Acute myeloid leukaemia

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Acute myeloid leukaemia at a glance

Acute myeloid leukaemia, or AML, is a blood cancer in which abnormal immature blood-forming cells build up in the bone marrow, the soft tissue inside bones that makes blood cells. These cells belong to the myeloid family, which normally produces red cells, platelets and several kinds of white cell. They interfere with healthy blood production. It can progress quickly and needs prompt specialist assessment. Treatment may aim for cure, a lasting remission or disease control, depending on the features of the leukaemia cells, your general health and your priorities. Remission means the disease has responded according to defined blood and marrow tests; it does not guarantee that it cannot return.

AML is not one uniform disease. Genetic tests can identify subtypes that respond to particular medicines and help estimate relapse risk. The first few days often involve both urgent supportive care and tests to select the most appropriate treatment. If your condition is stable, a short planned wait for crucial genetic results can help the team choose treatment. If your condition is unstable, urgent treatment and care to stabilise you cannot wait.

This guide focuses mainly on adults. Children, adolescents and young adults may need age-specific protocols and specialist paediatric or adolescent services. A treatment suitable for one age group should not be transferred to another without expert assessment.

Symptoms and when to seek help

Anaemia, a shortage of healthy red cells or the oxygen-carrying protein haemoglobin, may cause tiredness, dizziness or breathlessness. Platelets help blood clot; low platelets can cause bruising, bleeding gums or nosebleeds. Infection can occur because normal white cells are reduced or do not work properly, even when the total white-cell count is high. Some people have fever, bone discomfort, swollen gums or skin changes.

Severe breathlessness, confusion, visual changes, chest pain, major bleeding or a severe headache needs immediate assessment. Very high white-cell counts can impair blood flow in small vessels, a complication called leukostasis. Acute promyelocytic leukaemia, a particular AML subtype, can cause dangerous clotting and bleeding abnormalities at presentation. These are hospital emergencies, not situations for waiting for a routine appointment.

What happens first

The team assesses infection, bleeding, kidney function and hydration. They also check for tumour lysis syndrome: rapid breakdown of leukaemia cells that can upset blood chemistry and damage the kidneys. Blood and platelet transfusions, antibiotics and measures to control a very high white-cell count may be needed. Ask which decisions are urgent and which await the marrow or genetic report. Having one person help keep track of information can be useful while investigations happen quickly.

Making this information useful to you

A cancer diagnosis can arrive before you have had time to understand the tests. You do not need to learn every treatment name at once. Begin with the exact diagnosis, where the cancer is, and what the team recommends next. A relative or friend can join the appointment if you would find that helpful. Ask for an interpreter when language makes a discussion harder; important decisions should not depend on guessing unfamiliar words.

This guide explains possibilities, not a prescription for your situation. Your blood and tissue test results, scan findings, general health, previous treatment and priorities determine which possibilities apply. Two people with the same cancer name may appropriately receive different treatment. If a recommendation differs from something you have read, ask the team to explain the reason and the evidence behind it.

Keep a list of medicines, allergies and other health conditions, including non-prescription products, vitamins and herbal remedies. Tell the team about previous cancer treatment, pregnancy or breastfeeding, and anything that could affect attending appointments. Do not stop an established medicine without advice. Bring your questions in the order that matters most to you, so that the most important concern is discussed first.

Understand

The marrow and the leukaemia cells

Bone marrow makes red cells, white cells and platelets. In AML, an abnormal cell population disrupts this process. Immature cells are often called blasts. AML is diagnosed through a combination of cell appearance under the microscope, proteins on the cells and genetic findings; a simple blast percentage is not the whole diagnosis, because some defining genetic abnormalities can establish AML at lower percentages.

AML may arise without a recognised previous blood condition, after myelodysplastic syndromes (MDS), a group of disorders causing ineffective blood-cell production, or another marrow disorder, or following earlier cancer treatment. These backgrounds can influence classification and treatment. Most genetic abnormalities found in AML are acquired in the leukaemia cells. Selected patients need investigation for an inherited predisposition, especially with younger onset, a suggestive family history or characteristic findings.

Important biological groups

The report may mention changes involving genes with names such as NPM1, FLT3, IDH1, IDH2, CEBPA, TP53 or KMT2A. You do not need to memorise these names; ask which finding changes your treatment. Core-binding-factor AML includes particular chromosome rearrangements and may have a different treatment pathway. Some abnormalities predict sensitivity to a drug; others help estimate relapse risk. The same gene name does not mean the same outlook in every combination of findings.

Acute promyelocytic leukaemia, or APL, usually involves a joined gene, or fusion, called PML::RARA. It is treated differently from most AML and is often highly curable with the right urgent care. If APL is suspected, doctors may begin all-trans retinoic acid, known as ATRA, before the genetic result is complete because delaying treatment can be dangerous. This is a hospital-led decision.

Why risk assessment is individual

Genetic risk categories help guide treatment, particularly after intensive chemotherapy. Their meaning can differ with less intensive regimens. Age, organ function, frailty and response all matter. Ask which risk system applies to your proposed treatment and whether important information is still pending. Statistics from a broad AML group may not describe a person with a specific targetable subtype.

Tests and diagnosis

Blood and bone marrow testing

A full blood count, blood film, chemistry tests, clotting tests and infection assessment are usually obtained promptly. A marrow aspirate takes a liquid sample and a biopsy takes a small core, usually from the pelvic bone. These samples let the laboratory examine the cells, test their proteins with flow cytometry, and look for chromosome and gene changes. Blood samples may provide some information when many blasts are circulating, but marrow assessment is usually important for diagnosis and subsequent response.

Flow cytometry identifies proteins on and inside cells to help determine which family of blood cells the leukaemia comes from. Cytogenetics examines chromosomes, the packages containing our genes. Fluorescence in situ hybridisation (FISH) uses fluorescent probes to find particular chromosome changes. Molecular tests look for gene changes, including mutations and fusion genes, in which parts of two genes have joined.

Ask whether rapid tests for FLT3, NPM1 and IDH1/IDH2 changes, and for APL is needed, and when the complete panel will follow. A targeted drug should not be chosen solely because a gene appears somewhere on an old report.

Preparing for treatment safely

Kidney and liver function influence drug selection and dosing. Heart assessment may be needed before anthracycline chemotherapy, a drug family that includes daunorubicin and doxorubicin. Screening for infections, pregnancy testing where relevant and discussion of fertility preservation are part of planning. Fertility care should be offered promptly when feasible, while recognising that urgent leukaemia treatment sometimes limits the time available.

A central venous line is a thin tube in a large vein. It can make chemotherapy, blood sampling, transfusions and supportive treatment easier. Ask how to protect it and which symptoms suggest infection or a clot. Redness, pain, leakage, fever or swelling of the arm or neck should be reported.

Other investigations

Scans are directed by symptoms, suspected infection or disease outside the marrow. AML does not usually require the same routine whole-body staging scans as a solid tumour. A lumbar puncture takes a sample of the fluid around the brain and spinal cord through a needle in the lower back. It is used in selected circumstances, such as suspected involvement of the central nervous system (the brain and spinal cord), rather than automatically for every adult with AML.

If transplant may become part of the plan, tissue typing and donor assessment can begin early. Donor matching is different from ordinary blood-group matching. An inherited predisposition, if suspected, must be considered before a family member is selected as donor.

Response assessments

The timing of marrow reassessment depends on the regimen. Counts can remain low because treatment has suppressed normal marrow, because leukaemia persists, or both. The team uses the marrow result to distinguish these situations and adjust subsequent cycles. Ask what the next marrow test will answer and whether the result changes the duration of oral medicines or the timing of consolidation.

Preparing for your appointment

If scans have been performed, bring a CD containing your scan images in Digital Imaging and Communications in Medicine (DICOM) format, the standard used for medical images, together with the software needed to view them, along with the written scan reports. Bring your biopsy report and ask whether the pathology slides or tissue blocks are needed for specialist review or additional tests. Keep originals safe and agree how borrowed material will be returned. Having the actual images allows the team to compare changes and plan treatment accurately.

A repeat test is sometimes needed because an earlier sample was too small, a scan did not cover the necessary area, or a new result will change treatment. Ask what question the test will answer, how it may affect the plan, and when you should expect the result. A result described as uncertain does not automatically mean the cancer is worse. Sometimes discussion between the scan specialist (radiologist), tissue specialist (pathologist) and treating team resolves the uncertainty without another procedure.

Before a scan or procedure, mention kidney problems, previous contrast reactions, implanted devices, diabetes medicines, blood-thinning medicines and the possibility of pregnancy. Follow the testing centre's instructions about food, drink and medicines. If you have not received those instructions, contact the centre rather than making changes yourself.

Planning your treatment

Intensive and less intensive treatment

Treatment intensity is chosen using fitness and goals, not age alone. Intensive induction, the first treatment phase aimed at remission, often requires a prolonged hospital stay and a period of very low counts. Less intensive treatment may be delivered partly as an outpatient, but it can still cause serious infection, bleeding and tumour lysis and may require frequent visits or admission.

For some patients, controlling symptoms with transfusions, infection treatment and lower-burden care is the most appropriate approach. This should be an active, supported decision. Ask about expected benefit, time in hospital, likelihood of remission and what happens if the first plan does not work.

The overall sequence

Induction aims to produce remission. Consolidation reduces the chance of relapse after remission and may involve further chemotherapy or a donor transplant. Maintenance is used in selected settings. Each stage has a different purpose; feeling better after induction does not mean the remaining treatment can be omitted safely.

Genetic results and measurable residual disease (MRD) tests, which look for small amounts of leukaemia that routine tests may miss, may change the plan after treatment has started. A change can reflect better information rather than a mistake. Ask the team to explain which finding led to the recommendation and how the expected benefit compares with the risks.

Donor transplantation

A donor stem-cell transplant, also called an allogeneic transplant, can offer a stronger anti-leukaemia effect for selected patients, especially when relapse risk is substantial. It is not required for everyone with AML. The decision considers remission quality, MRD, genetics, donor options and the risks of conditioning (the treatment before transplant), infection and graft-versus-host disease, in which donor immune cells attack healthy tissues.

The transplant pathway involves assessment of organs, infections, medicines, social support and recovery arrangements. Some people require further disease control before proceeding. Even with a suitable donor, transplant may not be the safest option for a particular patient. Ask how the estimated relapse risk without transplant compares with the risks of the procedure.

Questions to ask your care team

• What is the exact blood cancer type and risk category, and are any important results still pending?
• Is the aim to cure the cancer, reduce the chance of recurrence, control it, or relieve a particular symptom?
• What benefit do you expect from this option, and what are the reasonable alternatives for me?
• How will treatment affect everyday activities, fertility, sexual health and the things I most value?
• How will we know whether it is working, and when will we review the plan?
• Who should I contact between visits, including at night or over a weekend?

Clinical trials and newer treatments

A clinical trial can be worth considering at diagnosis or when treatment needs to change. Trials ask a defined question; some compare established treatments and others study a newer approach. Eligibility depends on the cancer, previous treatment, test results and health. Taking part is voluntary, and choosing not to participate does not remove your right to standard care. Ask about extra visits, possible randomisation (allocation to a treatment group by chance), travel, side effects and what happens when the study ends.

Newer treatments are considered using the clinical evidence, recognised guidelines and the exact cancer type, biomarkers (features found by laboratory tests) and previous treatment. Some options have less mature evidence than established treatments. Ask what is known, what remains uncertain and whether a clinical trial would be appropriate.

Your priorities and supportive care

Tell the team what you are hoping treatment will make possible: returning to work, caring for family, keeping independence, reducing pain or attending an important event. These priorities can influence a choice between reasonable options. If the likely benefit is small and the burden is substantial, discussing a less intensive approach is appropriate. Decisions can be revisited when your health or circumstances change.

Supportive and palliative care help with symptoms, sleep, mood, family concerns and planning. They can be provided alongside treatment intended to cure or control cancer. A referral does not mean that active treatment is ending. If the cancer cannot be cured, ask what treatments may help you live longer or more comfortably and how their burdens compare with their likely benefits.

Treatment options

Intensive treatment for non-APL AML

A common intensive approach combines cytarabine with an anthracycline, often described as 7+3: cytarabine is given over seven days and the anthracycline over three days in the usual schedule. Additional medicines are selected for the AML subtype. Gemtuzumab ozogamicin may be added in suitable disease with the CD33 protein on its cells, including selected core-binding-factor AML. It has particular liver and transplant-related safety considerations.

For FLT3-mutated AML, a FLT3 inhibitor may be added to an appropriate intensive programme. Midostaurin and quizartinib have different eligible populations; quizartinib is directed at an FLT3 internal tandem duplication (FLT3-ITD), a repeated piece of the gene and requires careful heart-rhythm and interaction monitoring. These drugs are not substitutes for confirming the mutation and selecting the complete regimen.

Liposomal daunorubicin with cytarabine, often called CPX-351, is an option for specified AML arising after earlier cancer treatment or with myelodysplasia-related features. Liposomal means that the medicines are packaged together in tiny fat-like particles. Its formulation and schedule are different from ordinary daunorubicin and cytarabine. The team should explain why that formulation fits the diagnosis rather than treating it as a simple brand substitution.

Less intensive initial treatment

Azacitidine or decitabine with venetoclax is widely used for appropriate adults who are not suitable for intensive induction. Oral decitabine with cedazuridine plus venetoclax is also an option for newly diagnosed adults aged 75 or older, or with other illnesses that make intensive induction unsuitable. An oral regimen still requires tumour-lysis prevention, frequent blood tests and close assessment of marrow recovery.

Venetoclax interacts strongly with several antifungal medicines and other drugs commonly used during AML care. The haematology team adjusts its dose and duration accordingly. Do not copy a dose from another patient's schedule or continue tablets beyond the prescribed days. Marrow response and count recovery may lead to shorter exposure in later cycles.

For suitable newly diagnosed IDH1-mutated AML, ivosidenib with azacitidine is another targeted pathway, particularly when intensive induction is unsuitable. Other options are chosen according to molecular findings, health and prior treatment. No single less intensive combination is appropriate for every older patient.

Acute promyelocytic leukaemia

APL treatment centres on ATRA and arsenic trioxide, with extra treatment to bring down the number of leukaemia cells for appropriate higher-risk presentations. It also requires intensive support for clotting abnormalities, platelets and infection. The white-cell count at presentation helps determine the treatment approach. ATRA is a medicine related to vitamin A; it is not a supplement to take yourself.

ATRA and arsenic can cause differentiation syndrome, a serious inflammatory reaction as leukaemia cells mature. Fever, shortness of breath, rapid weight gain, swelling or low blood pressure must be reported immediately. Arsenic treatment also requires electrolyte and heart-rhythm monitoring. Although many people with APL are cured, the early period can be dangerous and specialist supervision is essential.

Follow-up includes molecular monitoring of PML::RARA at protocol-defined points. A result needs interpretation in relation to treatment timing; early persistence does not always have the same significance as molecular relapse after a completed response.

After remission

Consolidation may use cytarabine-based chemotherapy, a donor transplant or another risk-adapted approach. An oral azacitidine maintenance formulation is used for selected adults in first remission after intensive induction who cannot complete intensive curative therapy. It is not interchangeable with injectable azacitidine and is not a general replacement for transplant in everyone.

Some targeted treatment programmes include a continuation or maintenance phase. The evidence and eligibility depend on the specific drug and setting. Ask whether the medicine is part of the established initial regimen, a post-transplant strategy or a different later-line plan.

If AML returns or does not respond

Refractory AML has not responded adequately to treatment. Relapsed AML has returned after remission. In either situation, repeat gene testing may reveal a useful target or show that the disease has changed. Treatment choice considers the earlier regimen, remission duration, fitness and whether transplant is possible. Treatment may use another course aimed at remission, a less intensive combination or a medicine that targets a specific gene change.

Gilteritinib is an option for relapsed or refractory FLT3-mutated AML. Ivosidenib or olutasidenib may be used in appropriate IDH1-mutated relapsed disease, while enasidenib targets IDH2-mutated AML. The drugs differ in side effects and eligibility. Differentiation syndrome is an important risk with several of these targeted treatments.

Menin inhibitors block a protein interaction that helps certain leukaemia cells keep growing. They provide a further option for particular gene-defined subtypes. Revumenib is used for relapsed or refractory acute leukaemia with a KMT2A translocation and for relapsed or refractory AML with a susceptible NPM1 mutation when there is no satisfactory alternative. Its eligible age range includes patients aged one year and older.

Ziftomenib is an option for adults with relapsed or refractory susceptible NPM1-mutated AML without a satisfactory alternative. Neither medicine is a general treatment for all AML, and a repeated segment within KMT2A, called a partial tandem duplication, is not the same as a qualifying translocation, in which chromosome material has been rearranged.

These medicines require close monitoring, including differentiation syndrome, heart-rhythm effects where relevant and drug interactions. A response may provide a route to transplant in selected patients. If remission cannot be achieved safely, supportive care and symptom-focused treatment remain important options.

Managing side effects

Low counts, infection and bleeding

Blood counts often fall substantially during AML treatment. Antibiotics, antifungals, antiviral prevention and blood-product support are tailored to the regimen and individual risk. Follow the team's fever instructions, even if you have already started preventive medicines. Prevention reduces risk but does not exclude serious infection.

Avoid rectal thermometers and discuss procedures that may cause bleeding or infection when counts are low. Check before taking anti-inflammatory pain medicines or supplements. Ask the team about oral care and dental problems, because mouth ulcers and infection can make eating difficult during intensive treatment.

Tumour lysis and differentiation syndrome

Rapid destruction of leukaemia cells can disturb potassium, phosphate, calcium and uric acid and damage the kidneys. Prevention may include hydration and uric-acid-lowering medicine, with frequent laboratory monitoring. The fluid plan must consider heart and kidney function. Reduced urine, palpitations, muscle cramps or sudden weakness need prompt assessment.

Differentiation syndrome is a different complication of APL treatment and some targeted medicines. It can resemble infection or fluid overload. Fever, weight gain, swelling, chest discomfort or breathing difficulty during these therapies should be assessed urgently rather than managed by changing tablets at home.

Recovery between cycles

Tiredness, nausea, mouth soreness, altered taste and reduced appetite are common but treatable. Dietitian and rehabilitation input can help preserve strength. Some patients recover at home between courses; others need longer inpatient support. Discharge means that a home-care plan is appropriate, not that infection risk has disappeared.

If a donor transplant is performed, the team will provide additional instructions about graft-versus-host disease, infections, vaccination and immunosuppressive medicines. New rash, diarrhoea, jaundice, dry eyes or breathing changes after transplant should be reported. Do not stop immune-suppressing medicine without transplant-team advice.

Keeping treatment manageable

Keep the team's contact details available and know where to go outside clinic hours. Report side effects early rather than waiting until the next appointment. A short record of symptoms, temperature, fluid intake and medicines can help the team decide what is needed. Do not change cancer medicine doses, restart a stopped treatment, or use leftover antibiotics without instructions. A planned delay or dose adjustment can be an important part of safe treatment.

If you receive treatment that can lower white blood cells, a temperature of 38°C (100.4°F) or higher needs urgent advice. Follow any lower threshold or additional instructions your team gives you. Chills, sudden weakness, confusion or feeling very unwell also need urgent assessment, even without a fever. Do not wait for a blood test appointment or try to hide the fever with medicine before speaking to the team.

Severe breathlessness, chest pain, a new seizure, sudden weakness, heavy bleeding or collapse require emergency care. New severe back pain with leg weakness, numbness around the bottom, or loss of bladder or bowel control also needs immediate assessment. Contact the nearest emergency service when necessary; a website contact form or routine messaging service is not an emergency pathway.

Eating, drinking and energy

Treatment may change appetite, taste, bowel habits and energy. Small, frequent meals and foods containing protein can be easier than large meals. Ask for dietitian support if weight is falling, swallowing is difficult, or you are avoiding many foods. There is no single diet that treats cancer, and severe restriction or prolonged fasting can make it harder to tolerate treatment. Check supplements with your team because some interact with medicines.

Take anti-sickness medicine as prescribed and report vomiting that prevents you keeping down fluids or essential medicines. Seek advice for persistent diarrhoea, dizziness, very little urine or increasing weakness. Fluid recommendations may need adjustment if you have heart or kidney disease. Mouth ulcers, pain or infection that interferes with eating should be treated promptly rather than accepted as unavoidable.

Gentle movement, short walks and planned rest may help fatigue, but activity should match your condition and any surgical or bone-safety restrictions. Fatigue can also reflect anaemia, infection, hormone changes, pain, poor sleep or low mood. Let the team assess a marked or persistent change. Accepting help with shopping, transport or meals can conserve energy for the activities that matter to you.

Live well

Follow-up after treatment

Follow-up includes symptoms, examination, blood counts and marrow or molecular testing at appropriate intervals. The schedule is more intensive early on and may change with risk and recovery. New bruising, persistent fever, worsening fatigue or recurrent infections should prompt contact, but these symptoms do not prove relapse on their own.

Keep a record of treatments, transplant details and major complications for future clinicians. Heart, hormone, bone, fertility and psychological effects may need longer-term care. Vaccination after intensive treatment or transplant follows an individual plan because earlier protection may have been reduced.

Returning to everyday life

Recovery is often uneven. Concentration, stamina and confidence may take longer to recover than the blood count. A gradual return to work or study can be easier than trying to resume everything at once. Discuss infection exposure, travel and exercise with the team, particularly while immune recovery is incomplete.

The period after active treatment can bring anxiety because there are fewer hospital visits. Ask for a clear follow-up plan and support for fear of relapse. It is possible to make practical future plans while also acknowledging uncertainty. If the disease remains active, quality-of-life goals and supportive care should remain part of every treatment discussion.

Emotional and practical support

Feeling frightened, angry, sad or uncertain is understandable. You do not have to appear positive all the time. Ask about counselling, psycho-oncology (support for the emotional effects of cancer), support groups or a social worker if worry is affecting sleep, relationships or daily life. Persistent depression, panic or thoughts of self-harm need prompt professional help. Caregivers may need their own support and breaks; one person should not have to manage every appointment and household task alone.

It can help to agree who will update relatives and how much information you want shared. Children usually benefit from honest, age-appropriate explanations and reassurance about who will care for them. Ask permission before discussing private medical information with others. If concentration or memory is difficult, use reminders, a calendar and a trusted companion at important consultations.

Relationships, fertility and sexual health

Cancer and treatment can affect desire, comfort, fertility, body image and confidence. These are valid medical concerns. Raise fertility questions before treatment when possible, because some protective options need to be arranged early. The right approach depends on age, the treatment and how urgently it must start. Do not assume that absent periods or reduced fertility means pregnancy is impossible. Ask about contraception and the safe timing of pregnancy for your specific medicines.

Explain any pain, dryness, erection difficulties or changes in intimacy to the team. Appropriate treatments, rehabilitation and counselling can help. You can choose how much to involve a partner. Advice about hormone-containing treatments needs to take the cancer type and treatment into account; do not start them without discussing suitability.

Planning ahead while living well

Ask for a clear follow-up plan and know which symptoms should be reported between visits. Continue care for other conditions and discuss vaccinations with your oncology team; the timing and suitability of live vaccines can change during treatment. Avoid tobacco and ask for practical cessation support if needed. Activity, food and sleep plans should be realistic for your health rather than another source of pressure.

If treatment is long term, arrange periodic discussions about what is helping, what is difficult and what you want next. Planning for future care can protect your preferences and reduce uncertainty for family. You can discuss who should help with decisions if you become too unwell to speak for yourself, while continuing treatment and everyday life now.

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