally considered more intensive to treat. The exact cause in most cases is not known — it is not anything the child or family did. A small number of cases are associated with known genetic conditions such as Down syndrome or certain inherited bone marrow disorders.
How is childhood AML different from childhood ALL?
ALL (Acute Lymphoblastic Leukaemia) and AML (Acute Myeloid Leukaemia) are both acute leukaemias — they develop rapidly and need prompt treatment — but they arise from different bone marrow cell lines and have different treatment strategies. ALL arises from lymphoid cells and is the more common childhood leukaemia; it is treated with multi-agent chemotherapy over two to three years, including a long maintenance phase. AML arises from myeloid cells and is treated with a shorter but more intensive course of chemotherapy — usually six to eight months — with no prolonged oral maintenance phase. Because of the intensity of AML treatment, children typically need hospital admission for prolonged periods during each cycle. Another key difference is that a stem cell transplant (bone marrow transplant) is more often recommended in AML than in ALL — particularly for children whose AML carries certain high-risk genetic features or does not respond well to the first round of treatment.
What are the signs and symptoms of AML in children?
The signs of AML in children arise because the bone marrow is failing to make enough healthy blood cells. The most common signs include: unusual pallor and extreme tiredness (from anaemia — too few red blood cells); easy bruising, prolonged bleeding from small cuts, or tiny flat red or purple spots on the skin (petechiae) caused by a low platelet count; frequent or severe infections that take longer than expected to clear (from reduced healthy white blood cells); unexplained fever that persists without an obvious infection; bone or joint pain, particularly at night; and in some children, swollen lymph glands, a tender abdomen, or swelling around the eyes (called chloroma or myeloid sarcoma). These symptoms are not unique to AML — they are caused by many common and treatable conditions — but when several are present together, or when any one of them does not resolve within two to three weeks, a doctor should evaluate the child with a full blood count and blood film.
How is AML diagnosed in children?
Diagnosis of AML in children begins with a full blood count (CBC) and blood film. The CBC typically shows abnormal numbers of white blood cells (which may be very high, normal, or even low), anaemia, and low platelets. The blood film may reveal immature myeloid cells (blasts). These findings raise suspicion, but definitive diagnosis requires a bone marrow aspirate and biopsy — a procedure performed under sedation or general anaesthesia that takes a small sample of marrow from the back of the hip bone. The marrow sample is examined under a microscope, typed with immunophenotyping (to confirm it is myeloid leukaemia rather than lymphoid), and tested for genetic and chromosomal changes. These genetic results are critical — they classify the AML into subtypes that guide both the treatment protocol and the assessment of risk. A lumbar puncture (spinal tap) is also performed to check whether leukaemia cells have reached the fluid around the spinal cord and brain.
What does treatment for childhood AML involve?
Treatment for childhood AML is intensive and is typically divided into two or three phases. The first phase, induction, aims to bring the disease into remission — meaning leukaemia cells are no longer detectable on standard tests. This phase usually requires a prolonged hospital stay because the intensive chemotherapy also temporarily suppresses the bone marrow, making the child very vulnerable to infection and bleeding. Once remission is achieved, subsequent consolidation courses are given to eliminate any remaining leukaemia cells that may not be visible. The number of consolidation cycles depends on the child's risk category, which is determined by the specific genetic features of the AML at diagnosis and how well the disease responded to induction. For children classified as high risk, or for those who relapsed after their first treatment, a stem cell transplant may be recommended as part of consolidation. Throughout treatment, the CION team provides full supportive care — including infection management, blood and platelet transfusions, and nutritional support — alongside the oncology treatment itself.
Will my child need a bone marrow transplant for AML?
Not every child with AML requires a bone marrow (stem cell) transplant. Whether a transplant is recommended depends on several factors: the specific genetic subtype of the AML, how quickly and completely the disease responded to induction chemotherapy, whether any minimal residual disease is detectable after initial treatment, and whether the disease returns (relapses) after first-line chemotherapy. Certain subtypes of AML carry genetic changes that are associated with a favourable response to chemotherapy alone, and a transplant may not be needed for these children. For children with high-risk features — including specific genetic mutations that make the disease more resistant to chemotherapy — a transplant using cells from a matched sibling donor or an unrelated matched donor is often the recommended consolidation approach. The CION tumour board reviews each child's genetic report and treatment response data before making any recommendation. This decision is never made by one doctor alone.
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