Acute myeloid leukaemia in children: the decisions you may face
6 treatment settings, 6 with more than one named option. Each section lays out the options the standard of care names, what each is for, the trials behind them with their recorded results, the side effects and cautions on record, and questions to ask. Built from the cancer page's standard-of-care rows; nothing here is advice for your case.
Induction (two courses)
Cytarabine with daunorubicin or mitoxantrone and etoposide; gemtuzumab ozogamicin added to the first course for CD33-positive disease (AAML0531); MRD after course 1.
Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.
Daunorubicin is the anthracycline most often combined with cytarabine to bring acute myeloid leukaemia into remission; it is also part of induction for acute lymphoblastic leukaemia.
Mitoxantrone is a blue chemotherapy infusion used with cytarabine for acute myeloid leukaemia and, historically, to relieve pain in advanced prostate cancer; it is also licensed for multiple sclerosis.
Etoposide is a chemotherapy from the mayapple plant, essential to curing testicular cancer (BEP), treating small-cell lung cancer, lymphomas, childhood sarcomas and leukaemias, and used in transplant conditioning.
Gemtuzumab ozogamicin (Mylotarg) is an anti-CD33 antibody linked to the DNA-cutting payload calicheamicin, the first ADC approved, in 2000 for relapsed acute myeloid leukaemia. An unstable linker and no benefit in a confirmatory trial led to withdrawal in 2010; it returned in 2017 at a lower fractionated dose, with liver toxicity, including veno-occlusive disease, its defining risk.
Flow cytometry MRD counts leukaemia cells in the bone marrow one at a time by their surface proteins, down to one in ten thousand.
- Broadly applicable, fast (same day)
- Works without a molecular marker
- Tests Gemtuzumab ozogamicinNewly diagnosed AML, age 0-29: standard five-course chemotherapy with or without two doses of gemtuzumab ozogamicin
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- 3-year EFS 53.1% vs 46.9%, HR 0.83; relapse risk reduced.
3-year event-free survival (%): Chemotherapy + gemtuzumab ozogamicin 53.1 vs Chemotherapy 46.9 · HR 0.83 · source
- Reduce to 75% for CrCl 15-50.
- Operator and lab dependent
- Sensitivity below molecular methods; immunophenotype shift after therapy
- Between Cytarabine, Daunorubicin, Mitoxantrone and the other options, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- How closely do I match the people in COG AAML0531, and does that change what the results mean for me?Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (induction (two courses)), which of the standard options do you recommend and why?Why: Guideline options include: Cytarabine with daunorubicin or mitoxantrone and etoposide; gemtuzumab ozogamicin added to the first course for CD33-positive disease (AAML0531); MRD after course 1.
- Am I a candidate for Cytarabine, Daunorubicin, Mitoxantrone or related drugs, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of COG AAML0531 apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Add these to your appointment list, or take the full question set for this cancer.
Consolidation (two or three courses)
High-dose cytarabine-based courses; no maintenance.
Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.
Etoposide is a chemotherapy from the mayapple plant, essential to curing testicular cancer (BEP), treating small-cell lung cancer, lymphomas, childhood sarcomas and leukaemias, and used in transplant conditioning.
Mitoxantrone is a blue chemotherapy infusion used with cytarabine for acute myeloid leukaemia and, historically, to relieve pain in advanced prostate cancer; it is also licensed for multiple sclerosis.
Gemtuzumab ozogamicin (Mylotarg) is an anti-CD33 antibody linked to the DNA-cutting payload calicheamicin, the first ADC approved, in 2000 for relapsed acute myeloid leukaemia. An unstable linker and no benefit in a confirmatory trial led to withdrawal in 2010; it returned in 2017 at a lower fractionated dose, with liver toxicity, including veno-occlusive disease, its defining risk.
No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.
- Reduce to 75% for CrCl 15-50.
- Between Cytarabine, Etoposide, Mitoxantrone and the other options, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (consolidation (two or three courses)), which of the standard options do you recommend and why?Why: Guideline options include: High-dose cytarabine-based courses; no maintenance.
- Am I a candidate for Cytarabine, Etoposide, Mitoxantrone or related drugs, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Add these to your appointment list, or take the full question set for this cancer.
FLT3-ITD
Sorafenib added to chemotherapy for high allelic ratio (AAML1031); gilteritinib in paediatric trials.
The first drug ever to extend life in advanced liver cancer (2007), now mostly a comparator arm that newer combinations are measured against.
A single pill that beats salvage chemotherapy for relapsed FLT3-mutated AML, and the backbone of the triplets now being tested in frontline disease.
No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.
- Take without food (1 hour before or 2 hours after).
- Possible QT prolongation. Check ECG and electrolytes; review other QT-prolonging drugs.
| Side effect | Any grade | Grade 3+ |
|---|---|---|
| Differentiation syndrome · Boxed warning | 3% | - |
- Known QT prolongation. Avoid other QT-prolonging drugs where possible; check ECG and correct potassium and magnesium before and during treatment.
Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.
- Between Sorafenib and Gilteritinib, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- Which side effects of Gilteritinib are most likely for me, which are reversible, and which would make us stop?Why: The recorded rates below come from labels and trials; your own risk depends on dose, other medicines and your health.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (flt3-itd), which of the standard options do you recommend and why?Why: Guideline options include: Sorafenib added to chemotherapy for high allelic ratio (AAML1031); gilteritinib in paediatric trials.
- Am I a candidate for Sorafenib, Gilteritinib, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Add these to your appointment list, or take the full question set for this cancer.
High-risk genetics or persistent residual disease
Allogeneic transplant in first remission.
Allogeneic stem cell transplantation replaces a patient's blood system with a donor's after conditioning chemotherapy, so donor immune cells hunt down leukaemia left behind. It remains the only cure for adverse-risk acute myeloid leukaemia, high-risk acute lymphoblastic leukaemia and Richter transformation, at the price of graft-versus-host disease.
- Curative for otherwise incurable leukaemia
- Graft-versus-leukaemia is an antigen-agnostic immune therapy
Flow cytometry MRD counts leukaemia cells in the bone marrow one at a time by their surface proteins, down to one in ten thousand.
- Broadly applicable, fast (same day)
- Works without a molecular marker
No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.
- Treatment-related mortality 10-20%
- Chronic GVHD
- Relapse remains the main cause of failure
- Operator and lab dependent
- Sensitivity below molecular methods; immunophenotype shift after therapy
- Between Allogeneic stem cell transplantation and Multiparameter flow cytometry MRD, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (high-risk genetics or persistent residual disease), which of the standard options do you recommend and why?Why: Guideline options include: Allogeneic transplant in first remission.
Add these to your appointment list, or take the full question set for this cancer.
Relapsed or refractory
Fludarabine and cytarabine reinduction; revumenib for KMT2A-rearranged disease; gilteritinib for FLT3; venetoclax combinations in trials; transplant.
Fludarabine is a chemotherapy infusion for chronic lymphocytic leukaemia. It anchored the FCR regimen that was the first to give long remissions, and today it is used above all to prepare patients for CAR-T cells and transplants.
Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.
Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.
A single pill that beats salvage chemotherapy for relapsed FLT3-mutated AML, and the backbone of the triplets now being tested in frontline disease.
A pill that removes the survival shield from leukaemia cells, enabling chemotherapy-free, time-limited treatment for CLL.
Allogeneic stem cell transplantation replaces a patient's blood system with a donor's after conditioning chemotherapy, so donor immune cells hunt down leukaemia left behind. It remains the only cure for adverse-risk acute myeloid leukaemia, high-risk acute lymphoblastic leukaemia and Richter transformation, at the price of graft-versus-host disease.
- Curative for otherwise incurable leukaemia
- Graft-versus-leukaemia is an antigen-agnostic immune therapy
- Tests RevumenibRelapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy
CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort.
- Known QT prolongation. Avoid other QT-prolonging drugs where possible; check ECG and correct potassium and magnesium before and during treatment.
| Side effect | Any grade | Grade 3+ |
|---|---|---|
| Differentiation syndrome · Boxed warning | 3% | - |
- Known QT prolongation. Avoid other QT-prolonging drugs where possible; check ECG and correct potassium and magnesium before and during treatment.
Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.
- Take with a meal and water. Avoid grapefruit, Seville oranges and starfruit.
- Reduce by 50% in severe impairment.
- Treatment-related mortality 10-20%
- Chronic GVHD
- Relapse remains the main cause of failure
- Between Fludarabine, Cytarabine, Revumenib and the other options, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- How closely do I match the people in AUGMENT-101, and does that change what the results mean for me?Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
- Which side effects of Gilteritinib are most likely for me, which are reversible, and which would make us stop?Why: The recorded rates below come from labels and trials; your own risk depends on dose, other medicines and your health.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (relapsed or refractory), which of the standard options do you recommend and why?Why: Guideline options include: Fludarabine and cytarabine reinduction; revumenib for KMT2A-rearranged disease; gilteritinib for FLT3; venetoclax combinations in trials; transplant.
- Am I a candidate for Fludarabine, Cytarabine, Revumenib or related drugs, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of AUGMENT-101 apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Add these to your appointment list, or take the full question set for this cancer.
Supportive care and late effects
Dexrazoxane cardioprotection with anthracyclines, transfusion and antimicrobial support, cardio-oncology follow-up.
Dexrazoxane protects the heart from the cumulative damage of doxorubicin in women with metastatic breast cancer who need to keep receiving it, and, as Totect, limits tissue destruction when an anthracycline leaks out of a vein.
Cardio-oncology builds heart risk assessment, monitoring and prevention into cancer care so patients can finish curative treatment without trading cancer for heart failure. It targets anthracycline and trastuzumab damage, checkpoint-inhibitor myocarditis and radiation heart disease using echocardiography, troponin tests and protective drugs; specialist clinics are concentrated in large centres.
- Enables completion of curative therapy
Red cell and platelet transfusions, iron and erythropoiesis-stimulating agents keep patients safe through chemotherapy and marrow failure; restrictive thresholds and ESA caution reflect trials showing more is not better.
- Life-saving in acute leukaemia and transplant
- Restrictive strategies proven safe and cheaper
- New MDS agents reduce transfusion burden
No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.
- Workforce and access
- Blood supply shortages, especially in LMICs
- ESA safety restricts use
- Iron overload and alloimmunisation with chronic transfusion
- Between Dexrazoxane, Cardio-oncology and Transfusion support and anaemia management, which do you recommend for me, and what about my case would make you choose differently?Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
- What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?Why: The aim shapes how much side effect and disruption is worth accepting.
- What happens if I delay, or decline this step for now? Is the decision reversible?Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
- Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)), and if it departs from it, why?Why: Departures from guidelines are sometimes right for an individual; they should be explained.
- For my situation (supportive care and late effects), which of the standard options do you recommend and why?Why: Guideline options include: Dexrazoxane cardioprotection with anthracyclines, transfusion and antimicrobial support, cardio-oncology follow-up.
- Am I a candidate for Dexrazoxane, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Add these to your appointment list, or take the full question set for this cancer.