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Chronic myelogenous leukemia

Prognosis of long-term survival considering disease-specific death in patients with chronic myeloid leukemia

Abstract

In patients with chronic myeloid leukemia (CML), first-line imatinib treatment leads to 8-year overall survival (OS) probabilities above 80%. Many patients die of reasons unrelated to CML. This work tackled the reassessment of prognosis under particular consideration of the probabilities of dying of CML. Analyses were based on 2290 patients with chronic phase CML treated with imatinib in six clinical trials. ‘Death due to CML’ was defined by death after disease progression. At 8 years, OS was 89%. Of 208 deceased patients, 44% died of CML. Higher age, more peripheral blasts, bigger spleen and low platelet counts were significantly associated with increased probabilities of dying of CML and determined a new long-term survival score with three prognostic groups. Compared with the low-risk group, the patients of the intermediate- and the high-risk group had significantly higher probabilities of dying of CML. The score was successfully validated in an independent sample of 1120 patients. In both samples, the new score differentiated probabilities of dying of CML better than the Sokal, Euro and the European Treatment and Outcome Study (EUTOS) score. The new score identified 61% low-risk patients with excellent long-term outcome and 12% high-risk patients. The new score supports the prospective assessment of long-term antileukemic efficacy and risk-adapted treatment.

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Acknowledgements

The valuable assistance of Doris Lindörfer, Annett Schmitt, Christina Niedermeier and Kirsi Manz is gratefully appreciated. We would like to thank Sina Hehn for providing link and tool in order to calculate the ELTS score. The EUTOS is a common project of the European LeukemiaNet and Novartis Oncology Europe. Novartis Oncology Europe provided financial support for the EUTOS project.

Author contributions

MP, JH and BS designed the work. All authors were involved in the collection and assembling of the data. MP performed the statistical analysis. MP VSH, RH, MB, JH and JG had an important role in interpreting the results. MP wrote the first draft of the manuscript; all authors participated in the revision. All others approved the final version. MP has had full access to the data in the study and final responsibility for the decision to submit for publication.

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Correspondence to M Pfirrmann.

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Competing interests

MP acted as a consultant for and received honoraria from Novartis and Bristol-Myers Squibb. MB served on the speakers’ bureau of and received honoraria from ARIAD, Bristol-Myers Squibb, Novartis and Pfizer and acted as a consultant for ARIAD and Novartis. SS acted as a consultant for Novartis, and Pfizer, received honoraria ARIAD, Bristol-Myers Squibb, Novartis and Pfizer, and received research funding from Bristol-Myers Squibb and Novartis. FC served on the speakers’ bureau of and received honoraria from ARIAD, Bristol-Myers Squibb and Novartis and acted as a consultant for ARIAD and Pfizer. GO acted as a consultant for ARIAD, Bristol-Myers Squibb, Celgene, Johnson & Johnson, Novartis and Roche and received research funding from Celgene and Novartis. VSH received research funding from Novartis. F Castagnetti acted as a consultant for and received honoraria from ARIAD, Bristol-Myers Squibb, Novartis and Pfizer. JH received honoraria from Bristol-Myers Squibb and research funding from Novartis; RH received research funding from Bristol-Myers Squibb and Novartis. BS acted as a consultant for and received honoraria from Novartis and Bristol-Myers Squibb.

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Pfirrmann, M., Baccarani, M., Saussele, S. et al. Prognosis of long-term survival considering disease-specific death in patients with chronic myeloid leukemia. Leukemia 30, 48–56 (2016). https://doi.org/10.1038/leu.2015.261

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