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

H K G Garelius; William Thomas Johnston; Alexandra Gwen Smith; S Park; L de Swart; P Fenaux; A Symeonidis; G Sanz; J Cermak; R Stauder; L Malcovati; M. Mittelman; A A van de Loosdrecht; C J  van Marrewijk; David Bowen; Simon Crouch; T J M de Witte; E Hellstrom-Lindberg

doi:10.1111/joim.12579

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

H K G Garelius, William Thomas Johnston, Alexandra Gwen Smith, S Park, L de Swart, P Fenaux, A Symeonidis, G Sanz, J Cermak, R Stauder, L Malcovati, M. Mittelman, A A van de Loosdrecht, C J van Marrewijk, David Bowen, Simon Crouch, T J M de Witte, E Hellstrom-Lindberg

Health Sciences

Research output: Contribution to journal › Article › peer-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 language	English
Pages (from-to)	284-299
Number of pages	16
Journal	Journal of general internal medicine
Volume	281
Issue number	3
Early online date	7 Dec 2016
DOIs	https://doi.org/10.1111/joim.12579
Publication status	Published - 17 Feb 2017

Bibliographical note

Keywords

MDS
Myelodysplasia
anaemia
haematology
haemoglobin

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10.1111/joim.12579

Garelius_et_al-2016-Journal_of_Internal_Medicine
© 2016 The Authors
Final published version, 507 KBLicence: CC BY-NC-ND

Cite this

Garelius, H. K. G., Johnston, W. T., Smith, A. G., Park, S., de Swart, L., Fenaux, P., Symeonidis, A., Sanz, G., Cermak, 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., & Hellstrom-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 general internal medicine, 281(3), 284-299. https://doi.org/10.1111/joim.12579

@article{d767c59511a942d48ec28744680652c5,

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 = "MDS, Myelodysplasia, anaemia, haematology, haemoglobin",

author = "Garelius, {H K G} and Johnston, {William Thomas} and Smith, {Alexandra Gwen} and S Park and {de Swart}, L and P Fenaux and A Symeonidis and G Sanz and J Cermak and R Stauder and L Malcovati and M. Mittelman and {van de Loosdrecht}, {A A} and {van Marrewijk}, {C J} and David Bowen and Simon Crouch and {de Witte}, {T J M} and E Hellstrom-Lindberg",

note = "{\textcopyright} 2016 The Authors",

year = "2017",

month = feb,

day = "17",

doi = "10.1111/joim.12579",

language = "English",

volume = "281",

pages = "284--299",

journal = "Journal of general internal medicine",

issn = "0884-8734",

publisher = "Springer",

number = "3",

}

Garelius, HKG, Johnston, WT , Smith, AG, Park, S, de Swart, L, Fenaux, P, Symeonidis, A, Sanz, G, Cermak, J, Stauder, R, Malcovati, L, Mittelman, M, van de Loosdrecht, AA, van Marrewijk, CJ, Bowen, D, Crouch, S, de Witte, TJM & Hellstrom-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 general 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, William Thomas ; Smith, Alexandra Gwen et al.
In: Journal of general internal medicine, Vol. 281, No. 3, 17.02.2017, p. 284-299.

Research output: Contribution to journal › Article › peer-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, William Thomas

AU - Smith, Alexandra Gwen

AU - Park, S

AU - de Swart, L

AU - Fenaux, P

AU - Symeonidis, A

AU - Sanz, G

AU - Cermak, J

AU - Stauder, R

AU - Malcovati, L

AU - Mittelman, M.

AU - van de Loosdrecht, A A

AU - van Marrewijk, C J

AU - Bowen, David

AU - Crouch, Simon

AU - de Witte, T J M

AU - Hellstrom-Lindberg, E

PY - 2017/2/17

Y1 - 2017/2/17

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 - MDS

KW - Myelodysplasia

KW - anaemia

KW - haematology

KW - haemoglobin

UR - http://www.scopus.com/inward/record.url?scp=85008252451&partnerID=8YFLogxK

U2 - 10.1111/joim.12579

DO - 10.1111/joim.12579

M3 - Article

SN - 0884-8734

VL - 281

SP - 284

EP - 299

JO - Journal of general internal medicine

JF - Journal of general internal medicine

IS - 3

ER -

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

Abstract

Bibliographical note

Keywords

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Alex Smith

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

Abstract

Bibliographical note

Keywords

Access to Document

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Profiles

Alex Smith

Cite this