The Effects of PFS, Toxicity Risks on Choosing Novel Agents in CLL

The introduction of novel agents has changed the treatment landscape for chronic lymphocytic leukemia (CLL), but these agents have different toxicity profiles. A study evaluated how differences in efficacy and toxicity profiles influence treatment choices among patients with CLL and oncologists. The results were presented at the 62nd ASH Annual Meeting & Exposition.

Patients with CLL and oncologists filled out an online survey with a discrete choice experiment (DCE), which quantified first-line treatment preferences. The DCE allowed respondents to choose between hypothetical treatment profiles that had eight attributes with varying levels. The attributes were chosen after qualitative interviews with oncologists and patients garnered information on treatment priorities, while the levels of attributes were obtained from clinical trials and published literature. Using the preference weights, a base case hypothetical treatment profile was created, and other hypothetical profiles with different adverse event (AE) risks, and two-year progression-free survival (PFS) rates were assessed against it to determine the attributes and levels that most significantly influenced treatment choices.

Overall, 151 oncologists with a mean 16.3 years in practice responded. The majority practiced in a community setting (72%) and in a major metropolitan/urban area (64%). There were 220 patient respondents; the median age was 56 years, and at the time of the study, the mean disease duration was two years. Two-thirds of patients (68%) were receiving or had completed at least first-line therapy.

Among the oncologists, the most significant influencers of treatment preferences were decreasing two-year PFS from 95% to 75%, reducing the risk of atrial fibrillation from 20% to 5% and reducing the risk of infection from 30% to 7%. The oncologists preferred the profile with reduced two-year PFS and reduced risk of AEs. The factors that most significantly influenced treatment selection were the same for patients, but patients preferred the profile with higher PFS and higher risk of AEs.

“This is an area for future research and highlights the importance of oncologists explicitly communicating the known efficacy benefits and AE risks associated with each treatment option because these factors may influence patients’ treatment choice in a way that differs from their own,” the study authors concluded.