BACKGROUND: In addition to single-agent oral tyrosine kinase inhibitors, cancer immunotherapy (CIT) combinations are the new standard-of-care treatment for unresectable hepatocellular carcinoma (HCC). These treatments have varying benefit-risk profiles. This study quantified patients’ benefit-risk preferences for attributes associated with first-line systemic treatments for unresectable HCC and estimated the predicted likelihood of selecting hypothetical treatment profiles.
METHODS: A cross-sectional, web-based discrete-choice experiment survey was developed. Each question offered a choice between two hypothetical treatment profiles created by an experimental design and defined by six attributes with varying levels: overall survival (OS), number of months to maintain daily function, severity of palmar-plantar syndrome and hypertension, risk of bleeding in the digestive tract, and mode and frequency of administration. Data were analyzed using a random-parameters logit model, and the estimated preference weights were used to predict the probability of selecting between pairs of hypothetical treatment profiles.
RESULTS: 200 patients with self-reported unresectable HCC in the United States completed the survey (mean age 59 years, 25% White, and 56% male). On average, an additional 10 months of maintaining daily function was as important or more important than an additional 10 months of OS. Respondents placed the most importance on avoiding moderate-to-severe palmar-plantar syndrome and hypertension and the least importance on avoiding a 7% risk of bleeding and more convenient dosing. Table 1 presents the predicted probability of selecting between two treatments with different profiles for 3 distinct scenarios. In Scenarios 1 and 2, longer OS, maintaining daily function for more time, and a lack of palmar-plantar syndrome outweighed a greater risk of bleeding in the digestive tract and less convenient dosing. In Scenario 3, a lack of palmar-plantar syndrome, greater time of maintaining daily function and more convenient dosing outweighed a greater risk of bleeding in the digestive tract.
CONCLUSIONS: The results of the study provide insight into preferences across varying treatment attributes for unresectable HCC. When selecting between two hypothetical treatment profiles, patients preferred treatments that confer greater benefit in terms of OS and maintenance of daily function and avoid moderate-to-severe palmar-plantar syndrome and hypertension.
