A multinational study to explore patient preferences for chronic myeloid leukaemia treatments

Blood, vol. 146, pp. 4425

Abstract

Abstract INTRODUCTION: Tyrosine kinase inhibitors (TKIs) enable most patients with chronic phase chronic myeloid leukaemia (CP-CML) to achieve near normal life expectancy. However, they are associated with a range of side effects that can impact treatment adherence and eventually, efficacy. Maintaining tolerability is essential for sustaining optimal outcomes. Understanding how people with CP-CML weigh efficacy against toxicity is critical to ensuring treatment decisions reflect what matters most to them. METHODS: This study quantified preferences using a discrete choice experiment (DCE). Adults with CP-CML are recruited by patient advocacy groups from three regions: Europe (United Kingdom, France, Germany, Italy, Spain), North America (United States, Canada) and East Asia (China, Japan). The online DCE survey included 12 choice tasks, each asking participants to choose between two unlabelled, hypothetical TKIs described by six attributes. The six attributes that patients highlighted as important in qualitative interviews, and that were supported by published clinical data on existing TKIs were selected for the DCE and captured aspects of efficacy, toxicity and convenience: 1) severity of fatigue, 2) risk of gastrointestinal (GI) problems, 3) risk of respiratory problems, 4) risk of long-term cardiovascular (CV) problems, 5) chance of achieving deep molecular response (DMR) within two years and 6) mode of administration (MOA; combination of frequency of tablets +/- fasting requirements). The attribute levels were informed by efficacy and safety outcomes from recent TKI trials. The choice data were analysed using conditional logit models, and relative attribute importance (RAI) scores were calculated to capture the impact of each attribute on overall preferences. Benefit-risk trade-offs were estimated by dividing each attribute-level coefficient by the (continuous) DMR coefficient, to assess the trade-offs that patients may be willing to make. This pooled multinational analysis is interim; final results based on the full study sample will be ready and presented at the conference. RESULTS: As of 28 July 2025, a total of 423 respondents completed the survey in Europe and North America (333 and 90, respectively) and were included in the analysis. Overall, 66% of respondents were female and approximately 28% of respondents were aged between 18 and 44 inclusive, with 25% aged 65 and above. 46% of respondents were on first line treatment and 14% were in treatment-free remission. The most to least important attributes, as indicated by the RAI scores (in parentheses) were severity of fatigue (25.9%), chance of achieving DMR within two years (20.9%), risk of GI problems (17.7%), risk of respiratory problems (12.5%), MOA (11.6%), and risk of long-term CV problems (11.3%). There were no statistically significant differences in RAI scores by region (Europe vs. North America). Benefit-risk trade-off estimates showed that, on average, respondents would need a TKI to offer a 30-percentage point (pp) higher chance of achieving DMR within two years to accept a 75% risk of GI problems (compared to a 15% risk). A 21pp increase would be required to accept a 40% risk of respiratory problems (vs. 5%) and a 20pp increase would be required to accept a 25% risk of long-term CV problems (vs. 5%).CONCLUSIONS: In this multinational preference study of people living with CP-CML, attributes reflecting common symptoms and side effects (e.g., fatigue and GI problems) had a substantial impact on (hypothetical) TKI choice. Risk of long-term CV problems was not as important a concern as other side effects, potentially reflecting patients' experiences with these issues and their desire to avoid them. The chance of achieving DMR was a highly important factor in choices, however, the results indicate that patients are willing to accept a lower chance of achieving DMR to avoid side effects. These results highlight the importance of informed and shared decision-making for CP-CML patients in the context of their treatment decisions.

Authors 14

1. Jan Geißler
   Affiliation as printed

   1Leukemia Patient Advocates Foundation, Bern, Switzerland

2. Šarūnas Narbutas
   Affiliation as printed

   2Lithuanian Cancer Patient Coalition (POLA), Vilnius, Lithuania

3. Hannah Hussain

   Office Of Health Economics

   Affiliation as printed

   3The Office of Health Economics, London, United Kingdom

4. Priscila Radu

   Office Of Health Economics

   Affiliation as printed

   3The Office of Health Economics, London, United Kingdom

5. David John Mott

   Office Of Health Economics

   Affiliation as printed

   3The Office of Health Economics, London, United Kingdom

6. Tim H. Brümmendorf Aachen 4Department of Hematology

   Universitätsklinikum Aachen · RWTH Aachen University

   Affiliation as printed

   4Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Medical Center Aachen, Aachen, Germany

7. Anastasia Goussarova
   Affiliation as printed

   5CML Advocates Network, Bern, Switzerland

8. Thomas William LeBlanc

   Duke Cancer Institute

   Affiliation as printed

   6Duke Cancer Institute, Durham, United States

9. Mandy Ryan

   University of Aberdeen

   Affiliation as printed

   7University of Aberdeen, Aberdeen, United Kingdom

10. Nicole Schroeter
    Affiliation as printed

    8Olshan Patient Relations, Munich, Germany

11. Franz Alisch
    Affiliation as printed

    9Novartis Pharmaceuticals UK Ltd, London, United Kingdom

12. Mo Zhou
    Affiliation as printed

    10Novartis Pharmaceuticals Corporation, East Hanover, United States

13. Chris D. Skedgel

    Office Of Health Economics

    Affiliation as printed

    3The Office of Health Economics, London, United Kingdom

14. Denis Costello
    Affiliation as printed

    5CML Advocates Network, Bern, Switzerland