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J Intern Med. 2017 Mar;281(3):284-299. doi: 10.1111/joim.12579. Epub 2016 Dec 7.

Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome.

Author information

1
Department of Medicine, Section of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden.
2
Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK.
3
Clinique Universitaire d'hématologie, CHU de Grenoble, Université Grenoble, Grenoble, France.
4
Department of Hematology, Radboud university medical center, Nijmegen, the Netherlands.
5
Service d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7, Paris, France.
6
Department of Medicine, Division of Hematology, University of Patras Medical School, Patras, Greece.
7
Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
8
Department of Clinical Hematology, Institute of Hematology & Blood Transfusion, Praha, Czech Republic.
9
Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria.
10
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.
11
Department of Medicine A, Tel Aviv Sourasky (Ichilov) Medical Center, Tel Aviv, Israel.
12
Department of Hematology, VU Institute of Cancer and Immunology, VU University Medical Center, Amsterdam, the Netherlands.
13
St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, UK.
14
Department of Tumor Immunology, Nijmegen Center for Molecular Life Sciences, Radboud university medical center, Nijmegen, the Netherlands.
15
Department of Medicine, Division of Hematology, Karolinska Institutet, Stockholm, Sweden.

Abstract

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.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00600860.

KEYWORDS:

MDS; Myelodysplasia; anaemia; haematology; haemoglobin

PMID:
27926979
PMCID:
PMC5596334
DOI:
10.1111/joim.12579
[Indexed for MEDLINE]
Free PMC Article

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