Acute myeloid leukaemia in children
Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children.
Overview
Childhood AML is defined by chromosomal rearrangements: KMT2A fusions in about a fifth, more in infants; the core-binding-factor leukaemias RUNX1::RUNX1T1 and CBFB::MYH11 in about a quarter, with the best prognosis; NUP98 fusions, CBFA2T3::GLIS2 and RBM15::MRTFA in infants and young children, with the worst; and FLT3 internal tandem duplications in about 15 percent, more in adolescents. NPM1, DNMT3A, TP53 and spliceosome mutations, which dominate adult AML, are rare. Down syndrome brings a distinct GATA1-mutated myeloid leukaemia in the first four years of life that is highly curable with reduced chemotherapy. Risk is assigned by these lesions and by flow cytometry measurable residual disease after the first course.
The backbone came from the UK MRC AML10 and AML12 trials of the 1990s, which set four intensive courses of cytarabine and anthracycline-based chemotherapy with no maintenance, and from the Berlin-Frankfurt-Münster and Nordic groups. COG AAML0531 randomised 1,022 children to gemtuzumab ozogamicin, a CD33 antibody-drug conjugate, added to the first induction and second intensification course: three-year event-free survival 53.1 percent against 46.9 percent, driven by lower relapse, with no significant overall survival gain but a clear benefit in children with high CD33 expression; gemtuzumab was approved for newly diagnosed CD33-positive AML in children from one month of age in 2020. AAML1031 found no benefit from adding bortezomib, and showed that sorafenib added to chemotherapy for FLT3-ITD disease with a high allelic ratio improved event-free survival compared with the FLT3-ITD children on AAML0531. Allogeneic transplant in first remission is reserved for high-risk genetics or persistent residual disease, and the European MyeChild01 trial has been settling gemtuzumab dosing and the choice of anthracycline.
Relapse occurs in about a third of children and is cured in about half of them with fludarabine and cytarabine reinduction and transplant. Revumenib, approved in November 2024 for relapsed or refractory KMT2A-rearranged acute leukaemia from the age of one, is the first targeted therapy for the commonest genetic subgroup; gilteritinib for FLT3-mutated disease and venetoclax with cytarabine, which gave responses in the majority of relapsed children in a St Jude phase 1 study, are in paediatric trials. Anthracycline cardiotoxicity is the defining late effect, so dexrazoxane cardioprotection, liposomal anthracyclines and cumulative-dose limits run through every trial, and infection during the profound neutropenia of each course still kills more children than the leukaemia in many centres.
State of the art
- Gemtuzumab ozogamicin added to intensive chemotherapy lowered relapse in AAML0531 and is approved for children from one month of age.
- Revumenib is the first targeted therapy approved for KMT2A-rearranged leukaemia, the commonest genetic subgroup in young children.
- Flow cytometry residual disease after the first course, with genetics, decides who is transplanted in first remission.
Red cards
From the labels and guidelines behind the standard of care. Your team's thresholds win.- Emergency services nowBleeding or bruising
Bleeding that will not stop, black or bloody stools, or unexplained bruising when platelets are expected to be low.
- Emergency services nowFainting or palpitations
Fainting, near-fainting, or an irregular or racing heartbeat; several kinase inhibitors prolong the QT interval and the labels require ECG and electrolyte monitoring.
- Emergency services nowBlood clot (lenalidomide, pomalidomide, thalidomide)
A swollen painful calf, or sudden breathlessness with chest pain; venous and arterial thromboembolism is a boxed warning and blood-thinning prophylaxis is recommended.
- Emergency services nowBowel perforation
Sudden severe abdominal pain, a hard or very tender abdomen, or abdominal pain with vomiting and fever. Boxed warning for gastrointestinal perforation on bevacizumab.
- Emergency services nowDifferentiation syndrome
Fever, cough or breathlessness, rapid weight gain or swelling, bone pain, low blood pressure or reduced urine; the labels say to start steroids and monitor at the first suspicion, and the syndrome has been fatal.
- Emergency services nowSkin reaction
Blisters, peeling, or sores in the mouth or eyes with a rash. Enfortumab vedotin carries a boxed warning for Stevens-Johnson syndrome and toxic epidermal necrolysis, mostly in the first cycle.
See all on the product pages:CytarabineDaunorubicinEtoposideGemtuzumab ozogamicinGilteritinibMitoxantroneRevumenibSorafenib·Printable cards in the navigator
Anatomy and lymph node drainage
- Bone marrow (leukaemia, MDS, MPN, myeloma)
- Lymph node germinal centre (lymphomas)
- Spleen
- Blood (leukaemic phase)
- Lytic bone lesions (myeloma)
- Skin and extranodal sites
- Nodes: cervical
- Nodes: axillary
- Nodes: mediastinal
- Nodes: para-aortic and mesenteric
- Nodes: inguinal
Leukaemias, myeloma and MDS live in the marrow and blood; lymphomas grow in lymph nodes and spleen. The node stations are the disease map, not a route of spread, and staging counts them.
- Bone marrow (leukaemia, MDS, MPN, myeloma)Core-binding-factor AML (RUNX1::RUNX1T1, CBFB::MYH11; low risk) · KMT2A-rearranged AML (about a fifth; partner-dependent risk; menin inhibitors) · FLT3-ITD AML (about 15 percent; sorafenib or gilteritinib added) · Infant AML with NUP98, CBFA2T3::GLIS2 or RBM15::MRTFA fusions (high risk) · Myeloid leukaemia of Down syndrome (GATA1-mutated; reduced-intensity chemotherapy) · Acute promyelocytic leukaemia in children (retinoic acid and arsenic as in adults) · Therapy-related and secondary AML in children
- Lymph node germinal centre (lymphomas)
- Spleen
- Blood (leukaemic phase)
- Lytic bone lesions (myeloma)
- Skin and extranodal sites
- cervical
- axillary
- mediastinal
- para-aortic and mesenteric
- inguinal
In lymphoma the node stations are the disease itself; staging (Ann Arbor / Lugano) counts how many regions and sides of the diaphragm are involved.
Same organ: Marginal zone lymphoma, Cutaneous T-cell lymphoma (mycosis fungoides and Sezary syndrome), Primary mediastinal (thymic) large B-cell lymphoma, Leukaemia (all types), Acute myeloid leukaemia, Acute lymphoblastic leukaemia, Chronic lymphocytic leukaemia, Chronic myeloid leukaemia (CML), Diffuse large B-cell lymphoma, Follicular lymphoma, Hodgkin lymphoma, Mantle cell lymphoma, Multiple myeloma, Non-Hodgkin lymphoma (all types), Myelodysplastic syndromes / neoplasms (MDS), Myeloproliferative neoplasms (PV, ET, myelofibrosis), Polycythaemia vera (PV), Essential thrombocythaemia (ET), Waldenström macroglobulinaemia, Hairy cell leukaemia, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma), Blastic plasmacytoid dendritic cell neoplasm (BPDCN), Burkitt lymphoma, HIV-associated (AIDS-related) lymphomas, Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms, Systemic mastocytosis, Erdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms, Langerhans cell histiocytosis (LCH), Post-transplant lymphoproliferative disorder (PTLD), FLT3-mutated acute myeloid leukaemia, IDH1- and IDH2-mutated acute myeloid leukaemia, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Secondary and therapy-related acute myeloid leukaemia, Acute promyelocytic leukaemia, Acute myeloid leukaemia in older or unfit patients, Smouldering multiple myeloma, Newly diagnosed multiple myeloma, transplant-eligible, Newly diagnosed multiple myeloma, transplant-ineligible, Relapsed or refractory multiple myeloma, Plasma cell leukaemia, Lower-risk myelodysplastic syndromes, Higher-risk myelodysplastic syndromes, Chronic lymphocytic leukaemia, first treatment, Relapsed or refractory chronic lymphocytic leukaemia, Richter transformation of chronic lymphocytic leukaemia, Chronic myeloid leukaemia, chronic phase, Chronic myeloid leukaemia, accelerated and blast phase, Primary myelofibrosis, Standard-risk B-cell acute lymphoblastic leukaemia in children, High-risk acute lymphoblastic leukaemia in children (high-risk B-ALL and T-ALL), Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL), Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL), Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year), Relapsed and refractory acute lymphoblastic leukaemia in children
Acute myeloid leukaemia is about one in five childhood leukaemias, a few hundred children a year in the United Kingdom and United States; cure rates have climbed from under a third in the 1970s to around two thirds through more intensive chemotherapy and better supportive care.
Cases by country
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
One section per setting: the options named, what each is for, the trials behind them, the recorded trade-offs and the questions to ask.
Cytarabine with daunorubicin or mitoxantrone and etoposide; gemtuzumab ozogamicin added to the first course for CD33-positive disease (AAML0531); MRD after course 1.
High-dose cytarabine-based courses; no maintenance.
Sorafenib added to chemotherapy for high allelic ratio (AAML1031); gilteritinib in paediatric trials.
Allogeneic transplant in first remission.
Fludarabine and cytarabine reinduction; revumenib for KMT2A-rearranged disease; gilteritinib for FLT3; venetoclax combinations in trials; transplant.
Dexrazoxane cardioprotection with anthracyclines, transfusion and antimicrobial support, cardio-oncology follow-up.
Subtypes & biomarkers
top- Core-binding-factor AML (RUNX1::RUNX1T1, CBFB::MYH11; low risk)
- KMT2A-rearranged AML (about a fifth; partner-dependent risk; menin inhibitors)
- FLT3-ITD AML (about 15 percent; sorafenib or gilteritinib added)
- Infant AML with NUP98, CBFA2T3 ::GLIS2 or RBM15::MRTFA fusions (high risk)
- Myeloid leukaemia of Down syndrome (GATA1-mutated; reduced-intensity chemotherapy)
- Acute promyelocytic leukaemia in children (retinoic acid and arsenic as in adults)
- Therapy-related and secondary AML in children
- Karyotype and FISH for recurrent fusions
- RNA fusion panel (KMT2A, NUP98, CBFA2T3::GLIS2)
- FLT3-ITD and allelic ratio
- CD33 expression
- Flow cytometry MRD after course 1 and course 2
- GATA1 mutation in Down syndrome
- Germline predisposition (GATA2, RUNX1, CEBPA)
How often this target appears
- 1998MRC AML10: four intensive courses without maintenance become the paediatric standard
- 2014AAML0531: gemtuzumab ozogamicin lowers relapse in childhood AML
- 2017Gemtuzumab ozogamicin returns to the market after withdrawal in 2010
- 2020Gemtuzumab ozogamicin approved for newly diagnosed CD33-positive AML in children from one month
- 2022AAML1031: sorafenib improves event-free survival in high allelic ratio FLT3-ITD AML; bortezomib adds nothing
- 2024Revumenib approved for relapsed KMT2A-rearranged acute leukaemia from the age of one
Dated changes read from the records linked to this cancer: approvals, regulatory steps, reported trials, guideline versions and milestones. Newest first; no date is inferred.
All 9 changes by month →- 2026-09-17This recordAcute myeloid leukaemia in childrenFacts on this page last checked
When this page itself was last checked or edited.
- 2024Trial resultAUGMENT-101AUGMENT-101 reported
CR+CRh 22.
- 2024MilestoneRevumenibRevumenib approved for relapsed KMT2A-rearranged acute leukaemia from the age of one
A milestone in how this cancer is treated.
- 2022MilestoneSorafenibAAML1031: sorafenib improves event-free survival in high allelic ratio FLT3-ITD AML; bortezomib adds nothing
A milestone in how this cancer is treated.
- 2020MilestoneGemtuzumab ozogamicinGemtuzumab ozogamicin approved for newly diagnosed CD33-positive AML in children from one month
A milestone in how this cancer is treated.
- 2017MilestoneGemtuzumab ozogamicinGemtuzumab ozogamicin returns to the market after withdrawal in 2010
A milestone in how this cancer is treated.
What is in development for Acute myeloid leukaemia in children, drawn from the whole corpus: 5 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Trials under way · 4
- A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML · phase 2 · Novartis Pharmaceuticals
- Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors · phase 1/2 · Incyte Biosciences International Sàrl
- Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood · phase 1/2 · Daiichi Sankyo
- Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant · phase 1/2 · Bellicum Pharmaceuticals
Trials reported · 1
- COG AAML0531 · phase 3 · 2014 · positive
Open problems and what is being done
A third of children relapse and only half of those are cured.
and how the field plans to fix it →What is being done about thisRecurrence and residual diseaseAvailable now- Gemtuzumab ozogamicinApproved
- GilteritinibApproved
- Multiparameter flow cytometry MRDStandard of care
- ZiftomenibApproved
In trialsIdeas and roadmapsNothing recorded yet.
Background: Minimal / molecular residual disease (MRD). Also on OnCo: Treatment journeys · Survivorship planner.
Infant AML with NUP98 or CBFA2T3::GLIS2 fusions has no effective therapy.
and how the field plans to fix it →What is being done about thisCancers without a drug targetAvailable now- RevumenibApproved
- ZiftomenibApproved
In trialsNothing recorded yet.
Ideas and roadmapsNothing recorded yet.
Also on OnCo: Targets · KRAS roadmap.
Anthracycline cardiotoxicity and infection deaths from the most intensive chemotherapy given to children.
and how the field plans to fix it →What is being done about thisSide effects and quality of lifeAvailable now- Allogeneic stem cell transplantationStandard of care
- Antibody-drug conjugate (ADC)Approved
- Cardio-oncologyEstablished
- Gemtuzumab ozogamicinApproved
In trialsNothing recorded yet.
Ideas and roadmapsNothing recorded yet.
Also on OnCo: Side effects by symptom · Immune-related side effects · Toxicity compare · Survivorship planner.
Trials
topTrials recruiting now
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Landmark trials
Expert centres
topExpert centres
Monrovia, CA · consortium | United States | none recorded | 1 | 20 | 186 | none recorded | - |
Memphis, TN · cancer center | United States | 0 | 636 | 8,887 | - | ||
Melbourne · research institute | Australia | none recorded | 0 | 298 | 3,639 | - | |
Indianapolis, IN · cancer center | United States | 0 | 159 | 4,238 | - | ||
Hamilton, ON · cancer center | Canada | none recorded | 0 | 103 | 1,158 | - | |
Baltimore, MD · cancer center | United States | 0 | 101 | 2,306 | - | ||
Omaha, NE · cancer center | United States | 0 | 37 | 4,159 | - | ||
Rotterdam · consortium | Netherlands | none recorded | 0 | 8 | 207 | - | |
Minneapolis, MN · cancer center | United States | 0 | not matched | - | - | ||
Aurora, ON · consortium | Canada | none recorded | 0 | not matched | - | - |
These are the things we can measure; they are not a ranking of quality. Each column is a field on the institution record or a count over what OnCo has linked; a centre that treats many patients with Acute myeloid leukaemia in children but is thinly recorded here will look small.
Not known: OnCo holds no case-volume or outcome figures for centres, so none are shown. Where a national audit or registry publishes them, the centre's page links to it. Default order: Newsweek rank, then trials for this cancer, then research output.
Questions to ask
topQuestions to ask your oncologist about Acute myeloid leukaemia in children
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example Karyotype and FISH for recurrent fusions, RNA fusion panel, FLT3-ITD and allelic ratio, CD33 expression, Flow cytometry MRD after course 1 and course 2), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include Core-binding-factor AML, KMT2A-rearranged AML, FLT3-ITD AML.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Induction (two courses)
- 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.
Consolidation (two or three courses)
- 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.
FLT3-ITD
- 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.
High-risk genetics or persistent residual disease
- 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.
Relapsed or refractory
- 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.
Supportive care and late effects
- 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.
Any stage
- Are there clinical trials I could join, for example of Revumenib, Gilteritinib, Venetoclax, Ziftomenib?Why: Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “A third of children relapse and only half of those are cured”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Infant AML with NUP98 or CBFA2T3::GLIS2 fusions has no effective therapy”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
Newly diagnosed? Read the first 60 days with Acute myeloid leukaemia in children, then print the one-page appointment sheet with room for the answers.
Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.
Direct links plus the targets, companies, and technologies of this cancer's products.
technologies
14targets
8drugs
14companies
11institutions
2pathways
1terms
3trials
6Latest papers
topQuery for this cancer: (TITLE:"Acute myeloid leukaemia in children" OR ABSTRACT:"Acute myeloid leukaemia in children" OR TITLE:"Childhood AML" OR ABSTRACT:"Childhood AML" OR TITLE:"Paediatric AML" OR ABSTRACT:"Paediatric AML" OR TITLE:"Pediatric acute myeloid leukemia" OR ABSTRACT:"Pediatric acute myeloid leukemia") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Acute myeloid leukaemia in children, not a curated reading list.
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