Abstract
BackgroundThe 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.ObjectiveThe 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.MethodsThe 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).ConclusionAppropriate use of ESAs can significantly delay the onset of a regular transfusion need in patients with lower‐risk MDS.
Highlights
Lower-risk myelodysplastic syndrome (MDS) is a malignant condition that is treated at both Medicine and Haematology Departments in small and large hospitals
erythropoiesis-stimulating agents (ESAs) 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)
Summary
Lower-risk myelodysplastic syndrome (MDS) is a malignant condition that is treated at both Medicine and Haematology Departments in small and large hospitals. The anaemia of patients with lower-risk MDS has been associated with reduced quality of life in a number of small prospective Phase II trials [1,2,3,4] and with reduced survival in retrospective registry reports [5]. The efficacy of erythropoietin (EPO) can be enhanced by the addition of granulocyte colonystimulating factor (G-CSF), mainly in MDS with ring sideroblasts; the median duration of response to the combined treatment is around 30 months, with some patients responding for more than 10 years [1]. Patients with a low probability of response are characterized by a transfusion need exceeding 2 units per month combined with a serum EPO level of ≥500 U LÀ1 [3]. The EUMDS registry is an unique prospective, longitudinal observational registry enrolling newly diagnosed patients with lower-risk
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