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Chronic myelogenous leukemia

Treatment and outcome of 2904 CML patients from the EUTOS population-based registry

Abstract

The European Treatment and Outcome Study (EUTOS) population-based registry includes data of all adult patients newly diagnosed with Philadelphia chromosome-positive and/or BCR-ABL1+ chronic myeloid leukemia (CML) in 20 predefined countries and regions of Europe. Registration time ranged from 12 to 60 months between January 2008 and December 2013. Median age was 55 years and median observation time was 29 months. Eighty percent of patients were treated first line with imatinib, and 17% with a second-generation tyrosine kinase inhibitor, mostly according to European LeukemiaNet recommendations. After 12 months, complete cytogenetic remission (CCyR) and major molecular response (MMR) were achieved in 57% and 41% of patients, respectively. Patients with high EUTOS risk scores achieved CCyR and MMR significantly later than patients with low EUTOS risk. Probabilities of overall survival (OS) and progression-free survival for all patients at 12, 24 and 30 months was 97%, 94% and 92%, and 95%, 92% and 90%, respectively. The new EUTOS long-term survival score was validated: the OS of patients differed significantly between the three risk groups. The probability of dying in remission was 1% after 24 months. The current management of patients with tyrosine kinase inhibitors resulted in responses and outcomes in the range reported from clinical trials. These data from a large population-based, patient sample provide a solid benchmark for the evaluation of new treatment policies.

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Acknowledgements

For the contribution to patient registration, data collection and follow-up, we thank Hana Klamova, Prague and Olga Cerna, Prague. The contribution of Jiri Mayer, Brno, as a chairman of the Czech Leukemia Study Group for Life, is also acknowledged. For the Camelia and Infinity study groups, very important support was given by Institution of Biostatistics and analyses, Masaryk University, Brno, Czech Republic (Vladimir Dusek, head, Jan Muzik, Zuzana Zbozinkova, Tomas Pavlik). In addition, Barbara Braithwaite, Florence Tarantin and the KROHEM (Croatian Cooperative Group for Hematologic Diseases) are acknowledged for their valuable contributions. We thank Catherine Sodan-Boyer and Gabi Bartsch for For the administrative support.

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Correspondence to V S Hoffmann.

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VSH, MB, and JH receive research funding from Novartis. FC has a consulting role and receives honoraria from Novartis, BMS, ARIAD and Pfizer. FR receives honoraria from BMS and has a consulting role at Novartis. LFC has a consulting role and receives funding for clinical research from Novartis, BMS and Pfizer. AT has a consulting role at Novartis, BMS and Pfizer. DZ receives funding of travel, accommodations or expenses from Novartis and BMS. AZ has a consulting or advisory role at and participated in a speakers’ bureau for Novartis and received research funding from BMS. BS has a consulting or advisory role at BMS. ZS has a consulting or advisory role at Novartis. TS has a consulting role at Novartis, BMS, Adamed and Angelini. RC has had research funding from Novartis, Bristol-Myers-Squibb and Pfizer, and has been on the speakers bureau of Novartis and Pfizer in the past 5 years. AB has participated in a speakers’ bureau and received research support from Novartis Pharma Services/Office in Serbia via the patient assistant support program. AH participated in a speakers’ bureau for Novartis and BMS, receives research funding and funding of travel, accommodations or expenses from Novartis and BMS, and provides expert testimony for Novartis and BMS. LG receives research grants from Novartis. SB receives honoraria from Novartis, Celgene and AOP, has a consulting or advisory role at Novartis and Celgene and receives funding of travel, accommodations or expenses from Novartis and AOP. PK has a consulting or advisory role at GSK, BMS and Novartis, receives funding from Novartis, provides expert testimony for Pfizer and Novartis and receives funding of travel, accommodations or expenses from BMS and Ariad. DL receives research funding from Novartis. SS has received research support from Novartis and BMS and receives honoraria from Novartis, BMS, Ariad and Pfizer. AH has received research funding from Novartis. RH has received research support from Novartis, Consultant for BMS. KI, GSF, DS, JG, SL, IZ, HE, PC, GO and GR have no conflict of financial interests to declare.

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Hoffmann, V., Baccarani, M., Hasford, J. et al. Treatment and outcome of 2904 CML patients from the EUTOS population-based registry. Leukemia 31, 593–601 (2017). https://doi.org/10.1038/leu.2016.246

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