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

H. K.G. Garelius, W. T. Johnston, A. G. Smith, S. Park, L. de Swart, P. Fenaux, A. Symeonidis, G. Sanz, J. Čermák, R. Stauder, L. Malcovati, M. Mittelman, A. A. van de Loosdrecht, C. J. van Marrewijk, D. Bowen, S. Crouch, T. J.M. de Witte, E. Hellström-Lindberg

Research output: Contribution to journalArticleAcademicpeer-review

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.

Original languageEnglish
Pages (from-to)284-299
Number of pages16
JournalJournal of Internal Medicine
Volume281
Issue number3
DOIs
Publication statusPublished - 1 Mar 2017

Cite this

Garelius, H. K.G. ; Johnston, W. T. ; Smith, A. G. ; Park, S. ; de Swart, L. ; Fenaux, P. ; Symeonidis, A. ; Sanz, G. ; Čermák, J. ; Stauder, R. ; Malcovati, L. ; Mittelman, M. ; van de Loosdrecht, A. A. ; van Marrewijk, C. J. ; Bowen, D. ; Crouch, S. ; de Witte, T. J.M. ; Hellström-Lindberg, E. / Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome. In: Journal of Internal Medicine. 2017 ; Vol. 281, No. 3. pp. 284-299.
@article{f529729cd2e943c4aefb81026ba53911,
title = "Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome",
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.",
keywords = "anaemia, haematology, haemoglobin, MDS, Myelodysplasia",
author = "Garelius, {H. K.G.} and Johnston, {W. T.} and Smith, {A. G.} and S. Park and {de Swart}, L. and P. Fenaux and A. Symeonidis and G. Sanz and J. Čerm{\'a}k and R. Stauder and L. Malcovati and M. Mittelman and {van de Loosdrecht}, {A. A.} and {van Marrewijk}, {C. J.} and D. Bowen and S. Crouch and {de Witte}, {T. J.M.} and E. Hellstr{\"o}m-Lindberg",
year = "2017",
month = "3",
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doi = "10.1111/joim.12579",
language = "English",
volume = "281",
pages = "284--299",
journal = "Journal of Internal Medicine",
issn = "0954-6820",
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}

Garelius, HKG, Johnston, WT, Smith, AG, Park, S, de Swart, L, Fenaux, P, Symeonidis, A, Sanz, G, Čermák, J, Stauder, R, Malcovati, L, Mittelman, M, van de Loosdrecht, AA, van Marrewijk, CJ, Bowen, D, Crouch, S, de Witte, TJM & Hellström-Lindberg, E 2017, 'Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome' Journal of Internal Medicine, vol. 281, no. 3, pp. 284-299. https://doi.org/10.1111/joim.12579

Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome. / Garelius, H. K.G.; Johnston, W. T.; Smith, A. G.; Park, S.; de Swart, L.; Fenaux, P.; Symeonidis, A.; Sanz, G.; Čermák, J.; Stauder, R.; Malcovati, L.; Mittelman, M.; van de Loosdrecht, A. A.; van Marrewijk, C. J.; Bowen, D.; Crouch, S.; de Witte, T. J.M.; Hellström-Lindberg, E.

In: Journal of Internal Medicine, Vol. 281, No. 3, 01.03.2017, p. 284-299.

Research output: Contribution to journalArticleAcademicpeer-review

TY - JOUR

T1 - Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome

AU - Garelius, H. K.G.

AU - Johnston, W. T.

AU - Smith, A. G.

AU - Park, S.

AU - de Swart, L.

AU - Fenaux, P.

AU - Symeonidis, A.

AU - Sanz, G.

AU - Čermák, J.

AU - Stauder, R.

AU - Malcovati, L.

AU - Mittelman, M.

AU - van de Loosdrecht, A. A.

AU - van Marrewijk, C. J.

AU - Bowen, D.

AU - Crouch, S.

AU - de Witte, T. J.M.

AU - Hellström-Lindberg, E.

PY - 2017/3/1

Y1 - 2017/3/1

N2 - 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.

AB - 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.

KW - anaemia

KW - haematology

KW - haemoglobin

KW - MDS

KW - Myelodysplasia

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U2 - 10.1111/joim.12579

DO - 10.1111/joim.12579

M3 - Article

VL - 281

SP - 284

EP - 299

JO - Journal of Internal Medicine

JF - Journal of Internal Medicine

SN - 0954-6820

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