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Ferrata Storti Foundation
Haematologica 2020 Volume 105(11):2516-2523
Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome; the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective
Theo de Witte,1* Luca Malcovati,2* Pierre Fenaux,3 David Bowen,4 Argiris Symeonidis,5 Moshe Mittelman,6 Reinhard Stauder,7 Guillermo Sanz,8 Jaroslav Čermák,9 Saskia Langemeijer,10 Eva Hellström-Lindberg,11 Ulrich Germing,12 Mette Skov Holm,13 Krzysztof Mądry,14 Aurelia Tatic,15 António Medina Almeida,16 Aleksandar Savic,17 Inga Mandac Rogulj,18 Raphael Itzykson,3 Marlijn Hoeks,10 Hege Gravdahl Garelius,19 Dominic Culligan,20 Ioannis Kotsianidis,21 Lionel Ades,3 Arjan A. van de Loosdrecht,22 Corine van Marrewijk,10 Ge Yu,23 Simon Crouch,23 Alex Smith23; on behalf of the EUMDS Registry Participants
1Department of Tumor Immunology - Nijmegen Center for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, the Netherlands; 2Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy; 3Service d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7, Paris, France; 4St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, UK; 5Department of Medicine, Division of Hematology, University of Patras Medical School, Patras, Greece; 6Department of Medicine A, Tel Aviv Sourasky (Ichilov) Medical Center and Sackler Medical Faculty, Tel Aviv University, Tel Aviv, Israel; 7Department of Internal Medicine V (Haematology and Oncology), Innsbruck Medical University, Innsbruck, Austria; 8Department of Haematology, Hospital Universitario y Politécnico La Fe, Valencia, and CIBERONC, Madrid, Spain; 9Department of Clinical Hematology, Inst. of Hematology and Blood Transfusion, Praha, Czech Republic; 10Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands; 11Department of Medicine, Division Hematology, Karolinska Institutet, Stockholm, Sweden; 12Department of Haematology, Oncology and Clinical Immunology, Universitätsklinik Düsseldorf, Düsseldorf, Germany; 13Department of Haematology, Aarhus University Hospital, Aarhus, Denmark; 14Department of Haematology, Oncology and Internal Medicine, Warszawa Medical University, Warszawa, Poland; 15Center of Hematology and Bone Marrow Transplantation, Fundeni Clinical Institute, Bucharest, Romania; 16Department of Clinical Hematology, Hospital da Luz, Lisbon, Portugal; 17Clinic of Hematology - Clinical Center of Vojvodina, Faculty of Medicine, University of Novi Sad, Novi Sad, Serbia; 18Department of Internal Medicine, Division of Hematology, Merkur University Hospital, Zagreb, Croatia; 19Department of Specialist Medicine, Sahlgrenska University Hospital, Göteborg, Sweden; 20Department of Haematology, Aberdeen Royal Infirmary, Aberdeen, UK; 21Department of Hematology, Democritus University of Thrace Medical School, University Hospital of Alexandroupolis, Alexandroupolis, Greece; 22Department of Hematology – Cancer Center Amsterdam, Amsterdam UMC, Location VU University Medical Center, Amsterdam, the Netherlands and 23Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK
*TdW and LM both contributed equally as co-first authors.
Introduction
Myelodysplastic syndromes (MDS) are chronic bone marrow (BM) disorders char- acterized by peripheral blood cytopenias, predominantly in older persons with an average age at diagnosis of 75 years.1,2 The natural history of MDS is heterogeneous, ranging from indolent conditions to forms similar to acute myeloid leukemia (AML). In 1997, an International Prognostic Scoring System (IPSS) was established based on the percentage of BM blasts, number of cytopenias and cytogenetic characteristics.3 In 2012, this prognostic scoring system was refined (IPSS-R) to include better catego- rization of cytopenias, blast cell percentage and an improved risk stratification of the cytogenetic risk groups.4 Generally, MDS are divided into two prognostic groups: lower-risk MDS (LR-MDS) with patients from the (very) low risk or intermediate risk groups, and higher-risk MDS (HR-MDS) with patients from the (very) poor risk groups, as defined within the IPSS-R.2
The majority of patients with MDS (75%) have LR-MDS, which was the focus of the European LeukemiaNet MDS (EUMDS) Registry until 2017. The EUMDS Registry is a pragmatic, observational study, which has collected prospectively longi-
Correspondence:
THEO DE WITTE,
theo.dewitte@radboudumc.nl
Received: July 15, 2020.
Accepted: August 17, 2020. Pre-published: September 21, 2020.
doi:10.3324/haematol.2020.266817 ©2020 Ferrata Storti Foundation
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