AML19

AML19 was a trial for younger adults with acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS), open to patients aged 16 to 60, though older patients could also enter.

Watch a video of the AML19 Results on YouTube

*This short film shares results from the Cancer Research UK AML trials programme, including AML18 and AML19.

AML 19 replaced the AML17 trial and, like previous AML trials supported by the NCRI AML working group, took the form of a platform trial, allowing questions to be asked at different stages of the patient’s treatment pathway and building on the results of earlier trials.

Sponsored by Cardiff University, the trial recruited 2,083 patients across 104 hospitals in the UK, Denmark and New Zealand between 2014 and 2021. AML19 tested “risk-adapted” therapy, where different treatments were available depending on the features of each patient’s disease.

With enormous thanks to all the patients and families who took part in this important research.

Results

The trial asked these key questions

Are two doses of Mylotarg better than one alongside chemotherapy?

No — one dose worked just as well and may cause fewer side effects.

Is the stronger FLAG-Ida chemotherapy better than standard DA chemotherapy?

For many patients, yes. FLAG-Ida with Mylotarg lowered the chance of the leukaemia coming back (relapse) and meant fewer patients needed a stem cell transplant. It worked especially well for patients with certain genetic changes (called NPM1 or FLT3). Patients with a type called core binding factor AML did best on the standard DA treatment.

Is the newer drug CPX-351 better than FLAG-Ida for high-risk AML?

Not for everyone. Overall it did not improve survival compared with FLAG-Ida, but it did lower the chance of relapse and appeared to help some patients with specific genetic changes.

Does regular blood monitoring help?

Not for everyone, but for patients with NPM1 or FLT3 changes, regular monitoring (and adjusting treatment based on it) helped them live longer and stay well.

Is adding midostaurin safe for FLT3-mutated AML?

Yes. In a small pilot within the trial, adding midostaurin to chemotherapy and Mylotarg was safe and well tolerated, and helped clear the leukaemia more deeply on sensitive tests. Early survival results were promising. This is now being tested more fully in a larger trial called OPTIMISE-FLT3.

What this means for future patients

The most important thing AML19 has done is help doctors understand which treatment works best for each type of AML. Rather than one treatment for everyone, doctors can now match the right treatment to each patient’s leukaemia, reducing the chance of the leukaemia coming back and, for some patients, avoiding treatments (like extra chemotherapy or a transplant) that they don’t need.

Publications

The results of AML19 have been published in the following papers, and presented at the EHA and ASH international haematology congresses over the past three years:

• Chemotherapy results (FLAG-Ida vs DA and Mylotarg dosing): Russell NH et al., Journal of Clinical Oncology, 2024 — read here
• CPX-351 for high-risk AML: Othman J et al., Blood Advances, 2023 — read here
• Midostaurin for FLT3-mutated AML: Russell NH, Othman J et al., Blood Advances, 2025 — read here
• Molecular (MRD) monitoring: Othman J et al., Blood, 2024 — read here