The first 60 days: IDH1- and IDH2-mutated acute myeloid leukaemia
IDH-mutated acute myeloid leukaemia has a faulty metabolic enzyme that floods cells with a chemical that blocks maturation. Pills that shut the enzyme off, ivosidenib for IDH1 and enasidenib or olutasidenib for IDH2 and IDH1, let the leukaemia cells mature, and ivosidenib with azacitidine tripled survival in older patients. Below, week by week, is what OnCo's record of IDH1- and IDH2-mutated 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.
- Newly diagnosed IDH1-mutated, unfit for intensive chemotherapyNCCN Guidelines: Acute Myeloid Leukemia
Ivosidenib plus azacitidine (AGILE) or venetoclax plus azacitidine; triplets in trials.
7+3 induction with consolidation and transplant by ELN risk; IDH inhibitors added in trials.
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.
- Medical oncologistNamed in the standard of care for: Newly diagnosed IDH1-mutated, unfit for intensive chemotherapy, Newly diagnosed, fit for intensive chemotherapy, Relapsed or refractory.
- Transplant and cell therapy teamNamed in the standard of care for: Newly diagnosed, fit for intensive chemotherapy, Relapsed or refractory.
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.
Ivosidenib or olutasidenib for IDH1, enasidenib for IDH2; venetoclax-based combinations; transplant in responders.
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 IDH1 R132 and IDH2 R140/R172 mutations, 2-hydroxyglutarate level, NPM1, DNMT3A and RAS co-mutations, ELN 2022 risk group, IDH mutation clearance as MRD), 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 IDH1 R132-mutated AML, IDH2 R140- and R172-mutated AML, IDH-mutated AML with NPM1 co-mutation.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Newly diagnosed IDH1-mutated, unfit for intensive chemotherapy
- For my situation (newly diagnosed idh1-mutated, unfit for intensive chemotherapy), which of the standard options do you recommend and why?Guideline options include: Ivosidenib plus azacitidine (AGILE) or venetoclax plus azacitidine; triplets in trials.
- Am I a candidate for Ivosidenib, Azacitidine, Venetoclax, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of AGILE and VIALE-A apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Newly diagnosed, fit for intensive chemotherapy
- For my situation (newly diagnosed, fit for intensive chemotherapy), which of the standard options do you recommend and why?Guideline options include: 7+3 induction with consolidation and transplant by ELN risk; IDH inhibitors added in trials.
- Am I a candidate for Cytarabine + anthracycline ('7+3'), Ivosidenib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed or refractory
- For my situation (relapsed or refractory), which of the standard options do you recommend and why?Guideline options include: Ivosidenib or olutasidenib for IDH1, enasidenib for IDH2; venetoclax-based combinations; transplant in responders.
- Am I a candidate for Ivosidenib, Olutasidenib, Enasidenib or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Olutasidenib, Venetoclax, myeloMATCH, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)?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 “Ivosidenib-azacitidine or venetoclax-azacitidine first, or all three together”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Managing differentiation syndrome without stopping an effective drug”. 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.
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- IDH1- and IDH2-mutated acute myeloid leukaemia: the full pageIDH-mutated acute myeloid leukaemia has a faulty metabolic enzyme that floods cells with a chemical that blocks maturation. Pills that shut the enzyme off, ivosidenib for IDH1 and enasidenib or olutasidenib for IDH2 and IDH1, let the leukaemia cells mature, and ivosidenib with azacitidine tripled survival in older patients.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- Differentiation syndrome: When a targeted drug makes leukaemia cells mature all at once, causing fever, fluid in the lungs, and weight gain.
- Hypomethylating agents (azacitidine, decitabine): Low-intensity chemotherapy that strips chemical 'off' switches (methyl groups) from DNA so silenced genes can be read again.
- 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.
Every term links to the glossary.