The first 60 days: Acute myeloid leukaemia
Acute myeloid leukaemia is an aggressive blood cancer where, after 40 years of the same chemotherapy, a wave of targeted drugs (FLT3, IDH, BCL-2, menin) arrived. Below, week by week, is what OnCo's record of Acute myeloid leukaemia says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Marrow morphology, flow, karyotype/FISH, rapid FLT3/NPM1/IDH testing (results within days), NGS panel; ELN 2022 risk; fitness assessment. Menin-inhibitor and FLT3-inhibitor eligibility depends on these results.
Ask which of these were tested and what the results were; see the report reader for what each value means.
These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.
- PathologistNamed in the standard of care for: Diagnosis and risk assignment.
- Medical oncologistNamed in the standard of care for: Fit, Unfit, Relapsed, Diagnosis and risk assignment and 10 more.
- Transplant and cell therapy teamNamed in the standard of care for: Fit, Relapsed, Fit, FLT3-mutated, Fit, favourable or intermediate risk, CD33-positive and 6 more.
A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.
Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.
7+3 plus fractionated gemtuzumab ozogamicin (ALFA-0701); high-dose cytarabine consolidation; MRD-guided transplant for intermediate risk.
7+3 ± targeted agent; consolidation; allogeneic transplant by risk.
Azacitidine + venetoclax.
- 4.Fit, FLT3-mutatedNCCN category Category 1 (midostaurin, quizartinib), ESMO-MCBS A (RATIFY), NCCN AML 2026 / ESMO 2020
7+3 plus midostaurin (ITD or TKD) or quizartinib (ITD only), consolidation with continued inhibitor, allogeneic transplant for most FLT3-ITD in CR1, post-transplant FLT3-inhibitor maintenance if MRD-positive.
CPX-351 induction (Study 301) then transplant in CR1; alternatives include 7+3 or venetoclax-based therapy in trials.
- 6.Fit, adverse risk (TP53, complex karyotype, MDS-related)NCCN category Category 2A (clinical trial preferred), NCCN AML 2026
Intensive induction or venetoclax-azacitidine to remission, then allogeneic transplant as the only realistic cure; clinical trial strongly preferred; TP53-mutated disease has no effective targeted therapy after the magrolimab and eprenetapopt failures.
- 7.Unfit for intensive chemotherapy (most patients over 75)NCCN category Category 1 (venetoclax + HMA), ESMO-MCBS 4 (VIALE-A), NCCN AML 2026
Venetoclax + azacitidine (VIALE-A) or venetoclax + oral decitabine-cedazuridine (ASCERTAIN-V, 2026); ivosidenib + azacitidine if IDH1-mutated (AGILE); low-dose cytarabine + venetoclax as an alternative. Continue until progression; consider transplant in responders who become fit.
ATRA + arsenic trioxide without chemotherapy for standard risk (cure >95%); ATRA + arsenic + idarubicin or gemtuzumab for high risk. Differentiation syndrome prophylaxis.
Genotype-directed: gilteritinib, IDH inhibitors, menin inhibitors; transplant.
Gilteritinib monotherapy (ADMIRAL) or gilteritinib + venetoclax/azacitidine, then transplant; quizartinib in Japan.
Ivosidenib or olutasidenib (IDH1), enasidenib (IDH2), often with azacitidine or venetoclax; differentiation-syndrome monitoring.
Menin inhibitor: revumenib (KMT2Ar 2024; NPM1 2025) or ziftomenib (NPM1, November 2025), as a bridge to transplant; triplets with venetoclax-azacitidine in trials.
Oral azacitidine (Onureg) for patients not transplanted (QUAZAR AML-001); FLT3 inhibitor maintenance after transplant in FLT3-ITD (MORPHO for MRD-positive); menin inhibitor maintenance in trials.
Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example FLT3-ITD/TKD, NPM1, IDH1/2, KMT2A, TP53), and what were the results?These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include AML with defining genetic abnormalities: NPM1-mutated, CEBPA bZIP, RUNX1::RUNX1T1, CBFB::MYH11, KMT2A-rearranged, DEK::NUP214, BCR::ABL1, MECOM, Acute promyelocytic leukaemia, curable without chemotherapy in most cases, AML, myelodysplasia-related.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Fit
- For my situation (fit), which of the standard options do you recommend and why?Guideline options include: 7+3 ± targeted agent; consolidation; allogeneic transplant by risk.
- Am I a candidate for Gemtuzumab ozogamicin, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Unfit
- For my situation (unfit), which of the standard options do you recommend and why?Guideline options include: Azacitidine + venetoclax.
- Am I a candidate for Venetoclax, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed
- For my situation (relapsed), which of the standard options do you recommend and why?Guideline options include: Genotype-directed: gilteritinib, IDH inhibitors, menin inhibitors; transplant.
- Am I a candidate for Revumenib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Diagnosis and risk assignment
- For my situation (diagnosis and risk assignment), which of the standard options do you recommend and why?Guideline options include: Marrow morphology, flow, karyotype/FISH, rapid FLT3/NPM1/IDH testing (results within days), NGS panel; ELN 2022 risk; fitness assessment. Menin-inhibitor and FLT3-inhibitor eligibility depends on these results.
Fit, FLT3-mutated
- For my situation (fit, flt3-mutated), which of the standard options do you recommend and why?Guideline options include: 7+3 plus midostaurin (ITD or TKD) or quizartinib (ITD only), consolidation with continued inhibitor, allogeneic transplant for most FLT3-ITD in CR1, post-transplant FLT3-inhibitor maintenance if MRD-positive.
- Am I a candidate for Cytarabine + anthracycline ('7+3'), Midostaurin, Quizartinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of RATIFY (CALGB 10603) and QuANTUM-First apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Fit, favourable or intermediate risk, CD33-positive
- For my situation (fit, favourable or intermediate risk, cd33-positive), which of the standard options do you recommend and why?Guideline options include: 7+3 plus fractionated gemtuzumab ozogamicin (ALFA-0701); high-dose cytarabine consolidation; MRD-guided transplant for intermediate risk.
- Am I a candidate for Gemtuzumab ozogamicin, Cytarabine + anthracycline ('7+3'), and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of ALFA-0701 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Fit, secondary or therapy-related AML
- For my situation (fit, secondary or therapy-related aml), which of the standard options do you recommend and why?Guideline options include: CPX-351 induction (Study 301) then transplant in CR1; alternatives include 7+3 or venetoclax-based therapy in trials.
- Am I a candidate for CPX-351 (liposomal daunorubicin-cytarabine), and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of CPX-351 Study 301 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Fit, adverse risk (TP53, complex karyotype, MDS-related)
- For my situation (fit, adverse risk (tp53, complex karyotype, mds-related)), which of the standard options do you recommend and why?Guideline options include: Intensive induction or venetoclax-azacitidine to remission, then allogeneic transplant as the only realistic cure; clinical trial strongly preferred; TP53-mutated disease has no effective targeted therapy after the magrolimab and eprenetapopt failures.
- Am I a candidate for Venetoclax, Azacitidine, Magrolimab, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Unfit for intensive chemotherapy (most patients over 75)
- For my situation (unfit for intensive chemotherapy (most patients over 75)), which of the standard options do you recommend and why?Guideline options include: Venetoclax + azacitidine (VIALE-A) or venetoclax + oral decitabine-cedazuridine (ASCERTAIN-V, 2026); ivosidenib + azacitidine if IDH1-mutated (AGILE); low-dose cytarabine + venetoclax as an alternative. Continue until progression; consider transplant in responders who become fit.
- Am I a candidate for Venetoclax, Azacitidine, Decitabine + cedazuridine (oral) or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of VIALE-A and AGILE apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Maintenance after intensive therapy
- For my situation (maintenance after intensive therapy), which of the standard options do you recommend and why?Guideline options include: Oral azacitidine (Onureg) for patients not transplanted (QUAZAR AML-001); FLT3 inhibitor maintenance after transplant in FLT3-ITD (MORPHO for MRD-positive); menin inhibitor maintenance in trials.
- Am I a candidate for Azacitidine, Gilteritinib, Quizartinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed or refractory, FLT3-mutated
- For my situation (relapsed or refractory, flt3-mutated), which of the standard options do you recommend and why?Guideline options include: Gilteritinib monotherapy (ADMIRAL) or gilteritinib + venetoclax/azacitidine, then transplant; quizartinib in Japan.
- Am I a candidate for Gilteritinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of ADMIRAL apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Relapsed or refractory, IDH-mutated
- For my situation (relapsed or refractory, idh-mutated), which of the standard options do you recommend and why?Guideline options include: Ivosidenib or olutasidenib (IDH1), enasidenib (IDH2), often with azacitidine or venetoclax; differentiation-syndrome monitoring.
- Am I a candidate for Ivosidenib, Olutasidenib, Enasidenib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed or refractory, NPM1-mutated or KMT2A-rearranged
- For my situation (relapsed or refractory, npm1-mutated or kmt2a-rearranged), which of the standard options do you recommend and why?Guideline options include: Menin inhibitor: revumenib (KMT2Ar 2024; NPM1 2025) or ziftomenib (NPM1, November 2025), as a bridge to transplant; triplets with venetoclax-azacitidine in trials.
- Am I a candidate for Revumenib, Ziftomenib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of AUGMENT-101 and KOMET-001 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Acute promyelocytic leukaemia
- For my situation (acute promyelocytic leukaemia), which of the standard options do you recommend and why?Guideline options include: ATRA + arsenic trioxide without chemotherapy for standard risk (cure >95%); ATRA + arsenic + idarubicin or gemtuzumab for high risk. Differentiation syndrome prophylaxis.
Any stage
- Are there clinical trials I could join, for example of Revumenib, Venetoclax, AK117, HMPL-306?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?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?Supportive care improves quality of life and helps patients complete treatment.
- I read that “TP53-mutant AML: no drug class has yet improved survival, so it is the priority for new mechanisms”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Older patients”. How does that affect my plan?Open problems are where trials and second opinions matter most.
Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.
- A Pivotal Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in the Treatment of Acute Myeloid LeukemiaPhase 3 · recruiting · NCT06389292A Global, Multicenter, Randomized, Double-blind, Phase 3 Pivotal Registrational Clinical Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia (GLORA-3)
- A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase 3 (FLT3) MutationPhase 3 · active · NCT03182244Phase 3 Open-label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation
- A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)Phase 3 · recruiting · NCT06852222A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Study of Bleximenib, Venetoclax and Azacitidine for the Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia Harboring KMT2A Rearrangements or NPM1 Mutations Who Are Ineligible for Intensive Chemotherapy
- A Study to Evaluate HMPL-306 in Patients With IDH1or IDH2-mutated Acute Myeloid LeukemiaPhase 3 · recruiting · NCT06387069A Multicenter, Randomized, Open-Label, Phase III Clinical Study to Evaluate the Efficacy and Safety of HMPL-306 vs. Salvage Chemotherapy Regimens in Patients With IDH1- and IDH2-mutated Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
- An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.Phase 3 · recruiting · NCT05907057A Single Arm, Open-label Phase 3b Study to Describe the Safety and Tolerability of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Acute myeloid leukaemia: the full pageAcute myeloid leukaemia is an aggressive blood cancer where, after 40 years of the same chemotherapy, a wave of targeted drugs (FLT3, IDH, BCL-2, menin) arrived.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Guidelines comparedNCCN, ESMO and NICE side by side for this cancer.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- 7+3 induction chemotherapy: The classic first treatment for acute myeloid leukaemia, unchanged since 1973: seven days of continuous cytarabine plus three days of an anthracycline, given in hospital.
- FLT3-ITD allelic ratio: How much of the FLT3 gene in the leukaemia carries the internal duplication, measured as the ratio of mutant to normal copies.
- Metabolic theory of cancer: from Warburg to oncometabolites: Otto Warburg noticed a century ago that cancer cells ferment glucose even when oxygen is plentiful and concluded that damaged respiration causes cancer.
- Differentiation syndrome: When a targeted drug makes leukaemia cells mature all at once, causing fever, fluid in the lungs, and weight gain.
- Epigenetic progenitor theory: cancer without a first mutation: The proposal that cancer begins not with a mutation but with a reversible change in how genes are switched on and off in a stem or progenitor cell, which then makes later mutations more likely and more dangerous.
- Ageing tissue and clonal fields: cancer as a disease of old tissue: Sequencing of healthy skin, gullet and blood shows that by middle age they are patchworks of mutant clones, many carrying classic cancer mutations, yet cancer stays rare until old age.
- Cancer stem cell theory and phenotypic plasticity: The idea that a tumour is organised like a tissue, with a small pool of stem-like cells that renew it and a bulk that cannot, so killing the bulk shrinks the tumour but the stem-like cells regrow it.
- ELN 2022 risk classification: The three-tier system (favourable, intermediate, adverse) that decides how aggressively an adult with AML is treated and whether a transplant is recommended.
- Allogeneic stem cell transplant (allo-SCT): Replacing a patient's blood system with a donor's: chemotherapy wipes out the marrow, donor stem cells rebuild it, and the donor's immune cells hunt down leftover leukaemia.
- Consolidation therapy: Treatment given after a good response to kill the cancer cells that are presumably left but cannot be seen, to make the remission last.
Every term links to the glossary.