Last Updated on November 20, 2025 by Ugurkan Demir

Acute Myeloid Leukemia (AML) is a rare but serious blood cancer that affects children. It needs quick and effective treatment. At Liv Hospital, we focus on giving complete care and helping families with the latest treatments for pediatric AML.
Choosing the right treatment for childhood AML is key for a good life and survival. Our team works with families to find the best treatment. We consider the child’s health and the disease’s details.
We have many treatments, like chemotherapy, targeted therapy, and stem cell transplantation. These are important for managing AML leukemia in children.

Childhood acute myeloid leukemia is a rare but aggressive cancer. It affects the bone marrow and blood. Finding and treating it quickly is key.
Understanding pediatric AML is vital for new treatments. The disease often comes with genetic changes that affect treatment. We will dive into these details.
Pediatric AML is a big part of childhood leukemia. It happens more in some age groups. Doctors use several tests to diagnose it.
AML symptoms in kids can be hard to spot early. They might feel tired, pale, bleed easily, or get sick often. This is because their bone marrow isn’t working right.
| Diagnostic Criteria | Description |
| Morphological Analysis | Examination of bone marrow and blood smears to identify blast cells |
| Immunophenotyping | Identification of specific cell surface markers to classify leukemia cells |
| Cytogenetic Analysis | Detection of chromosomal abnormalities that may influence prognosis |
| Molecular Genetic Testing | Identification of genetic mutations that can guide targeted therapy |
Recent years have brought big changes in treating pediatric AML. Chemotherapy, targeted therapies, and care support have all improved. Now, 65-70% of kids with AML survive five years.
We’ve learned a lot about AML’s genetics. This knowledge has led to treatments that work better for certain patients.
Now, treatments are more tailored to each child. Doctors adjust the intensity based on the child’s risk level. This makes care more personal.

Children with AML get chemotherapy that targets leukemia cells hard. It also tries to lessen side effects. The treatment is complex and uses many chemotherapy agents in different phases.
We’ll talk about the two main phases of chemotherapy for AML in kids: induction and consolidation. Knowing these phases helps us see how AML is treated fully.
The induction phase is the first part of chemotherapy. It aims to get rid of as many leukemia cells as it can. This lets the bone marrow work normally again.
During this phase, kids get a mix of chemotherapy drugs. These might include:
These drugs are given in strong cycles. The exact mix depends on the treatment plan. Doctors watch how well the treatment works with bone marrow biopsies and blood tests.
They check for:
After getting remission from induction, consolidation therapy starts. It aims to get rid of any leukemia cells that might not be found. This phase is key to lowering the chance of relapse and improving survival chances.
Consolidation therapy often uses high-dose chemotherapy. Sometimes, it’s followed by a stem cell transplant. The choice of treatment depends on the child’s risk, leukemia genetics, and how they did in the first treatment.
| Therapy Phase | Primary Goals | Common Chemotherapy Agents |
| Induction | Achieve remission, reduce leukemia cells | Cytarabine, Anthracyclines, Etoposide |
| Consolidation | Eliminate remaining leukemia cells, prevent relapse | High-dose Cytarabine, other agents based on risk category |
Supportive care is very important during both phases. It helps manage side effects and toxicity. This includes preventing infections, supporting nutrition, and helping with mental health.
Understanding how pediatric AML is treated helps us see the full effort to fight this disease. Research keeps working to make treatments better, aiming to help more kids with AML.
Targeted therapy has changed how we treat pediatric Acute Myeloid Leukemia (AML). It focuses on specific genetic mutations. This method has greatly improved outcomes for kids with AML, thanks to treatments that target specific genetic changes.
We now tailor treatments to each patient’s genetic makeup. This makes treatments more effective and less harsh. The growth of targeted therapies comes from better genetic testing and understanding AML’s molecular roots.
FLT3 mutations are common in AML, found in 15-20% of kids. FLT3 inhibitors aim to tackle these mutations. Studies show they help patients with FLT3-mutated AML a lot.
Midostaurin and gilteritinib are FLT3 inhibitors approved for AML. They block the FLT3 tyrosine kinase, stopping the growth and survival of AML cells.
| FLT3 Inhibitor | Mechanism of Action | Clinical Benefit |
| Midostaurin | Inhibits FLT3 tyrosine kinase | Improved overall survival in FLT3-mutated AML |
| Gilteritinib | Potent inhibition of FLT3 and other kinases | High response rates in relapsed/refractory FLT3-mutated AML |
IDH1 and IDH2 mutations are found in some AML patients. IDH1/IDH2 inhibitors target these mutations. Early trials show they’re promising.
Ivosidenib and enasidenib are IDH inhibitors for AML patients with these mutations. They reduce 2-HG levels, helping leukemic cells differentiate.
Other targets like KIT, WT1, and CD33 are being explored in AML. Therapies for these targets are in various stages, from trials to approved treatments.
Targeted therapy in pediatric AML is a big step forward. It offers new options for patients with specific genetic mutations. As we learn more about AML’s genetics, we’ll see even better treatments.
Hematopoietic stem cell transplantation (HSCT) is a key treatment for kids with acute myeloid leukemia (AML). It replaces the bone marrow with healthy stem cells. These can come from the patient or a donor.
There are different HSCT options for kids with AML:
Deciding on HSCT depends on several things:
We look at these factors to find the best treatment for each child with AML.
After HSCT, it’s important to watch for complications:
Good post-transplant care is key to the best outcomes for kids with AML after HSCT.
Immunotherapy is becoming key in treating AML in kids. It uses the body’s immune system to fight cancer cells. This method is promising for better treatment results.
Antibody-based therapies use monoclonal antibodies to target leukemia cells. These antibodies help the immune system destroy cancer cells or deliver drugs to them.
Examples of antibody-based therapies include:
Cellular immunotherapies use engineered cells, like T cells, to fight cancer. CAR-T cell therapy is a notable example. It modifies T cells to target specific proteins on AML cells.
Immune checkpoint inhibitors help the immune system fight cancer more effectively. They are being explored for AML treatment. This could offer a new treatment strategy.
| Type of Immunotherapy | Mechanism of Action | Potential Benefits |
| Antibody-Based Therapies | Target specific proteins on leukemia cells | Precise targeting of cancer cells, reduced harm to healthy cells |
| Cellular Immunotherapies | Engineered cells recognize and attack cancer cells | Potential for long-term cancer control, adaptability to different cancer types |
| Immune Checkpoint Inhibitors | Release brakes on the immune system | Enhanced immune response against cancer, possible combination therapies |
Immunotherapy is a big step forward in treating pediatric AML. It brings new hope for kids with this tough disease. As research keeps improving, we’ll see more innovations in immunotherapy.
Precision medicine is changing how we treat AML leukemia in kids. It makes treatments fit each child’s genetic makeup. This has led to better results by giving kids the right treatments for their needs.
Genetic testing is key in treating pediatric AML. It looks at the genes of leukemia cells to find mutations. This helps doctors pick the best treatments for each child’s disease.
Genetic testing leads to treatments that fit each child. Knowing the AML’s genetics helps doctors choose the best options. This approach lowers side effects and boosts treatment success.
Biomarkers are becoming more important in AML treatment. They help predict how well treatments will work and track the disease. New biomarkers are being found, which could lead to even better treatments for kids with AML.
Supportive care is key in treating pediatric AML. It makes life better for young patients. We focus on many areas of care to help them do well.
Children with AML face a big risk of getting sick. We must prevent infections to keep them safe.
When infections do happen, we treat them fast. This includes strong antibiotics and sometimes antifungals or antivirals.
| Infection Prevention Measure | Description | Benefits |
| Prophylactic Antibiotics | Administering antibiotics to prevent infections | Reduces risk of bacterial infections |
| Environmental Precautions | Isolating patients from infection sources | Minimizes exposure to pathogens |
Nutrition is a big part of caring for kids with AML. Good food helps them stay strong and healthy while they’re sick.
We work together with dietitians, nurses, and doctors. They help make a special diet plan for each child.
Psychosocial support is vital for kids with AML and their families. It includes counseling and support groups.
This support helps lessen the emotional burden of AML treatment. It makes life better for everyone involved.
New and experimental treatments are being looked into for kids with AML leukemia. These new methods aim to make treatments better, cut down on side effects, and help more kids survive.
Epigenetic modifiers are drugs that work on how genes are turned on or off. They can change how cancer cells grow. This might help kill cancer cells or make them work right again.
Decitabine is one such drug. It’s being tested in kids with AML. Early signs show it might work well with other treatments, giving kids new hope.
“The use of epigenetic modifiers represents a significant shift in the treatment paradigm for AML, showing promise for better results in kids.”
Pediatric Oncologist
Combining new drugs with old ones is another approach. For kids with AML, this might mean mixing targeted drugs with chemo or stem cell transplants.
| Treatment Combination | Potential Benefits |
| FLT3 inhibitors + chemotherapy | Enhanced efficacy against FLT3-mutated AML |
| Epigenetic modifiers + targeted therapy | Potential to overcome resistance mechanisms |
| Immunotherapy + hematopoietic stem cell transplantation | Improved graft-versus-leukemia effect |
MRD-directed therapy tailors treatment based on how much cancer is left after first treatment. It helps find who needs stronger or different treatments.
Using MRD tests like flow cytometry or molecular testing gives important clues. For kids with AML, this therapy is being tested to better match treatments to each child’s needs.
As research moves forward, new and experimental treatments look promising for kids with AML. Ongoing trials and research are key to making these new methods safe and effective.
Recent advances in treatment and care have greatly improved life for kids with acute myeloid leukemia. We’ve talked about different treatments like standard chemotherapy, targeted therapy, and stem cell transplants. We’ve also looked at immunotherapy.
It’s key to give kids with AML a full care plan from many doctors. This helps improve their treatment results and life quality. We need to keep researching and finding new ways to help these kids.
Using precision medicine and looking at a patient’s genes can help find the best treatments. As we go on, we must keep researching and working together. This will help us give kids with AML the best care and improve their chances of survival.
AML (Acute Myeloid Leukemia) is a rare but serious blood cancer in kids. It needs quick and effective treatment.
Kids with AML might feel tired, have pale skin, get infections easily, bruise, and bleed. Symptoms can vary.
Doctors use a physical check-up, blood tests, bone marrow biopsy, and imaging to find cancer cells.
Kids with AML can get chemotherapy, targeted therapy, stem cell transplant, and supportive care. The goal is to get into remission and improve survival chances.
Chemotherapy is key in treating AML in kids. It aims to get the cancer into remission. There are many chemotherapy plans.
Targeted therapy uses drugs that target specific genetic changes in the cancer. It offers a personalized treatment.
This treatment replaces the child’s bone marrow with healthy stem cells. It can be from a donor or the child. It helps make normal blood cells again.
Supportive care is vital. It includes preventing infections, helping with nutrition, and supporting the child’s mental health. It helps manage treatment side effects and improves life quality.
Precision medicine tailors treatment to the child’s genetic makeup. It uses genetic testing to guide treatment and improve results.
New therapies like immunotherapy, epigenetic modifiers, and combination treatments are being studied. They aim to better treatment results and survival rates for kids with AML.
Thanks to better treatments like chemotherapy, targeted therapy, and stem cell transplant, the five-year survival rate for kids with AML has greatly improved.
Childhood AML is different from adult AML in its biology and treatment needs. It requires special treatments for kids.
https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq
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