What Are the Attributes Prioritized in the Choice of Therapy in Chronic Lymphocytic Leukemia? A Patient-physician Cross-matching Analysis of a Discrete Choice Experiment

Abstract

Several treatment options are available for chronic lymphocytic leukemia (CLL) and, for this reason, treatment choice can result challenging after introducing oral targeted agents. This study aims at comparing patients’ and hematologists’ preferences for attributes of CLL treatments. An online cross-sectional survey has been delivered to clinicians and patients affected by CLL in Italy. A discrete choice experiment has been conducted so to estimate each attribute’s relative importance (RI) and assess the preference weight for each level of each attribute. An expert panel agreed on investigating the following attributes: progression-free survival (PFS) and measurable residual disease, route of administration/therapy duration and follow-up frequency, incidence of diarrhea (episodes/day), serious infections (grade 3 or 4), and atrial fibrillation. Overall, 746 patients and 109 clinicians accessed the survey, and 215 and 69, respectively, filled it in. The most important attributes were PFS (RI 30%) for hematologists and the risk of severe infections (RI 24%) for patients. Clinicians rated preference for maximum efficacy and lowest risk of severe infection very high (30%). Both patients and clinicians preferred oral administration while considering duration of therapy less relevant. The frequency of hospital appointments was negligible for patients, while clinicians preferred a quarterly frequency. Considering all attributes, diarrhea was weighted more by clinicians than by patients. Atrial fibrillation was not relevant for clinicians, while it was not negligible for patients. In conclusion, clinicians and patients favor an oral therapy, including continuous treatment, if associated with prolonged PFS, albeit with particular attention to the risk of serious infections.

Realizing precision medicine in chronic lymphocytic leukemia: Remaining challenges and potential opportunities

“…67 Once the need for treatment is established, surveys have shown that patients with CLL pay more attention to the occurrence of adverse events rather than to survival advantages when discussing therapy initiation. 68 Efficacy and safety are strongly dependent upon patients' situation at the time of treatment decision, and their balance should be a priority for personalized approaches, especially since patients with CLL are elderly with a life expectancy that might be dependent on other concomitant diseases rather than CLL itself.…”