Acute myeloid leukemia (AML)

Childhood Cancer Name:

Acute Myeloid Leukemia (AML)

Disclaimer:

The information below is for general education only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always talk with your child’s doctor or another qualified healthcare provider about any questions you have. Feel free to share this page with them.

Explanation:

Acute Myeloid Leukemia (AML) is a fast-moving cancer that starts in the bone marrow from immature myeloid blood cells — the precursors of red blood cells, platelets, and several kinds of white blood cells. In AML these early myeloid cells stop maturing normally and instead grow and multiply uncontrollably, crowding out the marrow and spilling abnormal cells into the bloodstream. Because the disease disrupts normal blood production, children with AML commonly develop symptoms from low red blood cells (tiredness, pallor), low platelets (easy bruising or bleeding), and low normal white blood cells (infections). The biology of pediatric AML is diverse: many cases are driven by identifiable chromosomal changes or gene mutations, and these molecular features affect prognosis and treatment choices. Diagnosis and initial treatment are urgent because AML progresses quickly, but modern, risk-adapted therapies delivered at experienced pediatric centers can induce high rates of remission. Cancer Research UK.  PMC

Characteristics:

Pediatric AML is less common than other acute childhood leukemias but is usually more aggressive. Symptoms tend to arise quickly over days to weeks, and because the disease is biologicly diverse, outcomes vary by subtype and initial response to therapy. Most children achieve a remission after induction chemotherapy, but longer-term event-free and overall survival depend heavily on molecular risk features and on whether a stem-cell transplant is recommended.

Because of this heterogeneity, care is individualized: two children with AML can receive very different treatments depending on their genetic tests and early treatment response.

Cancer Localization:

Although AML is fundamentally a marrow and blood disease, leukemia cells can be found beyond the marrow and cause organ-specific signs. The common sites and patterns to know about are:

• Bone marrow and peripheral blood — this is the primary site and the basis for most symptoms and tests.

• Liver and spleen — these organs often enlarge when leukemia cells infiltrate them.

• Skin and gums — some subtypes show skin nodules or gum overgrowth.

• Central nervous system — less commonly involved at diagnosis, but evaluated when suspected because it changes treatment (lumbar-puncture testing).

Childhood Cancer Diagnostic Methods:

Diagnosis starts with blood tests that typically show low red cells, low platelets, and circulating blasts. The definitive test is a bone-marrow aspirate and biopsy: a hematopathologist examines the cell appearances and identifies the genetic abnormalities and mutations that stratify risk. A lumbar puncture is performed if there is clinical concern for CNS disease. Baseline heart, liver, and kidney tests are also obtained to plan safe drug dosing.

Childhood Cancer Treatment:

Treatment is intensive and occurs in phases: induction (to induce a marrow remission), consolidation/intensification (to remove residual disease), and, for many high-risk patients, consideration of allogeneic hematopoietic stem-cell transplant (HSCT). Induction regimens most commonly combine cytarabine with an anthracycline such as daunorubicin; additional agents (for example etoposide) are used depending on the protocol. Supportive care — infection prevention and rapid treatment, transfusion support, and metabolic monitoring — is essential throughout therapy. For specific molecular subgroups, targeted drugs have become part of standard practice: for example, gemtuzumab ozogamicin (an anti-CD33 antibody-drug conjugate) is used in some pediatric protocols and FLT3 inhibitors are added for FLT3-mutated disease. For relapse or refractory AML, options include clinical trials of novel agents, antibody-drug conjugates, bispecific antibodies, and early-phase cellular therapies such as CAR-T directed at myeloid antigens; stem-cell transplant is often discussed for eligible patients. Because treatment is intense and can be life-threatening if complications occur, care at an experienced pediatric oncology center following cooperative-group protocols (Children’s Oncology Group or international consortia) is recommended.