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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.
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.
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.
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.
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: High-grade B-cell lymphoma with MYC and BCL2 rearrangements (double-hit lymphoma), Mediastinal grey zone lymphoma, Primary effusion lymphoma, Plasmablastic lymphoma, T-cell/histiocyte-rich large B-cell lymphoma, EBV-positive diffuse large B-cell lymphoma, Primary large B-cell lymphoma of the testis, Gastric MALT lymphoma, Ocular adnexal MALT lymphoma, Extranodal NK/T-cell lymphoma, Adult T-cell leukaemia/lymphoma, ALK-positive anaplastic large cell lymphoma, ALK-negative anaplastic large cell lymphoma, Breast implant-associated anaplastic large cell lymphoma, Primary cutaneous anaplastic large cell lymphoma, Lymphomatoid papulosis, Mycosis fungoides, Enteropathy-associated T-cell lymphoma, Monomorphic epitheliotropic intestinal T-cell lymphoma, Extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma), Splenic marginal zone lymphoma, Nodal marginal zone lymphoma, Primary cutaneous marginal zone lymphoma, Primary cutaneous follicle centre lymphoma, Sezary syndrome, Nodal T-follicular helper cell lymphoma, angioimmunoblastic type (angioimmunoblastic T-cell lymphoma), Hepatosplenic T-cell lymphoma, Intravascular large B-cell lymphoma, Lymphomatoid granulomatosis, T-cell prolymphocytic leukaemia, Splenic B-cell lymphoma/leukaemia with prominent nucleoli (formerly B-cell prolymphocytic leukaemia and hairy cell leukaemia variant), T-cell large granular lymphocytic leukaemia, Mixed-phenotype acute leukaemia, Myeloid leukaemia of Down syndrome, Burkitt leukaemia, 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, Erdheim-Chester disease, Rosai-Dorfman-Destombes disease, Single-system Langerhans cell histiocytosis (bone, skin or one other organ), Multisystem Langerhans cell histiocytosis (with or without risk-organ involvement), Indolent and smouldering systemic mastocytosis, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia), Early-stage classical Hodgkin lymphoma (stage I to II), Advanced-stage classical Hodgkin lymphoma (stage III to IV), Nodular lymphocyte-predominant Hodgkin lymphoma (nodular lymphocyte-predominant B-cell lymphoma), Relapsed and refractory classical Hodgkin lymphoma
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Also on OnCo: Symptoms and red flags · Early detection roadmap.
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.
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FLT3 inhibitors are now incorporated into frontline paediatric AML therapy for FLT3-ITD, with gilteritinib being studied in the successor trial.
Bortezomib should not be added to standard chemotherapy for childhood acute myeloid leukaemia; the trial's FLT3-ITD sorafenib cohorts were reported separately.
Paediatric AML risk classification and targeted therapy development now rest on this landscape rather than on adult data.
Gemtuzumab ozogamicin is part of standard induction for CD33-positive childhood AML in Children's Oncology Group protocols.
Query 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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Bleeding that does not stop by itself, bleeding from more than one site, or new bruising in several places or one large area.
Fainting, near-fainting, or an irregular or racing heartbeat; several kinase inhibitors prolong the QT interval and the labels require ECG and electrolyte monitoring.
Sudden severe abdominal pain, a hard or very tender abdomen, or abdominal pain with vomiting and fever. Boxed warning for gastrointestinal perforation on bevacizumab.
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.
Nausea, vomiting, muscle cramps, palpitations, seizures, confusion or passing much less urine in the first days of a new dose; the label requires hydration, anti-hyperuricaemic drugs and blood tests around each ramp-up step.
Temperature of 38 C or higher, or feeling shivery and unwell even without a fever. Antibody-drug conjugates suppress the bone marrow, and several carry a boxed warning for severe neutropenia.
See all on the product pages:CytarabineDaunorubicinEtoposideGemtuzumab ozogamicinGilteritinibMitoxantroneRevumenibSorafenibVenetoclax·Printable cards in the navigator
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