Aim: Patient preferences for the features of targeted chronic lymphocytic leukemia (CLL) therapies may differ. Materials & methods: A discrete-choice experiment (DCE) survey was administered to 229 respondents recruited through the CLL Society. Results: Respondents placed most importance on increasing the chance of progression-free survival (PFS) at 2 years from 70 to 90% and confirming results with measurable residual disease (MRD) testing instead of routine testing. Respondents also preferred daily oral administration over intravenous infusion every 4 weeks, fixed-duration treatments over treat-to-progression treatments and treatments with lower side effect risks. Reducing risk of tumor lysis syndrome was least important relative to changes in other attributes. Conclusion: The combination of improving PFS combined with confirming results using MRD testing was more important than changes in all other study attributes included in the DCE. Results from this study can help inform shared decision-making when selecting therapies for CLL.
Several targeted treatments are available for people with chronic lymphocytic leukemia (CLL). These treatments target specific proteins present in CLL cancer cells. They differ in how long they keep cancer from progressing, how the results are measured and the side effects they cause. Some targeted CLL treatments are taken as a daily pill, and others are given by intravenous infusion. Some targeted treatments are given for a fixed amount of time, and others are given until CLL progresses. We surveyed 229 US patients with CLL to understand what features they most value in a targeted CLL treatment. Survey participants were recruited through the CLL Society, a nonprofit organization devoted to education, support, advocacy and research for the CLL community. Survey results indicated that participants placed the most importance on increasing the chance that the cancer would not progress after 2 years from 70 to 90% and confirming results with measurable residual disease testing (which can detect minute levels of leukemia cells) instead of routine testing. Participants also preferred taking a pill every day over receiving an intravenous infusion every 4 weeks and preferred treatments given for a fixed amount of time over treatments given until CLL progresses. Participants preferred treatments with lower chances of tumor lysis syndrome (a potentially organ-damaging condition that may result following treatment), irregular heartbeat and fatigue. It is important for doctors to understand the treatment features that matter to people living with CLL so that they can work with patients individually to choose the right treatment.
BACKGROUND: This study aimed to elicit and quantify preferences for treatments for juvenile idiopathic arthritis (JIA). METHODS: We conducted a discrete-choice experiment among adolescents with JIA in the United States (US) (n = 197) and United Kingdom (UK) (n = 100) and caregivers of children with JIA in the US (n = 207) and UK (n = 200). In a series of questions, respondents chose between experimentally designed profiles for hypothetical JIA treatments that varied in efficacy (symptom control; time until next flare-up), side effects (stomachache, nausea, and vomiting; headaches), mode and frequency of administration, and the need for combination therapy. Using a random-parameters logit model, we estimated preference weights for these attributes, from which we derived their conditional relative importance. RESULTS: On average, respondents preferred greater symptom control; greater time until the next flare-up; less stomachache, nausea, and vomiting; and fewer headaches. However, adolescents and caregivers in the US were generally indifferent across varying modes and frequencies of administration. UK adolescents and caregivers preferred tablets, syrup, or injections to intravenous infusions. US and UK adolescents were indifferent between treatment with monotherapy or combination therapy; caregivers in the UK preferred treatment with combination therapy to monotherapy. Subgroup analysis showed preference heterogeneity across characteristics including gender, treatment experience, and symptom experience in both adolescents and caregivers. CONCLUSIONS: Improved symptom control, prolonged time to next flare-up, and avoidance of adverse events such as headache, stomachache, nausea, and vomiting are desirable characteristics of treatment regimens for adolescents with JIA and their caregivers.
Arthritis, Juvenile , Child , Humans , Adolescent , United States , Arthritis, Juvenile/drug therapy , Caregivers , Headache , Nausea , Vomiting
Treatments for unresectable hepatocellular carcinoma (HCC) have varying benefit-risk profiles. We elicited 200 US patients' preferences for attributes associated with various first-line systemic treatments for unresectable HCC in a discrete-choice experiment (DCE) survey. Respondents answered nine DCE questions, each offering a choice between two hypothetical treatment profiles defined by six attributes with varying levels: overall survival (OS), months of maintained daily function, severity of palmar-plantar syndrome, severity of hypertension, risk of digestive-tract bleeding, and mode and frequency of administration. A random-parameters logit model was used to analyze the preference data. Patients regarded an additional 10 months of maintaining daily function without decline to be as important or more important than 10 additional months of OS, on average. Respondents valued avoiding moderate-to-severe palmar-plantar syndrome and hypertension more than extended OS. A respondent would require >10 additional months of OS (the greatest increase presented in the study) on average to offset the increased burden of adverse events. Patients with unresectable HCC prioritize avoiding adverse events that would severely impact their quality of life over mode and frequency of administration or digestive-tract bleeding risk. For some patients with unresectable HCC, maintaining daily functioning is as important or more important than the survival benefit of a treatment.
