Predicting complete cytogenetic response and subsequent progression-free survival in 2060 patients with CML on imatinib treatment: the EUTOS score

Joerg Hasford(Zimmer Biomet (Germany)), Michele Baccarani(IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola), Verena Hoffmann(Zimmer Biomet (Germany)), Joëlle Guilhot(Inserm), Susanne Saußele(Heidelberg University), Gianantonio Rosti(IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola), François Guilhot(Inserm), Kimmo Porkka(Helsinki University Hospital), Gert J. Ossenkoppele(Vrije Universiteit Amsterdam), Doris Lindoerfer(Zimmer Biomet (Germany)), Bengt Simonsson(Uppsala University Hospital), Markus Pfirrmann(Zimmer Biomet (Germany)), Rüdiger Hehlmann(Heidelberg University)
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Abstract

The outcome of chronic myeloid leukemia (CML) has been profoundly changed by the introduction of tyrosine kinase inhibitors into therapy, but the prognosis of patients with CML is still evaluated using prognostic scores developed in the chemotherapy and interferon era. The present work describes a new prognostic score that is superior to the Sokal and Euro scores both in its prognostic ability and in its simplicity. The predictive power of the score was developed and tested on a group of patients selected from a registry of 2060 patients enrolled in studies of first-line treatment with imatinib-based regimes. The EUTOS score using the percentage of basophils and spleen size best discriminated between high-risk and low-risk groups of patients, with a positive predictive value of not reaching a CCgR of 34%. Five-year progression-free survival was significantly better in the low- than in the high-risk group (90% vs 82%, P = .006). These results were confirmed in the validation sample. The score can be used to identify CML patients with significantly lower probabilities of responding to therapy and survival, thus alerting physicians to those patients who require closer observation and early intervention.


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