Treatment expectations and goals among patients with chronic myeloid leukemia in Germany: a patient-centered perspective

Leukemia, vol. 40, pp. 29–36

Abstract

Chronic myeloid leukemia (CML) is a chronic condition with excellent long-term survival under tyrosine kinase inhibitor (TKI) therapy. However, patient priorities regarding treatment goals and quality of life remain insufficiently understood. We conducted a nationwide online survey among German CML patients in collaboration with the German CML Alliance and patient organizations to assess treatment goals at diagnosis, during therapy, and in the treatment-free remission phase. The questionnaire included validated measures of treatment satisfaction, fear of progression, and quality-of-life priorities, supplemented by newly developed items. Between November 2024 and February 2025, 582 patients (median age 56 years, 48.8% female) completed the survey. Overall TKI tolerability was rated positively (median 4/5), particularly in first-line therapy and among patients with shorter disease duration, whereas long-term survivors reported more daily-life limitations. Younger patients ( < 45 years) emphasized fertility, sexuality, and work-related concerns, while older patients prioritized tolerability, independence, and mental health. Treatment history shaped expectations: those with discontinuation experience were more willing to accept adverse effects for the prospect of remission. Overall, patient priorities diverged between achieving deep molecular response and maintaining optimal tolerability. Integrating patient-reported preferences into shared decision-making may enhance satisfaction, adherence, and long-term outcomes of patients with CML.

Authors 15

1. Philipp Ernst corresponding

   Jena University Hospital

   Affiliation as printed

   Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany. philipp.ernst@med.uni-jena.de

2. Michael Lauseker

   Ludwig-Maximilians-Universität München · Institut für Medizinische Informationsverarbeitung, Biometrie und Epidemiologie

   Affiliation as printed

   Institut für Medizinische Informationsverarbeitung, Biometrie und Epidemiologie (IBE), Ludwig-Maximilians-Universität, München, Germany

3. Jan Geißler
   Affiliation as printed

   LeukaNET e.V., Riemerling, München, Germany

4. Philipp le Coutre

   Charité - Universitätsmedizin Berlin

   Affiliation as printed

   Klinik für Hämatologie, Onkologie und Tumorimmunologie, Charité-Universitätsmedizin Berlin, Berlin, Germany

5. Tim H. Brümmendorf Aachen Klinik für Onkologie Hämatologie und Stammzelltransplantation

   RWTH Aachen University · Universitätsklinikum Aachen

   Affiliation as printed

   Klinik für Onkologie, Hämatologie und Stammzelltransplantation, Universitätsklinikum RWTH Aachen, Center for Integrated Oncology (CIO) Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany

6. Susanne Saußele

   University Medical Centre Mannheim

   Affiliation as printed

   III. Medizinische Klinik, Universitätsmedizin Mannheim, Universität Heidelberg, Mannheim, Germany

7. Andreas Burchert

   Universitätsklinikum Gießen und Marburg

   Affiliation as printed

   Klinik für Hämatologie, Onkologie und Immunologie, Universitätsklinikum Marburg, Marburg, Germany

8. Georg‐Nikolaus Franke

   University Hospital Leipzig · Leipzig University

   Affiliation as printed

   Klinik und Poliklinik für Hämatologie, Zelltherapie, Hämostaseologie und Infektiologie, Universitätsklinikum Leipzig, Comprehensive Cancer Center Central Germany, Campus Leipzig, Leipzig, Germany

9. Paul La Rosée

   Schwarzwald-Baar Klinikum

   Affiliation as printed

   Klinik für Innere Medizin II, Schwarzwald-Baar Klinikum, Villingen-Schwenningen, Germany

10. Annamaria Brioli

    Medizinische Hochschule Hannover

    Affiliation as printed

    Klinik für Hämatologie, Hämostaseologie, Onkologie und Stammzelltransplantation, Medizinische Hochschule Hannover, Hannover, Germany

11. Thomas Schenk

    Jena University Hospital

    Affiliation as printed

    Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany

12. Christian Fabisch

    Jena University Hospital

    Affiliation as printed

    Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany

13. Thomas Ernst

    Jena University Hospital

    Affiliation as printed

    Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany

14. Guido Mehlkop

    University of Erfurt

    Affiliation as printed

    Institute for Planetary Health Behaviour, Universität Erfurt, Erfurt, Germany

15. Andreas Hochhaus

    Jena University Hospital

    Affiliation as printed

    Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany