Abstract
The benefit of azacitidine treatment in survival of high-risk myelodysplastic syndromes (MDS) patients compared with conventional care treatment (CCT) has not been established outside clinical trials. To assess its effectiveness, we compared overall survival (OS) between azacitidine and conventional treatment (CCT) in high-risk MDS patients, excluding those undergoing stem cell transplantation, submitted to the Spanish MDS registry from 2000 to 2013. Several Cox regression and competing risk models, considering azacitidine as a time-dependent covariate, were used to assess survival and acute myeloblastic leukemia (AML) progression. Among 821 patients included, 251 received azacitidine. Median survival was 13.4 (11.8–16) months for azacitidine-treated patients and 12.2 (11–14.1) for patients under CCT (P=0.41). In a multivariate model, age, International prognostic scoring system and lactate dehydrogenase were predictors of OS whereas azacitidine was not (adjusted odds ratio 1.08, 95% confidence interval 0.86–1.35, P=0.49). However, in patients with chromosome 7 abnormalities, a trend toward a better survival was observed in azacitidine-treated patients (median survival 13.3 (11–18) months) compared with CCT (median survival 8.6 (5–10.4) months, P=0.08). In conclusion, our data show that, in spite of a widespread use of azacitidine, there is a lack of improvement in survival over the years. Identification of predicting factors of response and survival is mandatory.
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Acknowledgements
We thank all the contributors to the Spanish Registry of Myelodysplastic Syndromes for their efforts to keep the database. We also thank Teresa Cedena, Beatriz Arrizabalaga, Almudena Fernández, Fernando Ramos, Nicolas Diaz, Rosa Coll, María Calbacho, Jose Falantes, Bernardo González and Santiago Bonanad for their help to collect additional data.
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TB has served as advisory board member and consultant for Celgene. DV is part of the speaker bureau of Celgene, Amgen, GSK, Novartis, MSD and astellas, and member of advisory boards for Celgene, Amgen, GSK and Pfizer. GS has received honoraria and research funding from Celgene, Novartis and Amgen, and is on the advisory committee for Amgen, Böehringer-Ingelheim, Celgene, Merck-Sharp and Dohme and Novartis. The Spanish Group on Myelodysplastic Syndromes is sponsored by Celgene and Novartis. The remaining authors declare no conflict of interest.
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Bernal, T., Martínez-Camblor, P., Sánchez-García, J. et al. Effectiveness of azacitidine in unselected high-risk myelodysplastic syndromes: results from the Spanish registry. Leukemia 29, 1875–1881 (2015). https://doi.org/10.1038/leu.2015.115
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DOI: https://doi.org/10.1038/leu.2015.115
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