This section summarizes five key AML presentations from the European Hematology Association (EHA) Congress 2026. The studies looked at ways to improve existing treatments, including venetoclax and azacitidine, as well as new medicines called menin inhibitors that are being developed for some types of AML.

Together, these studies show how researchers are working to improve treatment by making therapies more effective, reducing side effects where possible, and developing new treatment options that could improve outcomes for people living with AML

All abstracts can be accessed here –> EHA2026 Abstracts

Updates in acute myeloid leukemia (AML)

Azacitidine with reduced duration of venetoclax¹

What was this study about?

This early study (phase II) explored whether giving venetoclax for fewer days could work just as well while reducing side effects and improving quality of life. Azacitidine (aza) was tested together with a shorter duration of venetoclax (ven).

Study overview

Who took part in this study? 152 patients with AML were in this study including newly diagnosed people and people whose disease had come back (relapsed) or hadn’t improved with other treatment (refractory). This was an older group of patients with an average age of between 71–76 years old across the different AML groups.

In standard treatment plans, ven is usually given daily for most of each 28-day treatment cycle. In this study, this was shortened to 14 days during the early cycles and then reduced further to 7 days from cycle 4 onwards.

Key results

In this study 70% of people with newly diagnosed AML had a complete remission. Among people with relapsed or refractory AML, 64% had a complete remission. A complete remission is when there areno signs of cancer cells in the blood or bone marrow after treatment.

On average, people with newly diagnosed AML lived for 15.5 months after treatment and those with relapsed or refractory AML lived for 13 months.

Key safety information

The number of side effects people experienced was similar or slightly lower with the shorter ven treatment schedule compared with the standard ven/aza treatment schedule. The researchers compared the current study results with those of a previous study called VIALE-A which tested ven/aza in untreated AML.

The top 3 most common side effects were:

• 39% of people treated had fever and low white blood cell counts (neutropenia)
• 35% of people treated had low white blood cell counts but no fever
• 22% of people treated had low platelet counts (thrombocytopenia

What does this mean for people with AML?

This study found that reducing the number of days ven was given did not appear to reduce how effective the treatment was when combined with aza. Giving ven for fewer days could also reduce treatment costs compared with the standard ven/aza treatment schedule. Side effects were similar, or slightly lower than those seen with the standard treatment plan.

This approach may reduce the burden of treatment and could improve quality of life, help some patients remain on treatment for longer, and could reduce treatment costs. Researchers are now exploring a tablet-only version of ven/aza to see whether it could make treatment more convenient and improve quality of life further.

Menin inhibitors in AML: Understanding how they work and where they may fit into treatment plans^2–5

Menin inhibitors are one of the most exciting new developments in AML treatment research. These drugs are particularly effective in AML with genetic changes known as KMT2A rearrangements (KMT2A-r) and NPM1 mutations (NPM1m). These genetic changes rely on a protein called menin to keep leukemia cells growing. Menin inhibitors block menin, allowing leukemia cells to mature into more normal blood cells. This slows the growth of the leukemia.

Two menin inhibitors, revumenib and ziftomenib, are now approved for people whose leukemia has returned after treatment (relapsed) or has not responded to previous therapies (refractory). Several newer menin inhibitors including bleximenib and enzomenib are being studied in clinical trials. Results so far have been encouraging, with many patients achieving remission (no sign of cancer cells in the blood or bone marrow) after treatment. Researchers are also exploring whether menin inhibitors can be used earlier in treatment and whether combining them with other therapies, such as venetoclax, FLT3 inhibitors, chemotherapy, or even CAR T-cell therapy, can produce better and longer-lasting responses.

One example presented at EHA 2026 was an early study combining the menin inhibitor ziftomenib with intensive “7+3” chemotherapy in 99 people with newly diagnosed KMT2A-r or NPM1m AML. The results were particularly encouraging, with 92 of 99 (93%) patients achieving complete remission (no cancer cells are found in the blood or bone marrow). Importantly, the number and types of side effects seen were largely similar to those expected with chemotherapy alone. Serious side effects of special interest, including differentiation syndrome (a serious inflammatory condition) and changes in heart rhythm (QTc prolongation), occurred in only a small number of patients and were successfully managed. These findings support larger ongoing studies and suggest that menin inhibitors could eventually be used earlier in treatment rather than being reserved for patients whose AML has returned after previous therapy.

Despite these advances, one of the biggest challenges is that some leukemia cells can become resistant to menin inhibitors over time. This means the treatment may become less effective, allowing the leukemia to start growing again. There are several ways this can happen. For example, leukemia cells can develop new genetic changes (mutations) that make them less responsive to treatment. Some leukemia cells can also survive by entering a dormant “drug-tolerant persister” state. These cells aren’t killed by treatment and could act as a source of cells that allow the disease to come back (relapse). Understanding how resistance develops is helping researchers identify new treatment targets and design new ways to tackle resistance when it occurs.

One of the most promising approaches is the use of combination therapies. Early studies combining menin inhibitors with venetoclax-based treatment have shown particularly high response rates in both newly diagnosed and relapsed AML. Researchers are also studying menin inhibitors in combination with newer drugs such as KAT6A/7 inhibitors, which may block other processes that leukemia cells use to survive and grow. As a result of all this research, menin inhibitors have rapidly progressed from laboratory research to an important new treatment option. While challenges remain, particularly around resistance and long-term disease control, the research presented at EHA 2026 suggests that menin inhibitors are likely to play an increasingly important role in AML treatment. Ongoing studies will help determine how they can be used most effectively and which patients are most likely to benefit.

Updates in acute promyelocytic leukemia (APL)

Understanding how arsenic trioxide (ATO) treatment affects quality of life in people with APL⁶

What is the study about?

This large phase III study examined the impact of ATO on quality of life and fatigue in people with high-risk APL. A treatment combination of all-trans retinoic acid (ATRA), ATO and idarubicin was compared with ATRA plus chemotherapy.

Study overview

96 people with newly diagnosed high-risk APL completed questionnaires about their quality of life and fatigue during treatment. The average age of this group was 42 years old.

Key results

People treated with ATRA-ATO reported better quality of life in four areas than those treated with ATRA plus chemotherapy.

These included:

1. Physical functioning – how well people could move their bodies
2. Role functioning – how well people felt they could perform daily activities
3. Social functioning – how well people felt able to socialize
4. Overall quality of life

In terms of specific symptoms, people in this study reported:

• Feeling less fatigued (tiredness)
• Feeling less sick (nauseous) or being sick less
• An improved appetite

Following these results the team plans to investigate whether using a tablet form of ATO provides further quality of life benefits for people with APL.

What does this mean for people with APL?

This study showed that people treated with ATRA-ATO reported better quality of life and less fatigue compared with those treated with ATRA and chemotherapy. Along with previously published safety and effectiveness results, these findings further support using ATRA-ATO as one of the first treatment options in people with newly diagnosed high-risk APL. It also supports further study into what potential quality of life benefits could be gained from a tablet version of ATO for people with APL.

References

1. Tølbøll Sørensen, AL. Presentation at the EHA 2026 Congress, Stockholm, Sweden, June 11, 2026. Abstract S129.
2. Kühn M. Presentation at the EHA 2026 Congress, Stockholm, Sweden, June 12, 2026. Presentation ID p112-1.
3. Armstrong S. Presentation at the EHA 2026 Congress, Stockholm, Sweden, June 12, 2026. Presentation ID p112-2.
4. Garciaz S. Presentation at the EHA 2026 Congress, Stockholm, Sweden, June 12, 2026. Presentation ID p112-3.

6 studies explained : What the New AML Research Means for Patients

A Quick Word Before You Read

This summary translates six scientific studies presented at the European Hematology Association (EHA) 2026 Congress into everyday language. It is not medical advice. Every patient’s situation is different — please always talk to your own doctor before making any decisions about your treatment.

Study 1: OPTI-AML – How Long Should Venetoclax Be Given?

Who is this for? Older patients (60+) with newly diagnosed AML who cannot tolerate intensive chemotherapy — for example, due to age or other health conditions.

What did the study ask?

The standard treatment for this group combines two drugs: azacitidine and venetoclax. The question was simple: does venetoclax need to be given for the full 28 days of each cycle, or is 14 days enough?

What did they find?

• The 28-day schedule worked better overall: more patients went into full remission (no detectable leukemia in the bone marrow) — 49% vs. 43%.
• However, the difference was not large enough to statistically prove the 14-day schedule is clearly inferior. The study was designed to answer that question definitively, and it couldn’t.
• Patients with specific gene mutations (NPM1 or IDH1/2) did noticeably better with 28 days (61% vs. 42% remission with NPM1).
• The depth of remission (whether tiny amounts of leukemia cells remained undetectable) was equally good in both groups.
• A practical problem: only 68% of patients in the 28-day group actually completed the full course, mainly because infections interrupted treatment.

What does this mean for patients? The 28-day schedule remains the standard — but it is harder to complete. For patients with an NPM1 or IDH1/2 mutation, the longer schedule appears especially important. For patients and doctors considering shortening treatment due to side effects, this study suggests caution: the shorter version has not been proven to be equally effective.

Patient perspective: This study is reassuring in one way — the deep response rates (MRD negativity) were similar between both groups, which is the thing that matters most for long-term outcomes. But it also highlights a real challenge: getting through the full 28 days is tough, and infections are a major obstacle. Better infection prevention and management during treatment will be key.

Study 2: KOMET-007 – A New Drug (Ziftomenib) Added to Standard Chemo

Who is this for? Younger or fitter adults (18+) with newly diagnosed AML and either an NPM1 mutation or a KMT2A rearrangement, who are fit enough for intensive chemotherapy.

What is Ziftomenib?

Ziftomenib is a targeted drug called a menin inhibitor. It blocks a protein (menin) that helps leukemia cells grow. In NPM1 and KMT2A-related AML, this protein plays a particularly important role — making these patients good candidates for this drug.

What did they find?

The results were striking:

• 93% of patients achieved a composite complete remission (meaning no or nearly no detectable leukemia)
• 88% achieved a full complete remission
• 83% had no detectable remaining leukemia cells at a very sensitive level (MRD negativity)
• After one year, 94% of NPM1-mutated patients were still alive — a very high figure for AML
• Side effects were mostly expected chemotherapy effects (fever with low white cell counts, low platelets). Serious specific side effects of ziftomenib — including differentiation syndrome and heart rhythm changes — occurred in only 3–4% of patients and were manageable.

What does this mean for patients? This is one of the most exciting results in newly diagnosed AML in years. Remission rates of 88–96% are very high by historical standards. If confirmed in the ongoing larger Phase 3 trial (KOMET-017), ziftomenib could become a new standard treatment for patients with NPM1 or KMT2A mutations who receive intensive chemotherapy.

Patient perspective: These numbers are genuinely impressive. For patients with NPM1 mutation in particular, achieving 94% one-year survival is remarkable. The safety profile also appears manageable. Patients with these mutations should ask their doctor whether they might be eligible for the follow-up Phase 3 trial or compassionate access programs.

Study 3: AK117 – An Immune System Booster Added to Standard Therapy

Who is this for? Older or frailer patients (75+, or younger with serious other health conditions) with newly diagnosed AML who cannot have intensive chemotherapy. Standard treatment for this group is azacitidine + venetoclax.

What is AK117?

AK117 is a new type of drug called a CD47 inhibitor (also called an “anti-phagocytic checkpoint inhibitor”). Here’s the idea in plain language: leukemia cells carry a signal on their surface that says “don’t eat me” to the immune system. AK117 blocks that signal, so the immune system’s cleanup cells (macrophages) can recognize and destroy the leukemia cells.

What did they find?

This was a small Phase 2 study (60 patients), so results are preliminary:

• Remission rates were similar between the two groups at 2 months (57% vs. 53%)
• But patients on AK117 stayed in remission longer (10.4 vs. 6.5 months)
• 9-month survival was strikingly different: 79% (AK117) vs. 43% (placebo)
• Fewer patients died from treatment-related causes in the AK117 group (17% vs. 30%)
• Side effects were broadly comparable, with no new safety concerns

What does this mean for patients? The survival difference at 9 months (79% vs. 43%) is striking — but this was a small study and the results need to be confirmed in larger trials. The drug did not cause extra anemia beyond what was already expected from the standard drugs.

Patient perspective: The 9-month survival difference is the most eye-catching number here, and it gives real hope. However, the study is small (30 patients per group), so it’s too early to draw firm conclusions. Larger trials are needed. Patients in this category should ask their doctors whether they might be eligible for trials combining AK117 or similar CD47 inhibitors with standard therapy.

Study 4: APOLLO – Quality of Life With Chemotherapy-Free Treatment for APL

Who is this for? Patients with acute promyelocytic leukemia (APL) — a rare and special subtype of AML — particularly high-risk APL. This study focused on how patients felt during treatment.

Background

APL has a chemotherapy-free treatment option: ATRA + arsenic trioxide (ATO). It was already known to work at least as well medically as ATRA + traditional chemotherapy. But did patients actually feel better on it? That’s what this study measured.

What did they find?

After the consolidation phase of treatment (the follow-up treatment cycles after the initial intensive phase), patients on the chemotherapy-free combination had:

• Significantly less fatigue (the difference was more than double the minimum patients would notice)
• Better physical ability to do daily activities
• Less nausea and vomiting
• Less loss of appetite
• Better social functioning and overall quality of life

During the initial intensive treatment phase, both groups felt similarly. The quality of life advantages emerged during the consolidation cycles.

What does this mean for patients? For patients with high-risk APL, the chemotherapy-free approach doesn’t just match chemotherapy for effectiveness — it also makes patients feel noticeably better during treatment. For many patients, this will tip the balance firmly in favour of ATRA+ATO.

Patient perspective: This study is especially important because it captures what patients actually experience — not just whether their blood tests look good. Fatigue, nausea, and the ability to carry on with life during treatment matter enormously to patients and families. Having evidence that the gentler treatment is also the better-tolerated one is powerful.

Study 5: Quizartinib vs. Midostaurin – Which FLT3 Drug Is Better?

Who is this for? Patients with newly diagnosed AML and a FLT3-ITD mutation (present in about 25–30% of AML cases), who are fit enough for intensive chemotherapy.

Background

Two drugs — midostaurin and quizartinib — are both approved to be added to intensive chemotherapy in FLT3-mutated AML. But they had never been directly compared in real patients. This study did exactly that — looking at real-world data from 215 patients at 12 US hospitals.

What did they find?

• 1-year survival: 93% (quizartinib) vs. 78% (midostaurin)
• 1-year event-free survival: 77% vs. 56%
• Remission rates: 85% vs. 73%
• Quizartinib had fewer treatment interruptions (11% vs. 25%) and fewer ICU admissions (5% vs. 15%)
• Side effects during treatment were broadly similar
• The FLT3 mutation was cleared from the blood at similar rates in both groups

What does this mean for patients? This is important real-world evidence suggesting quizartinib may produce better outcomes than midostaurin in FLT3-ITD AML. Both drugs are approved, but this study provides the first meaningful direct comparison. The advantage for quizartinib was seen across survival, remission, and tolerability during induction.

Patient perspective: For FLT3-ITD AML patients, it is worth having a specific conversation with their doctor about which FLT3 inhibitor they plan to use, and why. This study is not a randomized clinical trial (patients weren’t randomly assigned), so it has limitations — but it’s the best comparison we have. Asking “would quizartinib be appropriate for me?” is a very reasonable question.

Study 6: PALOMA – Better “Bridge” Treatment Before Stem Cell Transplant

Who is this for? Patients with higher-risk MDS (myelodysplastic syndrome — a blood disorder that can progress to AML) or AML with a low blast count (≤29%), for whom an allogeneic stem cell transplant (receiving healthy stem cells from a donor) is planned within 6 months.

Background

Before transplant, patients need treatment to get the disease under control. The standard options are azacitidine (a gentler drug) or standard intensive chemotherapy. This study tested CPX-351 — a specially formulated liposomal version of two chemotherapy drugs — as an alternative “bridge” to transplant.

What did they find?

• Event-free survival was significantly longer with CPX-351: the median EFS was not yet reached vs. 11.1 months with standard therapy
• The same proportion of patients in both groups (85–86%) successfully went on to receive the transplant
• Overall survival showed a trend in favour of CPX-351, but was not yet statistically significant
• Side effects were similar to what would be expected from intensive treatment in both groups
• Fewer patients had disease that failed to respond to treatment (primary refractory disease): 8 vs. 17

What does this mean for patients? For patients in this category and transplant is being planned, CPX-351 may give a better chance of reaching transplant in good condition — and staying disease-free longer. The transplant rate was the same in both groups, which is reassuring: the more intensive drug didn’t make it harder to get to transplant.

Patient perspective: The key message is that the “bridge” treatment matters — it’s not just a waiting room before transplant. Choosing the right treatment at this stage can meaningfully affect how long you stay disease-free. Patients should ask whether CPX-351 is available and appropriate for them.

Quality of Life Before Treatment Can Predict Outcomes — EORTC AML 21

Who is this for? Older adults (60+) with newly diagnosed AML.

What did this analysis find?

Using data from over 600 patients, researchers found that how patients felt before starting treatment — their fatigue level, physical function, and overall wellbeing, measured by questionnaire — independently predicted how long they lived. Even after accounting for age, blood counts, and genetic risk:

• Higher fatigue = higher mortality risk
• Better physical and role functioning = better survival (each 10-point improvement was linked to a ~4–7% reduction in mortality risk)
• These effects held even after adjusting for frailty scores that doctors typically use

What does this mean for patients? Patients’ subjective wellbeing before treatment matters beyond just doctor’s test results. This study supports the idea that how patients report feeling should be formally captured before treatment starts — not as a replacement for medical tests, but as additional important information.

Patient perspective: This is a quiet but important study. It validates something patients have long known: how patients feel going into treatment matters. It also sends a message to medical teams: simple questionnaires about quality of life aren’t just “nice to have” — they carry real prognostic information. Patients should feel empowered to report their symptoms honestly, and should not minimise fatigue or limitations when speaking with their medical team.

RevSTAR-123 – First Glimpse of a New CAR-T Approach for Relapsed AML

Who is this for? Adults with AML that has come back or stopped responding to prior treatments, where standard options are exhausted. The leukemia cells must carry a marker called CD123 (present in most AML cases).

What is this treatment?

This is an entirely new type of therapy: “off-the-shelf” CAR-T cells combined with a switchable adapter molecule. Here’s the plain-language version:

• CAR-T cells are immune cells that have been re-engineered to seek and destroy cancer cells
• Usually, CAR-T cells are made from the patient’s own blood, which takes weeks. These are pre-made from a healthy donor (“off-the-shelf”), meaning they can be given more quickly
• A special “adapter” molecule (R-TM123) is given at the same time to direct the CAR-T cells precisely at leukemia cells carrying CD123
• Importantly, the adapter can be switched on or off — giving doctors a safety switch if side effects occur

What did they find (very early data — 17 patients)?

• Only 1 serious dose-related side effect occurred
• No treatment-related deaths, no nerve damage, no graft-versus-host disease (a major risk with donor-based therapies)
• Immune side effects (cytokine release syndrome) occurred in 12 patients but were manageable
• 2 patients achieved full remission, 1 had a partial bone marrow response, 3 had their minimal residual disease disappear — at the highest doses tested

What does this mean for patients? This is very early, Phase 1 data with only 17 patients — so firm conclusions cannot yet be drawn. But the safety signal is encouraging, and seeing any responses (including full remissions) in heavily pre-treated patients is meaningful.

Patient perspective: For patients who have exhausted standard options, this kind of innovation matters enormously. The “switchable” and “off-the-shelf” features are important: switchable means doctors can reduce toxicity if needed, and off-the-shelf means faster access than personalised CAR-T therapies. This is one to watch as it progresses through clinical trials.

Summary: What Is New and What Does It Mean?

What’s new	Why it matters
Ziftomenib (menin inhibitor) added to standard chemo shows 88–96% remission in NPM1/KMT2A AML	Potentially practice-changing for a large subgroup
Quizartinib appears better than midostaurin in real-world FLT3 AML data	Supports reconsidering the default FLT3 drug choice
AK117 (CD47 inhibitor) adds survival benefit in unfit, older patients	New immune approach for patients with few options
ATRA+ATO is also better for quality of life, not just effectiveness, in APL	Chemotherapy-free treatment confirmed as the better all-round choice
CPX-351 beats standard bridging therapy before stem cell transplant in MDS/AML	Better disease control before transplant without sacrificing transplant access
Off-the-shelf switchable CAR-T is safe and shows early signals in relapsed/refractory AML	Genuinely novel approach for a very hard-to-treat group
Pre-treatment quality of life predicts survival in older AML patients	Validates self-reported wellbeing as a meaningful clinical tool

The Big Picture

AML treatment is becoming more personalized. More and more, your specific gene mutations — NPM1, FLT3, KMT2A, IDH1/2 — determine which treatment is most likely to help you. At the same time, the field is moving toward treatments that are not just more effective, but also better tolerated and less disruptive to life.

There is also growing recognition that what patients experience matters — fatigue, quality of life, and the ability to stay active during treatment are being measured and taken seriously in clinical research.

Sources: EHA 2026 abstracts S126, S130, S131, S132, S133, S170, S327, LBA5005, LBA5007. This  is for information purposes only and does not constitute medical advice.

https://patientstoday.org/congresses/eha-2026/onDemand

Otsuka Pharmaceuticals Europe Ltd provided financial support through sponsorship for this EHA congress report. Otsuka has had no influence or involvement in the creation of the materials.