Effects of different doses of erythropoietin in patients with myelodysplastic syndromes: A propensity score-matched analysis

Enrico Balleari, Rosa Angela Filiberti, Chiara Salvetti, Bernardino Allione, Emanuele Angelucci, Marco Bruzzone, Tullio Calzamiglia, Marina Cavaliere, Maurizio Cavalleri, Daniela Cilloni, Marino Clavio, Elena Crisà, Anna Da Col, Paolo Danise, Federica Pilo, Dario Ferrero, Carlo Finelli, Daniela Gioia, Roberto Massimo Lemoli, Elisa MasieraEmanuela Messa, Maurizio Miglino, Pellegrino Musto, Esther Natalie Oliva, Antonella Poloni, Flavia Salvi, Alessandro Sanna, Marco Scudeletti, Rodolfo Tassara, Valeria Santini

Research output: Contribution to journalArticle

Abstract

Background: Erythropoiesis-stimulating agents effectively improve the hemoglobin levels in a fraction of anemic patients with myelodysplastic syndromes (MDS). Higher doses (HD) of recombinant human erythropoietin (rhEPO) have been proposed to overcome suboptimal response rates observed in MDS patients treated with lower “standard doses” (SD) of rhEPO. However, a direct comparison between the different doses of rhEPO is lacking. Methods: A cohort of 104 MDS patients treated with HD was retrospectively compared to 208 patients treated with SD in a propensity score-matched analysis to evaluate hematological improvement-erythroid (HI-E) rate induced by the different doses of rhEPO. The impact of rhEPO doses on survival and progression to leukemia was also investigated. Results: Overall HI-E rate was 52.6%. No difference was observed between different rhEPO doses (P =.28) in matched cohorts; in a subgroup analysis, transfusion-dependent patients and patients with higher IPSS-R score obtained a higher HI-E rate with HD, although without significant impact on overall survival (OS). Achievement of HI-E resulted in superior OS. At univariate analysis, a higher HI-E rate was observed in transfusion-independent patients (P <.001), with a lower IPSS-R score (P <.001) and lower serum EPO levels (P =.027). Multivariate analysis confirmed that rhEPO doses were not significantly related to HI-E (P =.26). There was no significant difference in OS or progression to leukemia in patients treated with HD vs SD. Conclusion: SD are substantially equally effective to HD to improve anemia and influencing survival in MDS patients stratified according to similar propensity to be exposed to rhEPO treatment.

Original languageEnglish
JournalCancer Medicine
DOIs
Publication statusAccepted/In press - Jan 1 2019

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Propensity Score
Myelodysplastic Syndromes
Erythropoietin
Survival
Leukemia
Hematinics
Anemia
Hemoglobins
Multivariate Analysis

Keywords

  • anemia
  • erythropoietin
  • myelodysplastic syndromes

ASJC Scopus subject areas

  • Oncology
  • Radiology Nuclear Medicine and imaging
  • Cancer Research

Cite this

Effects of different doses of erythropoietin in patients with myelodysplastic syndromes : A propensity score-matched analysis. / Balleari, Enrico; Filiberti, Rosa Angela; Salvetti, Chiara; Allione, Bernardino; Angelucci, Emanuele; Bruzzone, Marco; Calzamiglia, Tullio; Cavaliere, Marina; Cavalleri, Maurizio; Cilloni, Daniela; Clavio, Marino; Crisà, Elena; Da Col, Anna; Danise, Paolo; Pilo, Federica; Ferrero, Dario; Finelli, Carlo; Gioia, Daniela; Lemoli, Roberto Massimo; Masiera, Elisa; Messa, Emanuela; Miglino, Maurizio; Musto, Pellegrino; Natalie Oliva, Esther; Poloni, Antonella; Salvi, Flavia; Sanna, Alessandro; Scudeletti, Marco; Tassara, Rodolfo; Santini, Valeria.

In: Cancer Medicine, 01.01.2019.

Research output: Contribution to journalArticle

Balleari, E, Filiberti, RA, Salvetti, C, Allione, B, Angelucci, E, Bruzzone, M, Calzamiglia, T, Cavaliere, M, Cavalleri, M, Cilloni, D, Clavio, M, Crisà, E, Da Col, A, Danise, P, Pilo, F, Ferrero, D, Finelli, C, Gioia, D, Lemoli, RM, Masiera, E, Messa, E, Miglino, M, Musto, P, Natalie Oliva, E, Poloni, A, Salvi, F, Sanna, A, Scudeletti, M, Tassara, R & Santini, V 2019, 'Effects of different doses of erythropoietin in patients with myelodysplastic syndromes: A propensity score-matched analysis', Cancer Medicine. https://doi.org/10.1002/cam4.2638
Balleari, Enrico ; Filiberti, Rosa Angela ; Salvetti, Chiara ; Allione, Bernardino ; Angelucci, Emanuele ; Bruzzone, Marco ; Calzamiglia, Tullio ; Cavaliere, Marina ; Cavalleri, Maurizio ; Cilloni, Daniela ; Clavio, Marino ; Crisà, Elena ; Da Col, Anna ; Danise, Paolo ; Pilo, Federica ; Ferrero, Dario ; Finelli, Carlo ; Gioia, Daniela ; Lemoli, Roberto Massimo ; Masiera, Elisa ; Messa, Emanuela ; Miglino, Maurizio ; Musto, Pellegrino ; Natalie Oliva, Esther ; Poloni, Antonella ; Salvi, Flavia ; Sanna, Alessandro ; Scudeletti, Marco ; Tassara, Rodolfo ; Santini, Valeria. / Effects of different doses of erythropoietin in patients with myelodysplastic syndromes : A propensity score-matched analysis. In: Cancer Medicine. 2019.
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abstract = "Background: Erythropoiesis-stimulating agents effectively improve the hemoglobin levels in a fraction of anemic patients with myelodysplastic syndromes (MDS). Higher doses (HD) of recombinant human erythropoietin (rhEPO) have been proposed to overcome suboptimal response rates observed in MDS patients treated with lower “standard doses” (SD) of rhEPO. However, a direct comparison between the different doses of rhEPO is lacking. Methods: A cohort of 104 MDS patients treated with HD was retrospectively compared to 208 patients treated with SD in a propensity score-matched analysis to evaluate hematological improvement-erythroid (HI-E) rate induced by the different doses of rhEPO. The impact of rhEPO doses on survival and progression to leukemia was also investigated. Results: Overall HI-E rate was 52.6{\%}. No difference was observed between different rhEPO doses (P =.28) in matched cohorts; in a subgroup analysis, transfusion-dependent patients and patients with higher IPSS-R score obtained a higher HI-E rate with HD, although without significant impact on overall survival (OS). Achievement of HI-E resulted in superior OS. At univariate analysis, a higher HI-E rate was observed in transfusion-independent patients (P <.001), with a lower IPSS-R score (P <.001) and lower serum EPO levels (P =.027). Multivariate analysis confirmed that rhEPO doses were not significantly related to HI-E (P =.26). There was no significant difference in OS or progression to leukemia in patients treated with HD vs SD. Conclusion: SD are substantially equally effective to HD to improve anemia and influencing survival in MDS patients stratified according to similar propensity to be exposed to rhEPO treatment.",
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T1 - Effects of different doses of erythropoietin in patients with myelodysplastic syndromes

T2 - A propensity score-matched analysis

AU - Balleari, Enrico

AU - Filiberti, Rosa Angela

AU - Salvetti, Chiara

AU - Allione, Bernardino

AU - Angelucci, Emanuele

AU - Bruzzone, Marco

AU - Calzamiglia, Tullio

AU - Cavaliere, Marina

AU - Cavalleri, Maurizio

AU - Cilloni, Daniela

AU - Clavio, Marino

AU - Crisà, Elena

AU - Da Col, Anna

AU - Danise, Paolo

AU - Pilo, Federica

AU - Ferrero, Dario

AU - Finelli, Carlo

AU - Gioia, Daniela

AU - Lemoli, Roberto Massimo

AU - Masiera, Elisa

AU - Messa, Emanuela

AU - Miglino, Maurizio

AU - Musto, Pellegrino

AU - Natalie Oliva, Esther

AU - Poloni, Antonella

AU - Salvi, Flavia

AU - Sanna, Alessandro

AU - Scudeletti, Marco

AU - Tassara, Rodolfo

AU - Santini, Valeria

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Background: Erythropoiesis-stimulating agents effectively improve the hemoglobin levels in a fraction of anemic patients with myelodysplastic syndromes (MDS). Higher doses (HD) of recombinant human erythropoietin (rhEPO) have been proposed to overcome suboptimal response rates observed in MDS patients treated with lower “standard doses” (SD) of rhEPO. However, a direct comparison between the different doses of rhEPO is lacking. Methods: A cohort of 104 MDS patients treated with HD was retrospectively compared to 208 patients treated with SD in a propensity score-matched analysis to evaluate hematological improvement-erythroid (HI-E) rate induced by the different doses of rhEPO. The impact of rhEPO doses on survival and progression to leukemia was also investigated. Results: Overall HI-E rate was 52.6%. No difference was observed between different rhEPO doses (P =.28) in matched cohorts; in a subgroup analysis, transfusion-dependent patients and patients with higher IPSS-R score obtained a higher HI-E rate with HD, although without significant impact on overall survival (OS). Achievement of HI-E resulted in superior OS. At univariate analysis, a higher HI-E rate was observed in transfusion-independent patients (P <.001), with a lower IPSS-R score (P <.001) and lower serum EPO levels (P =.027). Multivariate analysis confirmed that rhEPO doses were not significantly related to HI-E (P =.26). There was no significant difference in OS or progression to leukemia in patients treated with HD vs SD. Conclusion: SD are substantially equally effective to HD to improve anemia and influencing survival in MDS patients stratified according to similar propensity to be exposed to rhEPO treatment.

AB - Background: Erythropoiesis-stimulating agents effectively improve the hemoglobin levels in a fraction of anemic patients with myelodysplastic syndromes (MDS). Higher doses (HD) of recombinant human erythropoietin (rhEPO) have been proposed to overcome suboptimal response rates observed in MDS patients treated with lower “standard doses” (SD) of rhEPO. However, a direct comparison between the different doses of rhEPO is lacking. Methods: A cohort of 104 MDS patients treated with HD was retrospectively compared to 208 patients treated with SD in a propensity score-matched analysis to evaluate hematological improvement-erythroid (HI-E) rate induced by the different doses of rhEPO. The impact of rhEPO doses on survival and progression to leukemia was also investigated. Results: Overall HI-E rate was 52.6%. No difference was observed between different rhEPO doses (P =.28) in matched cohorts; in a subgroup analysis, transfusion-dependent patients and patients with higher IPSS-R score obtained a higher HI-E rate with HD, although without significant impact on overall survival (OS). Achievement of HI-E resulted in superior OS. At univariate analysis, a higher HI-E rate was observed in transfusion-independent patients (P <.001), with a lower IPSS-R score (P <.001) and lower serum EPO levels (P =.027). Multivariate analysis confirmed that rhEPO doses were not significantly related to HI-E (P =.26). There was no significant difference in OS or progression to leukemia in patients treated with HD vs SD. Conclusion: SD are substantially equally effective to HD to improve anemia and influencing survival in MDS patients stratified according to similar propensity to be exposed to rhEPO treatment.

KW - anemia

KW - erythropoietin

KW - myelodysplastic syndromes

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