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Clinical evaluation of the European LeukemiaNet response criteria in patients with essential thrombocythemia treated with anagrelide

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Abstract

This study investigates whether the response criteria proposed by the European LeukemiaNet (ELN) to evaluate cytoreductive therapies in essential thrombocythemia (ET) correlate with clinically relevant outcomes in patients receiving anagrelide. We evaluated 154 ET patients treated with anagrelide (upfront in 87) for a median of 2.9 years. Complete response (CR), partial response, and no response were observed in 56, 30.5, and 13.5 % patients, respectively. Only 38 patients (25 %) achieved a sustained CR. Overall, the aggregated time on CR and without CR was 200.1 and 333.6 person-years, respectively. The incidence rate of thrombosis and hemorrhage was independent of the CR status. The only factor associated with shorter survival after anagrelide start was the patient's age, whereas achieving a CR with anagrelide had no predictive value for subsequent survival. In conclusion, CR according to the ELN definition is not associated with any measurable clinical benefit in ET patients treated with anagrelide.

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Acknowledgments

The authors would like to thank the members of the Registro Español de Trombocitemia Esencial (RETE) for providing the patients' clinical information, as well as Juncal Sabaté for helping on collecting the database. This work was supported by the grants 09/02324 and 10/01807 from the Fondo de Investigaciones Sanitarias and RETICS RD12/0036/0010, from the Spanish Ministry of Health, and IMPULSA/2010 from the Conselleria de Sanitat, Generalitat Valenciana, Spain.

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The authors declare that they have no conflict of interest.

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Correspondence to Juan Carlos Hernández-Boluda.

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Hernández-Boluda, J.C., Pereira, A., Cervantes, F. et al. Clinical evaluation of the European LeukemiaNet response criteria in patients with essential thrombocythemia treated with anagrelide. Ann Hematol 92, 771–775 (2013). https://doi.org/10.1007/s00277-013-1683-7

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