Patient and physician decision-making on the use of novel agents in chronic lymphocytic leukemia (CLL): What drives preferences?

Abstract

148 Background: CLL treatment has changed dramatically with the approval of novel agents, but data to guide treatment decisions are still lacking. How patients (PTs) and oncologists (ONCs) prioritize treatment attributes is unknown. Methods: ONCs and PTs completed an online survey to quantify preferences for first-line (1L) CLL treatment with novel agents via a discrete choice experiment; ONCs and PTs chose between hypothetical treatment profiles with varying attribute levels taken from product labels, registrational trials, and real-world studies. Hierarchical Bayes models were used to estimate attribute level preference weights, which were used to compute relative importance (RI), a measure of how influential an attribute is in treatment choice out of a total of 100%. Results: For ONCs (N=151), 72% were in community practice. PTs (N=220) had a median age of 56 years; 32% were in active surveillance, 36% were in/had completed 1L treatment, and 32% were relapsed/refractory. Increasing 2-year progression-free survival (PFS) from 75% to 95% had the greatest impact on preferences, with a mean RI of 40% for PTs and 30% for ONCs (Table). When assessing trade-offs between 2-year PFS and other attributes, ONCs required the largest increase in PFS (11%) to compensate for an increased risk of atrial fibrillation (AF) from 5% to 20%. PTs required the largest increase in PFS (6%) to compensate for an increased risk of infection from 7% to 20%. ONCs (vs PTs) required 2–4-times higher increases in PFS to accept an increased risk of AF, discontinuation due to adverse events (AEs), bleeding, tumor lysis syndrome (TLS), and arthralgia/myalgia. Conclusions: ONCs and PTs valued PFS most when selecting a novel CLL agent. While both groups accepted potential risks in exchange for increased PFS, ONCs were less likely to accept a higher risk of AEs. ONCs and PTs may perceive the risks and benefits of novel agents differently. ONC–PT communication may be improved by a more focused discussion on the risks of AEs, relative to treatment outcomes, with patient goals in mind. [Table: see text]