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

Fecha de publicación: 01/11/2020 Fecha Ahead of Print: 01/05/2020

Autores de IIS La Fe

Guillermo Sanz Santillana

Autor

Participantes ajenos a IIS La Fe

Rochau U
Stojkov I
Conrads-Frank A
Borba HH
Koinig KA
Arvandi M
van Marrewijk C
Garelius H
Germing U
Symeonidis A
Fenaux P
de Witte T
Efficace F
Siebert U
Stauder R

Grupos

Hematología y Hemoterapia

Investigador Principal

Javier De La Rubia Comos

Abstract

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.

Datos de la publicación

ISSN/ISSNe:: 0007-1048, 1365-2141
Tipo:: Article
Páginas:: 405-417
DOI:: 10.1111/bjh.16654
Factor de Impacto:: 1,907 SCImago ℠
Cuartil:: Q1 SCImago ℠

Documentos

No hay documentos

Métricas

Filiaciones

Rochau U:: 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
Stojkov I:: 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
Conrads-Frank A:: 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
Borba HH:: Department of Pharmacy, Pharmaceutical Sciences Postgraduate Research Program, Federal University of Paraná, Curitiba, Brazil
Koinig KA:: Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria
Arvandi M:: 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
van Marrewijk C:: Department of Hematology, Radboud university medical center, Nijmegen, The Netherlands
Garelius H:: Department of Medicine, Section of Hematology and Coagulation, Sahlgrenska University Hospital, Göteborg, Sweden
Germing U:: Department of Hematology, Oncology and Clinical Immunology, Universitätsklinik Düsseldorf, Düsseldorf, Germany
Symeonidis A:: Department of Internal Medicine, Division of Hematology, University of Patras Medical School, Patras, Greece
Sanz GF:: Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain

Centro de Investigación Biomédica en Red de Cáncer, CIBERONC, Instituto de Salud Carlos III, Madrid, Spain
Fenaux P:: Service d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7, Paris, France
Efficace F:: Health Outcomes Research Unit, Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA), Rome, Italy
Siebert U:: 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

Center for Health Decision Science, Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston, MA, USA

Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA
Stauder R:: Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria