Patient Preference Study – Patient preferences for treatment in relapsed/refractory acute leukemia
Objective

While first-line therapies are often effective, some patients do not achieve remission (known as ‘refractory disease’) and some proportion of those that do may subsequently relapse. The outlook following relapse is considerably worse, with poor long-term survival rates and limited effective treatment options. As a result, treatment decisions in this context can be complicated and may ultimately depend on individual preferences, given the trade-offs between survival benefits and treatment-related toxicity.

The importance of patient preference research for informing decision-making has been highlighted in recent years. It has been widely acknowledged that regulatory and reimbursement decisions can be informed by information on patients’ preferences for different treatment options, provided that treatment decisions are, to some degree, “preference-sensitive”.

Despite the relevance of patient preferences in this context, relatively little is known about the treatment preferences of people with acute leukemia. Richardson et al. (2020) used discrete choice experiment methodology and found that people with AML in the US prioritize treatments that offer a greater chance at remission, but that preference differed depending on the age and gender of respondents. In a subsequent study Richardson et al. (2021) explored the priorities of people with AML in the US using a best-worst scaling exercise and found that the primary concerns of patients were death and long-term treatment side effects. Saini et al. (2023) found that people with AML in the US, UK and Canada that had previously received a stem cell transplant prioritized post-transplant maintenance therapies that offered better quality of life, lower hospitalization durations, and a better chance of two-year relapse-free survival. More recently, a small pilot study by LoCastro et al. (2023) explored the preferences of older adults with AML in the US across multiple timepoints. They found that preferences varied between patients that were on more intensive treatment compared to those on lower intensity treatments.

Study objectives

While these studies have provided some insights into the priorities of people with acute leukemia, the majority have focused on AML, none have explored how preferences might differ across the broader acute leukemia population, and none have focused specifically on the relapsed/refractory setting.

This study sought to address this gap in literature by examining the treatment preferences of people with acute leukemia in this setting.

Deliverables

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 3 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

Full report available : https://acuteleuk.org/publications/
Publications

We have partnered with OHE for this study. For more information, please visit OHE – Health Economics and Outcomes Research