title: Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome.
abstract: 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.
  
  
  
  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.
  
  
  
  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.
  
  
  
  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).
  
  
  
  Appropriate use of ESAs can significantly delay the onset of a regular transfusion need in patients with lower-risk MDS.
authors.affiliation: Department of Medicine, Section of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden.
authors.forename: H K G
authors.initials: HK
authors.lastname: Garelius
authors.affiliation: Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK.
authors.forename: W T
authors.initials: WT
authors.lastname: Johnston
authors.affiliation: Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK.
authors.forename: A G
authors.initials: AG
authors.lastname: Smith
authors.affiliation: Clinique Universitaire d'hématologie, CHU de Grenoble, Université Grenoble, Grenoble, France.
authors.forename: S
authors.initials: S
authors.lastname: Park
authors.affiliation: Department of Hematology, Radboud university medical center, Nijmegen, the Netherlands.
authors.forename: L
authors.initials: L
authors.lastname: de Swart
authors.affiliation: Service d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7, Paris, France.
authors.forename: P
authors.initials: P
authors.lastname: Fenaux
authors.affiliation: Department of Medicine, Division of Hematology, University of Patras Medical School, Patras, Greece.
authors.forename: A
authors.initials: A
authors.lastname: Symeonidis
authors.affiliation: Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
authors.forename: G
authors.initials: G
authors.lastname: Sanz
authors.affiliation: Department of Clinical Hematology, Institute of Hematology & Blood Transfusion, Praha, Czech Republic.
authors.forename: J
authors.initials: J
authors.lastname: Čermák
authors.affiliation: Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria.
authors.forename: R
authors.initials: R
authors.lastname: Stauder
authors.affiliation: Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.
authors.forename: L
authors.initials: L
authors.lastname: Malcovati
authors.affiliation: Department of Medicine A, Tel Aviv Sourasky (Ichilov) Medical Center, Tel Aviv, Israel.
authors.forename: M
authors.initials: M
authors.lastname: Mittelman
authors.affiliation: Department of Hematology, VU Institute of Cancer and Immunology, VU University Medical Center, Amsterdam, the Netherlands.
authors.forename: A A
authors.initials: AA
authors.lastname: van de Loosdrecht
authors.affiliation: Department of Hematology, Radboud university medical center, Nijmegen, the Netherlands.
authors.forename: C J
authors.initials: CJ
authors.lastname: van Marrewijk
authors.affiliation: St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, UK.
authors.forename: D
authors.initials: D
authors.lastname: Bowen
authors.affiliation: Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK.
authors.forename: S
authors.initials: S
authors.lastname: Crouch
authors.affiliation: Department of Tumor Immunology, Nijmegen Center for Molecular Life Sciences, Radboud university medical center, Nijmegen, the Netherlands.
authors.forename: T J M
authors.initials: TJ
authors.lastname: de Witte
authors.affiliation: Department of Medicine, Division of Hematology, Karolinska Institutet, Stockholm, Sweden.
authors.forename: E
authors.initials: E
authors.lastname: Hellström-Lindberg
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001803:Blood Transfusion; D018450:Disease Progression; D005260:Female; D005500:Follow-Up Studies; D006397:Hematinics / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000628:therapy*; D011446:Prospective Studies; D012307:Risk Factors; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006397:Hematinics
keywords: MDS; Myelodysplasia; anaemia; haematology; haemoglobin