In Acute Leukemia, Chance of Response Is Patient’s Top Concern

For patients with acute leukemia, the most important factor in choosing a therapy in the relapsed or refractory setting is the chance of a response, according to a new study.

However, authors also said they found a broad range of opinions among patients, with some caring about many different factors, and others focused narrowly on chance of response and quality of life. The study was published in the journal Patient Preference and Adherence.¹

Patients with relapsed or refractory acute leukemia tend to have poor outcomes, noted the study authors. For patients with both acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), about half of patients who achieve remission on first-line treatment will go on to experience a relapse. In the relapse setting, therapeutic options tend to be less effective, they added.

However, they said several studies examining patient therapeutic preferences have been published but that gaps in the scientific literature remain.

“For example, no studies have focused on the broader population of people with acute leukemia (ie, incorporating all types), which could enable a better understanding of how preferences differ by (sub) type,” they wrote. “Furthermore, no studies have focused specifically on the relapsed/refractory setting, where outcomes are typically poorer and preferences may therefore vary significantly.”

Five attributes of treatment were evaluated in this survey: mode of administration, quality of life during treatment, chance of response, duration of response, and quality of life during response | Image Credit: Tierney-stock.adobe.com

In the new study, the investigators created an online discrete choice survey seeking patients’ perspectives on 5 attributes of treatment: mode of administration, quality of life during treatment, chance of response, duration of response, and quality of life during response. The survey was made up of 12 scenarios in which patients had to choose between 2 hypothetical treatment options.

A total of 95 patients with acute leukemia completed the survey. Most (61 patients) had AML, and two-thirds (66 patients) had achieved remission and had not experienced a relapse. The mean age of the respondents was 53 years.

The investigators said the respondents generally broke down into 2 groups. The first group accounted for 60% of respondents; in this group, all of the attributes reached a level of statistical significance, meaning those patients cared about a wide variety of factors when making treatment decisions. Patients in this group also were more likely to have AML and to have a greater amount of time between their initial diagnosis and the time they completed the survey.

In the second group, which included 40% of respondents, the only attributes of treatment that were statistically important were quality of life during treatment and response, and the chance of a response. The authors said patients with ALL or acute promyelocytic leukemia were more likely to be in the second group.

Overall, the investigators said, the results show that treatment options with a greater chance of response would be most valued in the setting of relapsed or refractory disease.

The findings are largely similar to a 2021 study of more than 800 patients with AML in the US.² This study found risk of death and the risk of long-term averse effects to be the most important priorities for patients. However, this study also differed in that it was limited to patients with AML and it used a best-worst scaling instrument.

Mott and colleagues noted that the respondents in their new study came entirely through recruitment by a patient advocacy group, and they said it might be that people who seek help from a patient advocacy group have different priorities from the general patient population. They also cautioned that their sample size was small. They said future studies could evaluate whether there are differences in patient preferences across different countries and treatment settings. Another area worth investigating, they said, is how these preferences translate in feelings about specific treatment options.

References

1. Mott DJ, Hitch J, Nier S, Pemberton-Whiteley Z, Skedgel C. Patient preferences for treatment in relapsed/refractory acute leukemia in the United Kingdom: a discrete choice experiment. Patient Prefer Adherence. 2024;18:1243-1255. Published 2024 Jun 17. doi:10.2147/PPA.S442530

2. Richardson DR, Oakes AH, Crossnohere NL, et al. Prioritizing the worries of AML patients: Quantifying patient experience using best-worst scaling. Psychooncology. 2021;30(7):1104-1111. doi:10.1002/pon.5652