# AAML1031

Source: https://onco.cc/trials/aaml1031/  
OnCo record `aaml1031` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

AAML1031 was the children's leukaemia trial that found adding bortezomib to standard chemotherapy did not help and caused more nerve damage, while a separate part of the trial suggested that adding the FLT3 blocker sorafenib does improve outcomes for children whose leukaemia carries a high load of the FLT3-ITD mutation.

## Summary

AAML1031 was the Children's Oncology Group phase 3 trial for de novo acute myeloid leukaemia in patients under 30. Patients without a high allelic ratio FLT3-ITD mutation were randomised to standard chemotherapy (four courses, or three followed by allogeneic transplant for high-risk disease) with or without bortezomib on days 1, 4 and 8 of each course. Patients with high allelic ratio FLT3-ITD received sorafenib in three sequential cohorts that defined the dose and then added it to induction and as single-agent maintenance.

Bortezomib did not improve remission, event-free or overall survival and increased peripheral neuropathy and intensive care admissions. In the FLT3-ITD cohorts, patients given sorafenib had better event-free survival than a comparator group of high allelic ratio patients treated with identical chemotherapy without sorafenib, and multivariable analysis accounting for transplant and co-occurring favourable mutations confirmed the benefit. The trial is why the standard-of-care text for childhood acute myeloid leukaemia adds sorafenib to chemotherapy for high allelic ratio FLT3-ITD disease.

## Fields

- Kind: Trial
- Status: mixed
- Last checked: 2026-09-22
- Also known as: COG AAML1031
- Tags: soc-trials
- Registry id: NCT01371981
- Phase: 3
- Setting: Newly diagnosed acute myeloid leukaemia in patients under 30: standard chemotherapy with or without bortezomib (randomised), and for high allelic ratio FLT3-ITD disease the addition of sorafenib to chemotherapy and as maintenance (non-randomised cohorts)
- Sponsor: National Cancer Institute (NCI)
- Enrolled: 1645
- Result: Bortezomib added to chemotherapy did not improve three-year event-free survival (44.8 percent against 47.0 percent) and added toxicity; sorafenib for high allelic ratio FLT3-ITD disease roughly halved the hazard of an event compared with matched patients treated without it.
- Outcomes: Event-free survival at 3 years, bortezomib randomisation: Chemotherapy + bortezomib 44.8% vs Chemotherapy alone 47%; Overall survival at 3 years, bortezomib randomisation: Chemotherapy + bortezomib 63.6% vs Chemotherapy alone 67.2%; Complete remission after induction, bortezomib randomisation: Chemotherapy + bortezomib 89% vs Chemotherapy alone 91%; Event-free survival from study entry, high allelic ratio FLT3-ITD (hazard for patients not given sorafenib against sorafenib cohorts 2 and 3): Chemotherapy without sorafenib (comparator) vs Chemotherapy + sorafenib (cohorts 2 and 3), HR 2.37; Relapse risk from complete remission, high allelic ratio FLT3-ITD (hazard for patients not given sorafenib): Chemotherapy without sorafenib (comparator) vs Chemotherapy + sorafenib (cohorts 2 and 3), HR 3.03

## Sources

- ClinicalTrials.gov NCT01371981: https://clinicaltrials.gov/study/NCT01371981

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute myeloid leukaemia in children](https://onco.cc/cancers/aml-paediatric/), [FLT3-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-flt3/), [Leukaemia (all types)](https://onco.cc/cancers/leukaemia/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- drugs: [Bortezomib](https://onco.cc/drugs/bortezomib/), [Sorafenib](https://onco.cc/drugs/sorafenib/)
- institutions: [Children's Oncology Group (COG)](https://onco.cc/institutions/childrens-oncology-group/)
- key papers: [AAML1031: bortezomib with standard chemotherapy for children with acute myeloid leukaemia does not improve outcomes](https://onco.cc/key-papers/paper-aaml1031-bortezomib-aplenc-haematologica-2020/), [AAML1031: sorafenib combined with chemotherapy for children with high allelic ratio FLT3-ITD acute myeloid leukaemia](https://onco.cc/key-papers/paper-aaml1031-sorafenib-flt3-pollard-jco-2022/)

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