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. 2019 Dec;8(18):7567-7576.
doi: 10.1002/cam4.2638. Epub 2019 Oct 27.

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

Enrico Balleari  1   2 , Rosa Angela Filiberti  3 , Chiara Salvetti  1   4 , Bernardino Allione  1   5 , Emanuele Angelucci  1   6 , Marco Bruzzone  3 , Tullio Calzamiglia  1   7 , Marina Cavaliere  1   8 , Maurizio Cavalleri  1   9 , Daniela Cilloni  1   10 , Marino Clavio  1   11 , Elena Crisà  1   4 , Anna Da Col  1   2 , Paolo Danise  1   12 , Federica Pilo  1   13 , Dario Ferrero  1   4 , Carlo Finelli  1   14 , Daniela Gioia  1 , Roberto Massimo Lemoli  1   11 , Elisa Masiera  1 , Emanuela Messa  1   15 , Maurizio Miglino  1   11 , Pellegrino Musto  1   16 , Esther Natalie Oliva  1   17 , Antonella Poloni  1   18 , Flavia Salvi  1   19 , Alessandro Sanna  1   20 , Marco Scudeletti  1   9 , Rodolfo Tassara  1   8 , Valeria Santini  1   21
Affiliations

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

Enrico Balleari et al. Cancer Med. 2019 Dec.

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.

Keywords: anemia; erythropoietin; myelodysplastic syndromes.

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Conflict of interest statement

EB served as a member of local advisory board for Janssen‐Cilag, Novartis, and Celgene. EA has received honoraria from Novartis and Celgene, involvement in local advisory boards for Jazz Pharmaceuticals and Roche and participation in DMC for Celgene and Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. DC has received honoraria from Novartis and Celgene. CF has received research funding, advisory committees, and speaker fees from Novartis, Janssen, and Celgene. PM received honoraria from and participation to advisory boards for Janssen e Amgen. ENO received research funding from Janssen‐Cilag. FP served as a member of advisory board for Novartis. VS has received honoraria from Celgene, Janssen, and Novartis. Advisory boards for Celgene, Janssen, Abbvie, Astex, Karyopharma, Acceleron. RAF, CS, BA, MB, TC, MC, MC, EC, ADC, PD, DF, DG, RML, EM, EM, MM, AP, FS, AS, MS, and RT have nothing to disclose.

Figures

Figure 1
Figure 1
Overall survival of propensity score‐matched patients according to rhEPO doses
Figure 2
Figure 2
Overall survival of patients according to erythroid response in both cohorts (A) and within different subgroups according to different rhEPO doses (B)
Figure 3
Figure 3
Forest plot of subgroup analyses of erythroid response (A) and overall survival (B) comparing rhEPO high dose (HD) vs rhEPO standard dose (SD) within the strata of each relevant clinical factor
See this image and copyright information in PMC

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