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AML: Delphi Survey: Towards a Core Outcome Set for Acute Myeloid Leukemia

AML, Delphi, HARMONY, Leukemia

The AML Delphi Survey is closed. Patients, clinicians, drug developers and regulators have participated. 

Make use of our explanatory documentation:


At present, several innovative compounds are being tested in clinical trials in order to improve the treatment of Acute Myeloid Leukemia (AML). However, these trials measure different outcomes, thus thwarting their comparability. In addition, the majority of clinical trials do not assess long-term side effects and their influence on the patients’ quality of life. A core outcome set (COS) may assist researchers in selecting outcomes for future trials, thereby promoting harmonization of AML studies and improving clinical management of the disease.




Project Summary

AML is the most common acute leukemia in adults. Despite recent scientific advances, AML cure rates remain poor, compared to those for other HMs. To improve the treatment of AML, several novel compounds are currently being tested in clinical trials. Unfortunately, these trials are insufficiently comparable because they measure different outcomes. In addition, the majority of trials do not assess long-term side effects and their influence on patients’ quality of life. Defining a core outcome set (COS) may offer a solution to these problems.

A COS is a minimum set of outcomes to be collected in future clinical trials which, ideally, have been agreed upon by all stakeholders. The COS is intended as an international standard to evaluate AML treatments in clinical trials and other research settings (e.g., registry and observational studies). Using a COS will improve the comparability of clinical trials, enhance the consistency of reporting, and reduce selective reporting bias. The COS can also be incorporated into clinical guidelines and be used to improve patient management. This project aims to identify a COS for AML in adult patients that will be accepted by all of HARMONY’s stakeholder groups.

The researchers will use the Delphi method to develop the COS. First, a preliminary list of about 70 outcomes will be created, which will be based on literature research and expert interviews. Next, a panel of stakeholders will be asked to rate the importance of each outcome on the basis of +their personal experiences. This will be done in at least three iterative rounds, using an online survey tool (COMET). After each round, the participants will receive an anonymized summary of the survey results per stakeholder group. This will allow them to revise their answers in the next round. It is expected that the group will converge towards a consensus COS after a number of iterations. The process will be stopped after pre-defined consensus criteria have been achieved.

Panel participants will be recruited from HARMONY Partners, including clinicians, patients, representatives from the pharmaceutical industry, and health authorities. The aim is to recruit equal participant numbers for all stakeholder groups wherever possible. Potential participants will be contacted by email. After completion of this pilot in AML, similar Delphi surveys are planned for the other hematological malignancies that are being studied by the HARMONY Alliance. Finally, a COS applicable to all seven hematological malignancies will be defined.

Download the Delphi AML Study Protocol document to inspect the full outcome list in Annex 1 >

Read our news article: 'HARMONY Launches a Delphi Survey to Develop a Core Outcome Set for Acute Myeloid Leukemia' >

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