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Acute myeloid leukaemia, ELN 2022 risk, targeted therapy and allogeneic transplant.

A fast-moving marrow cancer with an equally fast-moving treatment landscape. Modern care matches the therapy to the biology, and matches intensity to the patient.

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A radiographer guides a patient onto the bed of an advanced 3 Tesla MRI scanner in a London imaging suite

Why trust this guide

  • 01

    Editorial team

    Written by our editorial team from the UK sources listed on each page.

  • 02

    Sourced from guidance

    Checked against BSH, ELN 2022, NICE and NCCN sources you can see at the end.

  • 03

    Current for 2026

    Reflects modern AML care including venetoclax + azacitidine, FLT3 and IDH inhibitors, and allogeneic transplant.

Key facts

AML at a glance.

The essentials, in plain English. What acute myeloid leukaemia is, how it is classified, and the treatment path that follows.

  • What it is

    Acute myeloid leukaemia is a clonal expansion of myeloid blasts in the bone marrow, defined by 20% or more blasts (or a defining genetic lesion).

  • Who it affects

    Median age at diagnosis is over 65. AML is the most common acute leukaemia in adults and rarer in children.

  • How it presents

    Fatigue, breathlessness, bruising, bleeding, fevers and infections from marrow failure - often over days to weeks.

  • Risk stratification

    ELN 2022 groups patients as favourable, intermediate or adverse based on cytogenetics and molecular findings.

  • Foundation therapy

    Intensive induction with 7+3 daunorubicin and cytarabine for fit patients; venetoclax + azacitidine for those unfit for intensive therapy.

  • Curative option

    Allogeneic haematopoietic stem cell transplant offers the best chance of long-term cure in intermediate and adverse-risk fit patients.

Why this guide matters

Biology drives the plan.

AML is not one disease. Cytogenetics, molecular profile and patient fitness together decide whether the goal is cure, long remission or comfort.

  • Genetics decides risk

    ELN 2022 uses cytogenetics and mutations - NPM1, FLT3, TP53, IDH1/2, CEBPA - to sort AML into favourable, intermediate and adverse risk.

  • Fitness decides intensity

    7+3 induction is standard for fit adults. Venetoclax + azacitidine is the modern default for older or unfit patients and now often preferred with NPM1 or IDH mutations.

  • Transplant is the cure route

    For intermediate and adverse risk, allogeneic stem cell transplant in first remission still gives the best chance of long-term cure.

How the diagnosis is made

From an abnormal blood count to a genetic risk profile.

The steps a UK haematology team will normally follow, in order - so you know what to expect and why every test matters.

  1. 01

    Suspecting

    Blood count and film

    FBC often shows anaemia, thrombocytopenia and abnormal white cells. Circulating blasts on the film raise immediate suspicion.

  2. 02

    Suspecting

    Urgent haematology referral

    Suspected acute leukaemia is a same-day referral to a specialist centre. Do not wait for further tests in the community.

  3. 03

    Suspecting

    Bone marrow aspirate and trephine

    Confirms the diagnosis. AML is defined by 20% or more myeloid blasts, or by an AML-defining genetic abnormality regardless of blast count.

  4. 04

    Confirming

    Immunophenotyping by flow cytometry

    Confirms myeloid lineage using markers such as MPO, CD13, CD33, CD34 and CD117 and distinguishes AML from ALL.

  5. 05

    Confirming

    Cytogenetics and FISH

    Karyotype and targeted FISH detect CBF translocations t(8;21) and inv(16), MLL/KMT2A rearrangements, monosomy 5 or 7 and complex karyotypes.

  6. 06

    Confirming

    Molecular panel

    Targeted sequencing for FLT3-ITD and FLT3-TKD, NPM1, IDH1, IDH2, TP53, CEBPA, RUNX1 and ASXL1 drives ELN 2022 risk and targeted therapy choices.

  7. 07

    Preparing

    APL rule-out and organ workup

    PML::RARA testing is done urgently if APL is possible. Baseline echo, viral serology, HLA typing and dental review prepare for treatment.

Typical timeline: from first abnormal count to a full genetic risk classification within days.

Symptoms

What AML looks and feels like.

Symptoms come from marrow failure - not enough red cells, platelets or normal white cells - plus features driven by the blasts themselves.

  • Fatigue and pallor

    Anaemia from marrow failure causes tiredness, breathlessness on exertion and washed-out skin.

  • Bruising and bleeding

    Thrombocytopenia causes easy bruising, petechiae, nosebleeds and gum bleeding. Heavy bleeding is a red flag.

  • Fevers and infections

    Neutropenia leaves patients vulnerable to bacterial and fungal infections. Any fever needs urgent assessment.

  • Bone and joint pain

    Marrow expansion can cause diffuse bone pain, especially in younger patients with a high blast burden.

  • Gum infiltration and skin lesions

    Monocytic subtypes can cause swollen gums and violaceous skin nodules (leukaemia cutis).

  • Leucostasis

    Very high white cell counts can cause breathlessness, confusion and visual changes - a medical emergency.

  • DIC in APL

    Acute promyelocytic leukaemia (APL, PML::RARA) presents with life-threatening coagulopathy and bleeding at diagnosis.

  • Red flag - neutropenic sepsis

    Fever with neutropenia during or after treatment needs antibiotics within one hour. Do not delay.

Treatment

How AML is treated in the UK.

Intensive induction, targeted therapy for FLT3 or IDH mutations, ATRA and arsenic for APL, and allogeneic transplant for those who need it.

  • 7+3 intensive induction

    Seven days of cytarabine plus three days of daunorubicin remains the standard induction for fit adults, aiming for complete remission.

  • Venetoclax + azacitidine

    The standard for older or unfit patients. Well tolerated relative to intensive chemo and highly active in NPM1 and IDH-mutated disease.

  • Midostaurin or quizartinib

    FLT3 inhibitors added to intensive induction and consolidation in FLT3-mutated AML to improve survival.

  • Gilteritinib for relapsed FLT3 AML

    Oral FLT3 inhibitor with superior outcomes to salvage chemotherapy in relapsed or refractory FLT3-mutated disease.

  • Ivosidenib and enasidenib

    Targeted IDH1 and IDH2 inhibitors, used in relapsed disease and increasingly in frontline combinations for older patients.

  • ATRA + arsenic trioxide for APL

    All-trans retinoic acid combined with arsenic trioxide cures the majority of APL cases without chemotherapy.

  • Consolidation and maintenance

    High-dose cytarabine cycles after remission, with oral azacitidine (CC-486) maintenance in selected patients.

  • Allogeneic stem cell transplant

    Curative option for intermediate and adverse-risk fit patients in first remission - the strongest anti-leukaemia effect available.

What this guide is based on

The sources behind every claim on this page.

UK national guidance and international specialist society standards, current at the time of last review.

Key references

Guidelines and standards we relied on.

A quiet reminder

This guide is for information, not medical advice.

Your haematology team knows your biology, fitness and treatment history and can tell you which parts apply to you.

  • European LeukemiaNet (ELN). 2022 recommendations for diagnosis and management of AML in adults.

  • British Society for Haematology (BSH). Guidelines on the management of adult acute myeloid leukaemia.

  • NICE. Technology appraisals on venetoclax, midostaurin, gilteritinib, ivosidenib and CPX-351.

  • NCCN. Clinical practice guidelines in oncology: acute myeloid leukemia.

Red flags

When AML needs urgent attention.

AML and its treatment carry specific emergencies. Every patient and family member should know the shortlist below.

  • Neutropenic sepsis

    Fever with neutrophils below 1.0 needs empirical broad-spectrum antibiotics within one hour. The single most important safety rule in AML care.

  • APL coagulopathy

    Suspected APL is a haematological emergency. Start ATRA on clinical suspicion before molecular confirmation to reduce early bleeding deaths.

  • Leucostasis

    White cell count above 100 with breathlessness, confusion or retinal haemorrhage needs urgent cytoreduction and specialist input.

  • Tumour lysis syndrome

    Rapid cell breakdown after treatment causes hyperkalaemia, hyperphosphataemia, hyperuricaemia and acute kidney injury. Prophylaxis is standard.

  • Differentiation syndrome

    ATRA or IDH inhibitors can cause fever, breathlessness, oedema and hypoxia. Dexamethasone reverses it when caught early.

  • Intracranial bleeding

    Severe thrombocytopenia with headache, vomiting or focal signs needs urgent imaging and platelet support.

  • CNS disease

    Cranial nerve palsies or new neurological signs raise suspicion of CNS leukaemia and warrant lumbar puncture with intrathecal chemotherapy.

  • Relapse signals

    Falling counts, new blasts on the film or rising measurable residual disease need urgent haematology review.

  • Transplant complications

    Graft-versus-host disease, CMV reactivation and veno-occlusive disease are life-threatening and need specialist care.

Living with it

A serious diagnosis, supported at every step.

The practicalities that matter as much as the drugs - team, infection vigilance, energy management and follow-up.

A quiet reminder

Fever during neutropenia is always an emergency.

Keep your 24-hour hospital number by the phone. Antibiotics within an hour of fever save lives.

  1. 01 Support

    You need a team, not just a doctor

    A clinical nurse specialist, dietitian and psychologist matter as much as your consultant during and after treatment.

  2. 02 Infection

    Fever is always an emergency

    During neutropenia any temperature of 38C or a single 37.5C reading needs a hospital call the same hour, not the next day.

  3. 03 Recovery

    Fatigue is the long tail

    Energy returns slowly over months after intensive treatment or transplant. Gentle graded activity beats pushing through.

  4. 04 Follow-up

    MRD-guided monitoring

    Measurable residual disease testing on marrow or blood guides how closely you are watched and when to intervene.

Frequently asked

Everything we get asked about AML.

Quick answers on risk classification, induction choices, targeted therapy, transplant and survival.

  • What is acute myeloid leukaemia?

    AML is a fast-moving cancer of the bone marrow in which immature myeloid cells (blasts) crowd out normal blood cell production. It is diagnosed when at least 20% of marrow cells are blasts, or when a defining genetic abnormality is present regardless of blast count.

  • How is AML risk classified in 2026?

    The European LeukemiaNet 2022 system groups AML into favourable, intermediate and adverse risk. Favourable includes core-binding factor translocations t(8;21) and inv(16), and NPM1-mutated disease without a high FLT3-ITD burden. Adverse risk includes TP53 mutation, complex karyotype, MLL/KMT2A rearrangement and monosomy 5 or 7.

  • What is the standard first treatment?

    Fit adults typically receive intensive induction with 7+3 (seven days of cytarabine and three days of daunorubicin). Older or medically unfit patients receive venetoclax combined with azacitidine, which has transformed outcomes for this group without the toxicity of intensive chemotherapy.

  • When are targeted therapies used?

    FLT3 inhibitors (midostaurin, quizartinib, gilteritinib) are added when a FLT3 mutation is present. IDH1 and IDH2 inhibitors (ivosidenib, enasidenib) target IDH-mutated disease. Acute promyelocytic leukaemia (APL, PML::RARA fusion) is treated with ATRA and arsenic trioxide, which is curative in the majority of cases without chemotherapy.

  • Who needs a stem cell transplant?

    Allogeneic haematopoietic stem cell transplant in first remission is the standard of care for fit patients with intermediate or adverse-risk AML. It offers the strongest anti-leukaemia effect but carries real risks including graft-versus-host disease and infection. The decision is individualised with your transplant team.

  • What are the survival figures?

    Outcomes depend heavily on age and biology. Five-year survival is around 30% in patients under 60 and drops to around 5% in those over 70 with adverse-risk disease. Favourable-risk AML in younger patients can exceed 60%, and modern targeted therapies and MRD-guided decisions continue to improve results.

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