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Survival and quality of life in patients with lower risk myelodysplastic syndromes exposed to erythropoiesis-stimulating agents: an observational cohort study
HKG. Garelius, T. Bagguley, A. Taylor, P. Fenaux, D. Bowen, A. Symeonidis, M. Mittelmann, R. Stauder, J. Čermák, G. Sanz, S. Langemeijer, L. Malcovati, U. Germing, L. Sanhes, M. d'Aveni, D. Culligan, I. Kotsianidis, KA. Koinig, C. van Marrewijk,...
Language English Country England, Great Britain
Document type Journal Article, Observational Study
- MeSH
- Hematinics * therapeutic use MeSH
- Cohort Studies MeSH
- Quality of Life * MeSH
- Middle Aged MeSH
- Humans MeSH
- Longitudinal Studies MeSH
- Myelodysplastic Syndromes * drug therapy mortality therapy complications MeSH
- Registries MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Erythrocyte Transfusion adverse effects MeSH
- Check Tag
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Aged, 80 and over MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Observational Study MeSH
BACKGROUND: In our previous study on erythropoiesis-stimulating agent (ESA) treatment in lower risk myelodysplastic syndromes from the European MDS (EUMDS) Registry, we showed that patients treated with ESAs had longer survival compared with patients who receive red blood cell transfusion (RBCT). In this study, with a longer follow up time and more patients included, we aimed to assess long-term effects on survival and health-related quality of life (HRQoL) of exposure to ESAs with or without RBCT in patients with lower risk myelodysplastic syndromes. METHODS: The EUMDS Registry is a non-interventional, longitudinal, real-world registry prospectively enrolling newly diagnosed patients older than 18 years with lower risk (International Prognostic Scoring System low or intermediate-1) myelodysplastic syndromes from 16 European countries and Israel. The analysis was restricted to patients with haemoglobin concentrations less than 100 g/L enrolled between Jan 1, 2008, and July 1, 2019, with last censoring of data on Dec 31, 2021. Patient management was recorded every 6 months, including treatment, transfusions, and HRQoL. ESA treatment followed local guidelines. The patients were separated into four groups at each study visit: no ESA or RBCT, ESA only, ESA plus RBCT, and RBCT only. The data were analysed longitudinally over time according to ESA and RBCT status during each 6-month interval, using propensity score matching. The main outcomes were median overall survival and leukaemia-free survival, and HRQoL. This study is registered with ClinicalTrials.gov, NCT00600860, as is ongoing. FINDINGS: 2448 patients (the ESA-unexposed group [n=1265] and ESA-exposed group [n=1183]) were diagnosed before July 1, 2019; 1520 (62·1%) were male and 928 (37·9%) were female. Median follow-up time was 3·9 years (IQR 1·6-6·5). After applying eligibility criteria and propensity matching, there were 426 patients in the ESA-unexposed group and 744 patients in the ESA-exposed group. Median overall survival in the ESA exposed group was 44·9 months (95% CI 40·2-50·5) compared with 34·8 months (28·6-39·2) in the ESA unexposed group; the absolute difference was 10·1 months (95% CI 2·2-18·0; hazard ratio [HR] 0·70 [95% CI 0·59-0·83]; p<0·0001). Patients without RBCT in the presence or absence of ESA exposure maintained significantly better HRQoL than those with RBCT, irrespective of ESA exposure (linear mixed effect model of EQ-5d-3L index score, RBCT coefficient -0·04 [95% CI -0·06 to 0·03], p<0·0001; linear mixed effect model of VAS, -4·57 [-6·02 to -3·13], p<0·0001). INTERPRETATION: ESA treatment in patients with lower risk myelodysplastic syndromes significantly improves overall survival when started before or early after the onset of regular transfusion therapy. Avoiding RBCT is associated with significantly better HRQoL. FUNDING: H2020 European Research Council, Novartis Pharmacy B V Oncology Europe, Amgen, BMS/Celgene International, Janssen Pharmaceutica, Takeda Pharmaceuticals International, and Gilead Sciences.
Aberdeen Royal Infirmary Aberdeen UK
Centre Hospitalier de Perpignan Perpignan France
Centre Hospitalier Régional Universitaire de Nancy Hôpitaux de Brabois Vandèuvre lès Nancy France
Consorcio Hospital General Universitario de Valencia Valencia Spain
Epidemiology and Cancer Statistics Group Department of Health Sciences University of York York UK
General University Hospital of Alexandroupolis Alexandroupoli Greece
Hôpital Saint Louis Paris France
Institute of Hematology and Blood Transfusion Prague Czech Republic
Karolinska Institute Solna Sweden
Leeds Teaching Hospitals NHS Trust Leeds UK
Medizinische Universität Innsbruck Innsbruck Austria
Radboud University Medical Center Nijmegen Netherlands
Sahlgrenska University Hospital Gothenburg Sweden
Tel Aviv Sourasky Medical Center Tel Aviv Yafo Israel
Universitätsklinikum Düsseldorf Düsseldorf Germany
University of Patras School of Medicine Patras Greece
University of Pavia and IRCCS Policlinico San Matteo Pavia Italy
References provided by Crossref.org
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