Patient preferences for treatment in relapsed/refractory acute leukemia

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Key takeaways:

  • Across all five countries studied (UK, USA, France, Germany, and Italy), the chance of responding to treatment was by far the most important factor influencing patient preferences. Quality of life during response and duration of response were also valued, but to a lesser degree. 
  • Response-focused (47%) – prioritized chance of response above all else. Balanced decision-makers (32%) – considered multiple factors such as efficacy, quality of life, and duration. Convenience + efficacy focused (21%) – valued outpatient or at-home treatments and longer response duration. 
  • These findings emphasize that “one size does not fit all” in leukemia care. 
  • In this study, U.S. patients placed greater weight on treatment convenience (e.g., preferring oral or outpatient therapies) compared with Europeans, who focused more on clinical outcomes. This may reflect healthcare system differences and cultural attitudes toward hospitalization and cost. 
  • Outcomes such as quality of life, treatment burden, and convenience should be integrated into clinical trial design, health technology assessments, and policy decisions. Using patient preference data can help to ensure that new therapies and care pathways better reflect what patients truly value. 

When acute leukemia relapses, treatment decisions can be complex. Patients may face trade-offs between survival benefits, side effects and how treatment is delivered. This study asked people with acute leukemia across five countries what matters most to them when making these choices.

How did we do it?

  • We surveyed 267 patients in the UK, USA, France, Germany, and Italy. 
  • The survey included a task called a discrete choice experiment (DCE). In the DCE, patients made choices between different hypothetical treatment options. 
  • The treatments in the DCE were described in terms of how effective they are (the chance of responding, as well as the duration of the response), the impact on quality of life (during and after the treatment), and how the treatment is given (mode of administration). 
  • The DCE data were analyzed to identify the treatment priorities of people with acute leukemia in the event of a relapse.

What patients told us

The most important factor was how likely the treatments were to work

  • The chance of responding to treatment was the most important characteristic by a significant margin.
  • The second most important was quality of life during response, followed by the duration of response, and quality of life during treatment, respectively.
  • Mode of administration was the least important characteristic overall – though it was very important to some people.

Different groups of patients had different treatment priorities

  • We identified three distinct groups:
    • “Response-focused” (47%) – chance of response was by far their biggest priority compared to other characteristics.
    • “Balanced decision-makers” (32%) – all characteristics except the mode of administration were important to this group.
    • “Convenience + efficacy focused” (21%) – preferred to avoid hospital stays and prioritized a longer response duration. 

Key messages

This research paper, Understanding the preferences of people with acute leukemia  for different health outcomes, was commissioned and funded by the Acute Leukemia Advocates Network (ALAN).  

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