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.
Acute myeloid leukaemia is a cancer of immature myeloid cells that floods the marrow and blood within weeks. It is defined molecularly: WHO 2022 and ICC 2022 classify by driver genetics, and ELN 2022 assigns favourable, intermediate, or adverse risk from NPM1, CEBPA, core-binding-factor fusions, FLT3, TP53, KMT2A, and myelodysplasia-related mutations. Median age at diagnosis is 68, and outcomes diverge sharply by age and fitness.
Treatment split into two paradigms. Fit patients receive intensive 7+3 induction (unchanged since 1973) with a targeted add-on chosen by genetics: midostaurin or quizartinib for FLT3, gemtuzumab ozogamicin for CD33+ favourable and intermediate risk, CPX-351 for secondary AML, then high-dose cytarabine consolidation and allogeneic transplant for adverse or MRD-positive disease. Unfit patients, once offered only supportive care, now receive venetoclax with azacitidine (VIALE-A) or, since May 2026, an all-oral regimen with decitabine-cedazuridine; IDH1-mutated patients may receive ivosidenib-azacitidine (AGILE). Relapse is treated by genotype: gilteritinib (FLT3), ivosidenib, olutasidenib or enasidenib (IDH), and the new menin inhibitors revumenib and ziftomenib (NPM1-mutated or KMT2A-rearranged), with transplant as the consolidating cure.
Measurable residual disease by flow cytometry, NPM1 qPCR, or error-corrected NGS now decides transplant and maintenance, and platform trials such as myeloMATCH assign therapy from rapid genomics at diagnosis. The unsolved problems are TP53-mutated and complex-karyotype AML, where every new class has failed so far, relapse after transplant, early death from infection in older patients, and the cost and logistics of the increasingly individualised pathway.
State of the art today
- Menin inhibitors.
- Venetoclax combinations.
- Genotype-directed induction: FLT3 inhibitors (two positive phase 3 trials), gemtuzumab for CD33+ favourable/intermediate risk, CPX-351 for secondary AML.
- Venetoclax + hypomethylating agent made unfit AML treatable, and since May 2026 the regimen can be fully oral.
- Menin inhibitors (revumenib 2024/2025, ziftomenib 2025) opened NPM1-mutated and KMT2A-rearranged leukaemia, ~40% of adult AML, to a new drug class within one year.
- MRD by flow and molecular methods now guides transplant, maintenance, and pre-emptive therapy; ELN 2021 MRD standards are in routine use.
- Platform trials (myeloMATCH) assign therapy from rapid genomics at diagnosis and re-assign at each MRD checkpoint.
- Allogeneic transplant is safer (post-transplant cyclophosphamide, haploidentical donors) and increasingly followed by targeted maintenance.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- About 20,000 new cases a year in the US and 120,000 worldwide; median age 68; 5-year survival roughly 30% overall, above 60% in favourable-risk younger adults and below 10% in TP53-mutated disease.
Where the cases are
Site: Leukaemia (all types) (shared total; subtype split not reported). World: 487,294 new cases, 305,405 deaths.
| # | Country | New cases | Deaths | Incidence ASR |
|---|---|---|---|---|
| 1 | China | 81,946 | 50,074 | |
| 2 | United States of America | 63,144 | 23,460 | |
| 3 | India | 49,883 | 36,871 | |
| 4 | Germany | 15,108 | 9,378 | |
| 5 | Russian Federation | 14,495 | 8,224 | |
| 6 | Indonesia | 13,959 | 10,370 | |
| 7 | Japan | 13,572 | 10,066 | |
| 8 | France (metropolitan) | 13,525 | 7,387 | |
| 9 | Brazil | 11,859 | 8,790 | |
| 10 | Italy | 10,799 | 7,197 |
GLOBOCAN reports leukaemia as one site; AML is roughly a quarter of cases worldwide.
7+3 ± targeted agent; consolidation; allogeneic transplant by risk.
Genotype-directed: gilteritinib, IDH inhibitors, menin inhibitors; transplant.
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.
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.
7+3 plus fractionated gemtuzumab ozogamicin (ALFA-0701); high-dose cytarabine consolidation; MRD-guided transplant for intermediate risk.
CPX-351 induction (Study 301) then transplant in CR1; alternatives include 7+3 or venetoclax-based therapy in trials.
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.
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.
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.
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.
ATRA + arsenic trioxide without chemotherapy for standard risk (cure >95%); ATRA + arsenic + idarubicin or gemtuzumab for high risk. Differentiation syndrome prophylaxis.
Subtypes & biomarkers
top- AML with defining genetic abnormalities (WHO 2022): NPM1-mutated, CEBPA bZIP, RUNX1::RUNX1T1, CBFB::MYH11, KMT2A-rearranged, DEK::NUP214, BCR::ABL1, MECOM
- Acute promyelocytic leukaemia (PML::RARA), curable without chemotherapy in most cases (ATRA + arsenic)
- AML, myelodysplasia-related (mutations in ASXL1, BCOR, EZH2, RUNX1, SF3B1, SRSF2, STAG2, U2AF1, ZRSR2 or MDS-type cytogenetics)
- TP53-mutated AML / complex karyotype (adverse; ~10-15%)
- Therapy-related AML after alkylators or topoisomerase II inhibitors
- FLT3-ITD or FLT3-TKD (30%; targetable)
- IDH1 or IDH2-mutated (15-20%; targetable)
- Blastic plasmacytoid dendritic cell neoplasm (CD123-high; related entity)
- FLT3-ITD/TKD
- NPM1
- IDH1/2
- KMT2A
- TP53
- CD33
- ELN risk
- MRD
- ELN 2022 genetic risk group
- FLT3-ITD and TKD (midostaurin, quizartinib, gilteritinib)
- NPM1 (favourable risk without FLT3-ITD; MRD marker; menin inhibitor eligibility)
- IDH1 / IDH2 (ivosidenib, olutasidenib, enasidenib)
- KMT2A rearrangement (revumenib)
- TP53 and complex karyotype (adverse; venetoclax less effective)
- CD33 (gemtuzumab), CD123 (tagraxofusp, pivekimab)
- Measurable residual disease (flow, NPM1 qPCR, NGS) after cycles 2 and before transplant
- Karyotype/FISH at diagnosis
- Myelodysplasia-related mutation set
Target prevalence in this cancer
| Target / alteration | Prevalence | Measure | Source |
|---|---|---|---|
| CD47 Magrolimab discontinued | >90% | Surface expression on blasts | Wikipedia |
| CD33 | 85-90% | Blast surface expression | Wikipedia |
| CD123 Universal in BPDCN | 70-80% | Blasts and leukaemic stem cells | Wikipedia |
| CD38 | 60-80% | Blast expression, variable intensity | Wikipedia |
| CD70 | 30-50% | Blasts and leukaemic stem cells | Wikipedia |
| NPM1 mutation ~50-60% of cytogenetically normal AML | 30% | mutation | |
| FLT3 | 25-30% | FLT3-ITD or TKD | cBioPortal (TCGA) |
| Menin KMT2A rearrangement ~5-10% | 25-30% | NPM1 mutation | cBioPortal (TCGA) |
| IDH1 / IDH2 | 15-20% | IDH1 or IDH2 mutation | cBioPortal (TCGA) |
| TP53 Higher in therapy-related AML | 8-10% | TP53 mutation | cBioPortal (TCGA) |
| KMT2A (MLL) rearrangement Higher in therapy-related AML | 5-10% | rearrangement | |
| BCL-2 | n/a | Dependency, not a prevalence threshold | Wikipedia |
How common each drug target or alteration is in this cancer. Population-level and approximate; see the target page for detail. Full matrix.
- 1948First chemotherapy remissions in leukaemia
Farber's aminopterin in childhood ALL; AML remissions follow with 6-MP and cytarabine in the 1960s.
- 19737+3 regimen
- 19737+3 induction defined
Cytarabine 7 days + daunorubicin 3 days (Yates et al.); still the intensive backbone.
- 1977First allogeneic transplants cure refractory leukaemia
Thomas (Seattle) reports long-term survival after HLA-matched sibling transplant; Nobel Prize 1990.
- 1988ATRA induces differentiation in APL
Shanghai group shows all-trans retinoic acid remissions; arsenic follows in the 1990s. First differentiation therapy.
- 2000Gemtuzumab: first ADC
- 2000Gemtuzumab ozogamicin, the first ADC
Accelerated approval in relapsed CD33+ AML; withdrawn 2010 after SWOG S0106 toxicity.
- 2004Azacitidine approved (MDS)
First hypomethylating agent; later the AML backbone for venetoclax and IDH/menin combinations.
- 2012ALFA-0701 rescues gemtuzumab
Fractionated dosing with 7+3 improves EFS; re-approval 2017.
- 2017Midostaurin, enasidenib, gemtuzumab re-approval
- 2017Four approvals in one year
Midostaurin (RATIFY), enasidenib, CPX-351, gemtuzumab re-approval; the first new AML drugs since 2000.
- 2018Ivosidenib, gilteritinib, and venetoclax combinations
Ivosidenib (IDH1) and gilteritinib (ADMIRAL) approved; venetoclax + HMA/LDAC gets accelerated approval for unfit AML.
- 2020VIALE-A: venetoclax-azacitidine improves survival in unfit AML
OS 14.7 vs 9.6 months; full approval October 2020; oral azacitidine maintenance approved (QUAZAR).
- 2022ELN 2022 risk, WHO/ICC classifications, AGILE, olutasidenib
Genetics-first classification; ivosidenib-azacitidine OS HR 0.44; second IDH1 inhibitor approved.
- 2023Quizartinib approved in frontline FLT3-ITD AML
QuANTUM-First OS 31.9 vs 15.1 months, including patients to age 75.
- 2024Revumenib: first menin inhibitor
- 2024Revumenib: first menin inhibitor; magrolimab fails
Approved for KMT2A-rearranged acute leukaemia (AUGMENT-101). Magrolimab (CD47) discontinued after ENHANCE trials, a setback for TP53-mutated AML.
- 2025Menin inhibitors reach NPM1-mutated AML
Revumenib NPM1 label (October) and ziftomenib approval (13 November, KOMET-001).
- 2026First all-oral AML regimen
Decitabine-cedazuridine + venetoclax approved 13 May 2026 (ASCERTAIN-V, CR 41.6%).
Open problems
- TP53-mutant AML remains lethal.
- Older patients.
- TP53-mutated and complex-karyotype AML: no class has improved survival; magrolimab (CD47) and eprenetapopt (p53 reactivator) both failed in phase 3.
- Relapse after allogeneic transplant remains the leading cause of death; which maintenance (FLT3, menin, azacitidine) helps whom is unresolved.
- Menin-inhibitor resistance through MEN1 mutations appears within months in a third of relapsing patients; combinations and next-generation inhibitors are needed.
- Early death from infection and cytopenias on venetoclax-based therapy in the very old; optimal venetoclax duration is untested in randomised trials.
- MRD thresholds and assays are not harmonised across labs, and CHIP-associated mutations confound NGS MRD.
- Whether triplets (menin or FLT3 inhibitor + venetoclax + HMA) improve survival over doublets, and at what toxicity, awaits phase 3.
- Access: rapid genomics within 72 hours and menin/FLT3 inhibitors are unavailable in most low- and middle-income settings.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
Expert centres
topCentres linked to this cancer in OnCo
- via Comprehensive genomic profiling
- Cancer Research UK Manchester InstituteManchester, GBvia this cancer, Comprehensive genomic profiling
- HOVONRotterdam, NLvia this cancer, Venetoclax
- Indiana University Melvin and Bren Simon Comprehensive Cancer CenterIndianapolis, IN, USNCI comprehensivevia Allogeneic stem cell transplantation, Cytotoxic chemotherapy
- Institut Paoli-CalmettesMarseille, FRvia this cancer, Comprehensive genomic profiling
- via this cancer, Allogeneic stem cell transplantation
- Rigshospitalet – Copenhagen University HospitalCopenhagen, DKvia this cancer, Comprehensive genomic profiling
- via this cancer, Azacitidine
- Walter and Eliza Hall Institute of Medical ResearchMelbourne, AUvia this cancer, Venetoclax
- A.C. Camargo Cancer CenterSão Paulo, BRvia Comprehensive genomic profiling
- Aichi Cancer CenterNagoya, JPvia Comprehensive genomic profiling
- American Society of HematologyWashington, DC, USvia this cancer
- via this cancer
- via Cytotoxic chemotherapy
- via Comprehensive genomic profiling
- Butaro Cancer Center of ExcellenceButaro, RWvia Cytotoxic chemotherapy
- Centre Léon BérardLyon, FRvia Comprehensive genomic profiling
- via this cancer
- via this cancer
- Chinese PLA General HospitalBeijing, CNvia this cancer
- Christian Medical College, VelloreVellore, INvia this cancer
- City of Hope Orange CountyIrvine, CA, USvia Comprehensive genomic profiling
- Comprehensive Cancer Center Freiburg (CCCF)Freiburg im Breisgau, DEvia this cancer
- via this cancer
- Comprehensive Cancer Center Tübingen-StuttgartTübingen, DEvia this cancer
- European Hematology AssociationThe Hague, NLvia this cancer
- via Allogeneic stem cell transplantation
- via Comprehensive genomic profiling
- GEICAM Spanish Breast Cancer GroupMadrid, ESvia Cytotoxic chemotherapy
- German Hodgkin Study GroupCologne, DEvia Cytotoxic chemotherapy
- German Lymphoma AllianceHomburg, DEvia Cytotoxic chemotherapy
- via Comprehensive genomic profiling
- GIMEMARome, ITvia this cancer
- via this cancer
- Guangdong Provincial People's HospitalGuangzhou, CNvia Comprehensive genomic profiling
- via this cancer
- HealthCare Global EnterprisesBengaluru, INvia Comprehensive genomic profiling
- Hospital Universitari i Politècnic La FeValencia, ESvia this cancer
- Hospital Universitario 12 de OctubreMadrid, ESvia this cancer
- via this cancer
- via Comprehensive genomic profiling
- via Comprehensive genomic profiling
- Institut BergoniéBordeaux, FRvia Comprehensive genomic profiling
- Instituto Alexander FlemingBuenos Aires, ARvia Comprehensive genomic profiling
- Instituto Nacional de Cancerología (Mexico)Mexico City, MXvia Comprehensive genomic profiling
- via Comprehensive genomic profiling
- Intermountain Health Cancer CenterSalt Lake City, UT, USvia Comprehensive genomic profiling
- International Association for the Study of Lung CancerDenver, CO, USvia Comprehensive genomic profiling
- via this cancer
- IRCCS Ospedale San RaffaeleMilan, ITvia this cancer
- Istanbul University Institute of OncologyIstanbul, TRvia Comprehensive genomic profiling
- Japanese Society of Medical OncologyTokyo, JPvia Comprehensive genomic profiling
- Juravinski Cancer Centre / Escarpment Cancer Research InstituteHamilton, ON, CAvia Allogeneic stem cell transplantation
- Keio University HospitalTokyo, JPvia Comprehensive genomic profiling
- Korean Cancer Study GroupSeoul, KRvia Comprehensive genomic profiling
- Kyoto University HospitalKyoto, JPvia this cancer
- Kyushu University HospitalFukuoka, JPvia Comprehensive genomic profiling
- via Comprehensive genomic profiling
- via this cancer
- National Cancer Center Hospital EastKashiwa, Chiba, JPvia Comprehensive genomic profiling
- National Cancer Institute (NIH)Bethesda, MD, USvia myeloMATCH
- National Institute of Oncology, HungaryBudapest, HUvia Comprehensive genomic profiling
- National Taiwan University HospitalTaipei, TWvia this cancer
- via this cancer
- via this cancer
- Nordic Lymphoma GroupStockholm, SEvia Cytotoxic chemotherapy
- via this cancer
- Ontario Institute for Cancer ResearchToronto, ON, CAvia Comprehensive genomic profiling
- Osaka International Cancer InstituteOsaka, JPvia Comprehensive genomic profiling
- via Comprehensive genomic profiling
- via this cancer
- via this cancer
- Queen Mary Hospital / University of Hong KongHong Kong, HKvia this cancer
- Rajiv Gandhi Cancer Institute and Research CentreNew Delhi, INvia Comprehensive genomic profiling
- Ramathibodi Hospital, Mahidol UniversityBangkok, THvia this cancer
- Rambam Health Care CampusHaifa, ILvia this cancer
- Royal Adelaide HospitalAdelaide, AUvia this cancer
- Ruijin Hospital, Shanghai Jiao Tong UniversityShanghai, CNvia this cancer
- via Comprehensive genomic profiling
- Seoul St. Mary's HospitalSeoul, KRvia this cancer
- Siriraj Hospital, Mahidol UniversityBangkok, THvia this cancer
- via this cancer
- Tata Medical Center, KolkataKolkata, INvia this cancer
- Tel Aviv Sourasky Medical CenterTel Aviv, ILvia Comprehensive genomic profiling
- The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research InstituteColumbus, OH, USNCI comprehensivevia this cancer
- Uganda Cancer InstituteKampala, UGvia Cytotoxic chemotherapy
- University Cancer Center Frankfurt (UCT)Frankfurt am Main, DEvia this cancer
- via Cytotoxic chemotherapy
- University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer CenterBaltimore, MD, USNCI comprehensivevia Allogeneic stem cell transplantation
- via Cytotoxic chemotherapy
- via Comprehensive genomic profiling
- via this cancer
- Weizmann Institute of ScienceRehovot, ILvia this cancer
- via this cancer
Questions to ask
topQuestions to ask your oncologist about Acute myeloid leukaemia
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 FLT3-ITD/TKD, NPM1, IDH1/2, KMT2A, TP53), 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 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?Why: Inherited variants can change treatment and matter for relatives.
Fit
- For my situation (fit), which of the standard options do you recommend and why?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?Why: 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?Why: Guideline options include: Azacitidine + venetoclax.
- Am I a candidate for Venetoclax, and what side effects should I expect?Why: 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?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?Why: 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?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?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?Why: 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?Why: 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?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?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of ALFA-0701 apply to someone like me?Why: 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?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?Why: 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?Why: 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?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?Why: 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?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?Why: 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?Why: 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?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?Why: 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?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?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of ADMIRAL apply to someone like me?Why: 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?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?Why: 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?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?Why: 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?Why: 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?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, Ziftomenib, Menin inhibitor + venetoclax + azacitidine?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 “TP53-mutant AML remains lethal”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Older patients”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
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
15targets
17drugs
24companies
17institutions
47pathways
4terms
6trials
11pairings
3ideas
7collections
1people
24bottlenecks
4key papers
6Revumenib proved that a transcriptional dependency, rather than a kinase, can be drugged in leukaemia, opening treatment for two genetic subgroups that together cover roughly a third of AML plus most infant ALL. It is now approved and is being combined with venetoclax-azacitidine and intensive chemotherapy in front-line trials. Single-agent remissions are often short without transplant.
QuANTUM-First gave FLT3-ITD AML patients a second front-line targeted option and showed that continuing a FLT3 inhibitor as long-term maintenance, including after transplant, pays off. Quizartinib was approved for this indication in 2023. Head-to-head data against midostaurin are lacking, and the design leaves open how much of the benefit came from maintenance.
AGILE showed that for the roughly 6-10% of AML patients with an IDH1 mutation, a targeted doublet produces survival in the range of two years, an outcome previously unimaginable in unfit patients. Ivosidenib-azacitidine is approved and is one option alongside venetoclax-azacitidine for these patients. Which regimen, or triplet, is best for IDH1-mutated disease has not been settled by a randomised trial.
VIALE-A turned a palliative regimen into one that produces remission in two-thirds of older AML patients and is now the reference treatment for anyone not fit for intensive chemotherapy. It shifted the field towards lower-intensity targeted combinations and opened the door to adding FLT3, IDH and menin inhibitors to the backbone. Cure remains uncommon and most patients relapse within two years.
ADMIRAL showed that a targeted oral drug can beat chemotherapy outright in relapsed AML, and made gilteritinib the standard bridge to transplant for FLT3-mutated relapse. Its success also underpinned FLT3 inhibitor use in first-line combinations. Resistance through FLT3-independent clones and RAS pathway mutations limits durability without transplant.
Many older people carry blood clones one or two steps from leukaemia, and those clones also drive heart disease through inflammation. CHIP is why blood-based cancer tests must filter out mutations from blood cells, and it opens a route to preventing both leukaemia and cardiovascular events in carriers.
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