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Prognostic Scores for Patients with Chronic Myeloid Leukemia under Particular Consideration of Disease-Specific Death

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Chronic Myeloid Leukemia

Part of the book series: Hematologic Malignancies ((HEMATOLOGIC))

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Abstract

Prognostic scores are used to predict the outcome for individual patients. In chronic myeloid leukemia (CML), the Sokal, the Euro, the EUTOS, and the EUTOS long-term survival (ELTS) score are prognostic scores that were addressed in the latest CML treatment recommendations of the European LeukemiaNet (ELN). Due to the therapeutic success of tyrosine kinase inhibitors (TKIs), the proportion of patients with causes of death unrelated to CML was growing. To assess the potential of imatinib to prevent dying of CML in different risk groups, the ELTS score was modeled to discriminate probabilities of dying of CML while considering causes of death unrelated to CML as competing risks. Here, the prognostic performance of the Sokal, the Euro, the EUTOS, and the ELTS scores are comparatively assessed, in particular for the primary event: death due to CML. Implicit statistical particularities when treating other causes of death as competing risks are highlighted. In the presence of competing risks, the application of both the cause-specific hazard model and the subdistribution hazard model is recommended when investigating the influence of prognostic factors on the event of interest. A detailed explanation fosters the ability of hematologists to interpret the outcome of these hazard models and to understand the differences between them. Methodological challenges in the development of a prognostic model and the importance of its validation are outlined. In the most recent ELN recommendations published in 2020, the authors advocate the use of the ELTS score as the preferred method to assess baseline risk. Their advice is backed with statistical evidence. This chapter is in parts a modified and updated version of a previously published review (Pfirrmann et al., Ann Hematol 94 Suppl 2:S209–S218, 2015) and introduces the results of a recent comparison between different prognostic scores (Pfirrmann et al., Leukemia 34(8):2138–2149, 2020).

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  1. Institut für Medizinische Informationsverarbeitung, Biometrie und Epidemiologie – IBE, Ludwig-Maximilians-Universität, Munich, Germany

    Markus Pfirrmann, Michael Lauseker, Verena S. Hoffmann & Joerg Hasford

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    Rüdiger Hehlmann

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Pfirrmann, M., Lauseker, M., Hoffmann, V.S., Hasford, J. (2021). Prognostic Scores for Patients with Chronic Myeloid Leukemia under Particular Consideration of Disease-Specific Death. In: Hehlmann, R. (eds) Chronic Myeloid Leukemia. Hematologic Malignancies. Springer, Cham. https://doi.org/10.1007/978-3-030-71913-5_9

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  • DOI: https://doi.org/10.1007/978-3-030-71913-5_9

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-030-71912-8

  • Online ISBN: 978-3-030-71913-5

  • eBook Packages: MedicineMedicine (R0)

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