Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments.
An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0-100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mild to moderate nausea, mild to moderate skin reactions). A Dirichlet regression was used to estimate average preference weights. Marginal utility and the reduction in ORR that patients would accept in exchange for a 10-point decrease or a 10% decrease in other attributes were calculated.
A total of 100 patients were recruited and self-completed the survey and MDT. Mean patient age was 64.9 years (standard deviation, 7.6), 54% were female, and 38% identified as white. All included treatment attributes had a statistically significant impact on preferences. Changes in ORR had the largest impact, followed by cancer-related pain and treatment-related risks. Patients were willing to accept an 8.4% decrease in ORR to reduce their pain level by 10 points or a 7.8% decrease in ORR to reduce the risk of peripheral neuropathy by 10%. For a 10% decrease in severe side effects, mild to moderate nausea, or skin reaction, patients would accept decreases in ORR of 5.5%, 3.7%, or 3.4%, respectively.
Of the attributes tested, changes in ORR were most important to patients. Patients made tradeoffs between treatment attributes indicating that a lower ORR may be acceptable for an improvement in other attributes such as reduced cancer-related pain or the risk of treatment-related adverse events.
The patient. 2024 Aug 28 [Epub ahead of print]
Andrea B Apolo, Christine Michaels-Igbokwe, Nicholas I Simon, David J Benjamin, Mallory Farrar, Zsolt Hepp, Lisa Mucha, Sebastian Heidenreich, Katelyn Cutts, Nicolas Krucien, Natasha Ramachandran, John L Gore
Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Evidera, Montreal, QC, Canada. ., Hoag Family Cancer Institute, Newport Beach, CA, USA., Pfizer Inc, Bothell, WA, USA., Astellas Pharma, Inc, Northbrook, IL, USA., Evidera, London, UK., Evidera, Montreal, QC, Canada., Department of Urology, University of Washington, Seattle, WA, USA.
PubMed http://www.ncbi.nlm.nih.gov/pubmed/39198374