Patients With CLL Place the Most Value on Treatments That Prolong PFS


Key Takeaways

  • Patients prioritize increasing 2-year PFS from 70% to 90% and prefer MRD testing over routine testing.
  • Daily oral administration and fixed-duration treatments are favored over IV infusions and treat-to-progression regimens.
Arliene Ravelo Mangalindan, MPH

Arliene Ravelo Mangalindan, MPH

Patients with chronic lymphocytic leukemia (CLL) valued therapies that increased the chance of progression-free survival (PFS) compared with other attributes, according to findings from a discrete-choice experiment survey published in Future Oncology.

Survey respondents (n = 229) placed the most importance on boosting the likelihood of 2-year PFS from 70% to 90% and confirming the findings via measurable residual disease (MRD) testing vs routine testing. Additionally, patients preferred the daily oral administration of anticancer agents compared with intravenous (IV) infusion every 4 weeks, treatments with a fixed duration over treat-to-progression treatments, and treatments with a lower risk of adverse effects (AEs).

“This discrete-choice experiment survey of adults with CLL was conducted to quantify their preferences over a set of treatment attributes corresponding to available chemoimmunotherapy and targeted therapy regimens used to treat [patients with] CLL,” Arliene Ravelo Mangalindan, MPH, the director of Oncology Health Economics & Outcomes Research at Genentech, and coauthors wrote. “Given the set of attributes and the ranges of levels for these attributes included in the survey, improving the chance of PFS at 2 years with results confirmed by MRD testing was the most important driver of treatment preferences among adults with CLL.”

Investigators developed a web-based discrete-choice experiment survey and recruited respondents who provided informed consent through the CLL Society. The survey instrument was developed via qualitative interviews with 20 adult patients with CLL; all patients defined efficacy, safety, mode of administration, and duration of treatment as important considerations for their treatment.

Survey participants responded to a series of 12 items and were asked to decide between 2 experimentally designed hypothetical CLL treatment profiles. The hypothetical treatments consisted of 7 differing attributes: chance of achieving PFS at 2 years (70% vs 90%), test by which the treatment results were confirmed (MRD vs routine testing), mode and frequency of administration (IV infusion every 4 weeks vs oral pill daily at home), duration of treatment (fixed for 6 months vs until disease progression), risk of tumor lysis syndrome (TLS) in the first few weeks after therapy, risk of atrial fibrillation each year during treatment, and risk of fatigue for several days each month during treatment. The 3 risk questions present varying percentages of increased risk of the AE between the 2 hypothetical treatments.

The objective of the survey was “to further explore patient preferences for attributes associated with newer CLL treatments, including the relative importance of changes in treatment attributes and the willingness of patients with CLL to trade off treatment-related benefits and risks.”

Eligible respondents needed to be at least 18 years of age, a United States resident, have a self-reported physician diagnosis of CLL for at least 3 months, and be able to read and understand English to provide informed consent. There were no exclusion criteria.

Survey respondents were a median age of 67 years old (IQR, 62-72). Most respondents were White (95.2%), female (59.4%), and retired (58.1%). Among respondents who had received prior therapy for CLL (n = 152), 71.1% were currently receiving treatment, 44.7% had been required to interrupt or discontinue CLL therapy due to AEs, and 45.4% had received MRD testing.

Patients who were currently receiving therapy (n = 108) were being treated with an oral targeted therapy at a rate of 88.0%. The mean duration of current treatment was 28.5 months.

Additional findings from the survey showed that reducing the risk of TLS was the least important to respondents vs changes in the other study attributes; respondents were willing to accept over a 3% risk of TLS in exchange for improvements in PFS, treatment duration, and mode of agent administration. Respondents valued confirming treatment results via MRD testing vs routine testing when the chance of PFS was constant, but MRD confirmation was approximately 1.6-times more important with a 70% chance of PFS vs 90%. Apart from clinically relevant ranges the largest reduction in the risk of atrial fibrillation from 10% to 0% was valued by respondents approximately as much as the largest reduction in the risk of fatigue from 35% to 0%.

Results from a subgroup analysis demonstrated that none of the 3 predefined subgroups yielded differences in treatment preferences that were statistically significant. The subgroups consisted of median age (≥ 67 years vs < 67 years}, age (≥ 65 years vs < 65 years), and treatment experience (prior or current treatment for CLL vs treatment naive).

Overall, investigators noted that respondents were more likely to choose a hypothetical treatment profile with attribute levels based on venetoclax (Venclexta) plus obinutuzumab (Gazyva) vs any other relevant comparator.

“Adult [patients] with CLL were willing to accept clinically relevant levels of risk for improvements in treatment efficacy and reductions in the burden of treatment administration, including oral pills and fixed-duration treatments,” Mangalindan and coauthors wrote in conclusion. “The combination of improving PFS combined with confirming results using MRD testing was more important than changes in all other study attributes. The findings from this study provide insight into which features adult [patients] with CLL consider important for their treatments and can help inform shared decision-making when selecting alternative therapies for CLL.”


Ravelo A, Myers K, Brumble R, et al. Patient preferences for chronic lymphocytic leukemia treatments: a discrete-choice experiment. Future Oncol. Published online May 22, 2024. doi:10.1080/14796694.2024.2348440

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