Development of a core outcome set for myelodysplastic syndromes – a Delphi study from the EUMDS Registry Group

Treatment options for myelodysplastic syndromes (MDS) vary widely,
depending on the natural disease course and patient-related factors. Comparison of treatment effectiveness is challenging as different endpoints have been included in clinical trials and outcome reporting. Our goal was to
develop the first MDS core outcome set (MDS-COS) defining a minimum
set of outcomes that should be reported in future clinical studies. We performed a comprehensive systematic literature review among MDS studies to extract patient- and/or clinically relevant outcomes. Clinical experts from the European LeukemiaNet MDS (EUMDS) identified 26 potential MDS core outcomes and participated in a three-round Delphi survey. After the first survey (56 experts), 15 outcomes met the inclusion criteria and one additional outcome was included. The second round (38 experts) resulted in six included outcomes. In the third round, a final check on plausibility and practicality of the six included outcomes and their definitions was performed.

The final MDS-COS includes: health-related quality of life, treatment-
related mortality, overall survival, performance status, safety, and
haematological improvement. This newly developed MDS-COS represents
the first minimum set of outcomes aiming to enhance comparability across
future MDS studies and facilitate a better understanding of treatment effectiveness.

Keywords: myelodysplastic syndromes (MDS), core outcome set (COS),
Delphi survey, outcome study, clinical trial.

Contributors

Rochau U1, Stojkov I1, Conrads-Frank A1, Borba HH2, Koinig KA3, Arvandi M1, van Marrewijk C4, Garelius H5, Germing U6, Symeonidis A7, Sanz GF8,9, Fenaux P10, de Witte T11, Efficace F12, Siebert U1,13,14, Stauder R3.


1
Department of Public Health, Health Services Research and Health Technology Assessment, Institute of Public Health, Medical Decision Making and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria.
2
Department of Pharmacy, Pharmaceutical Sciences Postgraduate Research Program, Federal University of Paraná, Curitiba, Brazil.
3
Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria.
4
Department of Hematology, Radboud university medical center, Nijmegen, the Netherlands.
5
Department of Medicine, Section of Hematology and Coagulation, Sahlgrenska University Hospital, Göteborg, Sweden.
6
Department of Hematology, Oncology and Clinical Immunology, Universitätsklinik Düsseldorf, Düsseldorf, Germany.
7
Department of Internal Medicine, Division of Hematology, University of Patras Medical School, Patras, Greece.
8
Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
9
Centro de Investigación Biomédica en Red de Cáncer, CIBERONC, Instituto de Salud Carlos III, Madrid, Spain.
10
Service d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7, Paris, France.
11
Department of Tumor Immunology - Nijmegen Center for Molecular Life Sciences, Radboud university medical center, Nijmegen, the Netherlands.
12
Health Outcomes Research Unit, Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA), Rome, Italy.
13
Center for Health Decision Science, Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston, MA, USA.
14
Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Publication

Journal: British Journal of Haematology
Volume:
Issue:
Pages: -
Year: 2020
DOI: 10.1111/bjh.16654

Further Study Information

Current Stage: Completed
Date:
Funding source(s):


Health Area

Disease Category: Cancer

Disease Name: Myelodysplastic syndromes (MDS)

Target Population

Age Range: Unknown

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Clinical experts

Study Type

- COS for clinical trials or clinical research

Method(s)

- Delphi process
- Systematic review

We performed
a comprehensive systematic literature review among MDS studies
to extract patient- and/or clinically relevant outcomes. Clinical experts from
the European LeukemiaNet MDS (EUMDS) identified 26 potential MDS
core outcomes and participated in a three-round Delphi survey. After the
first survey (56 experts), 15 outcomes met the inclusion criteria and one
additional outcome was included. The second round (38 experts) resulted
in six included outcomes. In the third round, a final check on plausibility
and practicality of the six included outcomes and their definitions was performed.

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