BACKGROUND: The EUMDS registry is an unique prospective, longitudinal observational registry enrolling newly diagnosed patients with lower-risk myelodysplastic syndrome (MDS) from 17 European countries from both university hospitals and smaller regional hospitals. OBJECTIVE: The aim of this study was to describe the usage and clinical impact of erythropoiesis-stimulating agents (ESAs) in 1696 patients enrolled between 2008 and 2014. METHODS: The effects of ESAs on outcomes were assessed using proportional hazards models weighting observations by propensity to receive ESA treatment within a subset of anaemic patients with or without a regular transfusion need. RESULTS: ESA treatment (median duration of 27.5 months, range 0-77 months) was administered to 773 patients (45.6%). Outcomes were assessed in 897 patients (484 ESA treated and 413 untreated). ESA treatment was associated with a nonsignificant survival benefit (HR 0.82, 95% CI: 0.65-1.04, P = 0.09); this benefit was larger amongst patients without prior transfusions (P = 0.07). Amongst 539 patients for whom response to ESA treatment could be defined, median time to first post-ESA treatment transfusion was 6.1 months (IQR: 4.3-15.9 months) in those transfused before ESA treatment compared to 23.3 months (IQR: 7.0-47.8 months) in patients without prior transfusions (HR 2.4, 95% CI: 1.7-3.3, P < 0.0001). Responding patients had a better prognosis in terms of a lower risk of death (HR 0.65, 95% CI: 0.45-0.893, P = 0.018), whereas there was no significant effect on the risk of progression to acute myeloid leukaemia (HR 0.71, 95% CI: 0.39-1.29, P = 0.27). CONCLUSION: Appropriate use of ESAs can significantly delay the onset of a regular transfusion need in patients with lower-risk MDS.

Clinique Universitaire d'hématologie CHU de Grenoble Université Grenoble Grenoble France

Department of Clinical Hematology Institute of Hematology and Blood Transfusion Praha Czech Republic

Department of Hematology Hospital Universitario y Politécnico La Fe Valencia Spain

Department of Hematology Oncology Fondazione IRCCS Policlinico San Matteo University of Pavia Pavia Italy

Department of Hematology Radboud university medical center Nijmegen the Netherlands

Department of Hematology VU Institute of Cancer and Immunology VU University Medical Center Amsterdam the Netherlands

Department of Internal Medicine 5 Innsbruck Medical University Innsbruck Austria

Department of Medicine A Tel Aviv Sourasky Medical Center Tel Aviv Israel

Department of Medicine Division of Hematology Karolinska Institutet Stockholm Sweden

Department of Medicine Division of Hematology University of Patras Medical School Patras Greece

Department of Medicine Section of Hematology and Coagulation Sahlgrenska University Hospital Gothenburg Sweden

Department of Tumor Immunology Nijmegen Center for Molecular Life Sciences Radboud university medical center Nijmegen the Netherlands

Epidemiology and Cancer Statistics Group Department of Health Sciences University of York York UK

Service d'Hématologie Hôpital Saint Louis Assistance Publique des Hôpitaux de Paris and Université Paris 7 Paris France

St James's Institute of Oncology Leeds Teaching Hospitals Leeds UK

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