Defining a core outcome set in acute myeloid leukemia

The HARMONY Alliance is a public-private European Network established in 2017, which currently includes 53 partners and 27 associated members from 22 countries. One of HARMONY’s goals is to use Big Data to improve understanding and treatment of hematological malignancies. In order to achieve this aim, HARMONY is structured into eight work packages of which work package 2 (WP2) is focused on defining outcomes that are relevant to each hematological malignancy. In accordance, a pilot study will be performed to define core outcome set (COS) in acute myeloid leukemia (AML), one out of seven hematological malignancies considered in HARMONY.
Within this study, key stakeholders that provide their expert feedback are selected based on their skills and experience relevant to the disease or project. By participating in a Delphi survey, they vote on which outcome should be included within the COS. The stakeholders include health service users, health service practitioners, researchers, drug developer and patient advocates. Participants of all stakeholder groups were in particular recruited from members of the HARMONY work packages 2 and 6, but also participants outside the HARMONY Alliance are welcome to take part of the Delphi survey within their stakeholder group.
A future use of COS can improve the comparability of clinical trials, improves the consistency of reporting, reduces selective reporting bias and ensures appropriate outcomes are measured that are valued by a range of stakeholders.


Lars Bullinger, WP2 Leader, HARMONY Partner Charité Berlin
Renate Schulze-Rath, WP2 Leader, HARMONY Partner Bayer
Healthcare Alliance for Resourceful Medicine Offensive against Neoplasms in Hematology


Journal: Blood
Volume: 140
Issue: (Supplement 1)
Pages: 5285 - 5287
Year: 2022
DOI: 10.1182/blood-2022-157549

Further Study Information

Current Stage: Completed
Date: April 2019 - June 2020
Funding source(s): Funded through the Innovative Medicines Initiative (IMI) 2 Joint Undertaking and listed under grant agreement No. 116026. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA)

Health Area

Disease Category: Blood disorders, Cancer

Disease Name: Leukemia (acute myeloid)

Target Population

Age Range: 18 - 100

Sex: Either

Nature of Intervention:

Stakeholders Involved

- Clinical experts
- Consumers (patients)
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Regulatory agency representatives
- Researchers

Study Type

- COS for clinical trials or clinical research
- COS for practice


- Consensus meeting
- Delphi process