Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.
Delphi survey
clinical trial
core outcome set (COS)
myelodysplastic syndromes (MDS)
outcome study
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
22
12
2019
accepted:
18
03
2020
pubmed:
16
5
2020
medline:
23
3
2021
entrez:
16
5
2020
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 32410281
doi: 10.1111/bjh.16654
pmc: PMC8221029
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Systematic Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
405-417Subventions
Organisme : Verein Senioren-Krebshilfe
Organisme : the European Commission
Organisme : European Union's Horizon 2020 research and innovation programme
ID : 634789
Organisme : Austrian Science Fund within ERA NetS
ID : I 1576
Organisme : Novartis Pharmacy B.V. Oncology Europe
Organisme : Amgen Limited
Organisme : Celgene International
Informations de copyright
© 2020 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
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