Understanding Patient and Physician Perceptions Regarding Treatment-Related Adverse Events in First-Line Chronic Lymphocytic Leukemia (CLL)
Authors: Sile Bersabeh , Horchi Dahbia , Rault Bleuenn , Mulvihill Emily , Beusterien Kathleen , Stewart Katherine , Palhares De Miranda Paulo Andre P. , Guillaume Xavier 
Published: December 2023

Background
Chronic Lymphocytic Leukemia (CLL) patients have various treatment options which are all associated with different adverse events. A limited number of studies have explored how patients and physicians perceive these adverse events in the frontline treatment setting. Understanding the similarities and differences in perceptions between physicians and CLL patients is an important factor in supporting shared decision-making.
Aims
The objective of this multi-country study was to understand the differences in patients' and physicians' perception on treatment-related adverse events in CLL.
Methods
CLL patients and physicians managing CLL (oncologists, hematologist-oncologists, and hematologists) were invited to complete an online survey. Participants were recruited from the US, UK, Germany, France, and Australia. The survey included a best-worst scaling (BWS) exercise to assess how ‘bothersome’ 14 potential adverse events associated with 1L CLL treatments were perceived. In a series of 18 BWS choice tasks, participants chose which was most ‘bothersome’ and which was ‘least bothersome’ among subsets of 4 adverse events. A hierarchical Bayesian (HB) model was used to estimate BWS scores for each treatment-related adverse event: BWS scores sum to 100 for each participant and a higher score indicates that an adverse event is relatively more ‘bothersome’.
Results
The study included 192 patients and 259 physicians. The top four most bothersome adverse events were the same for both patients and physicians, however, these were ranked in a different order (Table 1). Physicians ranked the ‘risk of cardiac events/atrial fibrillation/flutter that may require medical treatment and/or hospitalization’ and ‘risk of bleeding (hemorrhage) that requires medical intervention and/or hospitalization’ as their 2 most bothersome adverse events while patients ranked ‘Risk of infection, such as pneumonia or blood infections (sepsis) requiring medical intervention and/or hospitalization’ and ‘Risk of tumor lysis syndrome (TLS) which requires hospitalization’ as their 2 most bothersome adverse events (Table 1).
The 2 least bothersome adverse events for both physicians and patients were ‘Risk of mild-to-moderate muscle, joint or bone pain (mild-to-moderate pain not interfering with daily activities) that might go away with medication’ and ‘Risk of mild-to-moderate headache (not limiting daily activities or limiting only instrumental activities such as cooking, cleaning, transportation, laundry, etc.) that might go away with medication’ (Table 1). Among patients who had experienced an adverse event (N=111), 62 (56%) reported that it had impacted their quality of life. These patients rated the following adverse events as more bothersome compared to those who had not experienced adverse events impacting quality of life: ‘risk of infection, such as pneumonia or blood infections requiring medical intervention and/or hospitalization’ (BWS scores=13.88 vs. 11.79) and ‘a side effect resulting in stopping the medication’ (9.46 vs. 6.00). These patients also rated the following adverse events as least bothersome: ‘risk of severe headache that might lead to hospitalization’ (9.55 vs. 11.68), ‘risk of severe vomiting that might lead to hospitalization’ (8.97 vs. 10.70), and ‘risk of severe muscle joint or bone pain’ (7.32 vs. 10.59), compared to those not experiencing adverse events impacting quality of life.
Conclusions
Patients' and physicians' perceptions were generally aligned on what adverse events related to 1L CLL treatments are most and least bothersome. However, the rank-ordering of the most bothersome adverse events varied between patients and physicians: patients were more concerned about a risk of infection or tumor lysis syndrome whereas physicians thought that a risk of cardiac event and the risk of bleeding were the most bothersome adverse events. Patients expressed hardly any concern about a risk of mild-to-moderate headache that could be resolved with medication. Interestingly, patients who had experienced a prior adverse event had differing results to patients who had not. These results should further inform and facilitate conversations between physicians and patients and improve shared decision-making when selecting a 1L regimen to treat CLL.