Apolo Andrea B, Michaels-Igbokwe Christine, Simon Nicholas I, Benjamin David J, Farrar Mallory, Hepp Zsolt, Mucha Lisa, Heidenreich Sebastian, Cutts Katelyn, Krucien Nicolas, Ramachandran Natasha, Gore John L
Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Evidera, Montreal, QC, Canada.
Patient. 2025 Jan;18(1):77-87. doi: 10.1007/s40271-024-00709-3. Epub 2024 Aug 28.
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.
患者偏好有可能影响局部晚期/转移性尿路上皮癌（la/mUC）新治疗方法的研发，因此我们探讨了la/mUC患者如何评估一线治疗的不同属性。
在针对文献进行综述并对医生、la/mUC患者及其护理人员进行定性访谈之后，开展了一项在线偏好调查和多维阈值设定（MDT）活动。治疗属性包括两项益处（总缓解率[ORR]、与膀胱癌相关的疼痛[评分0 - 100；100为可能的最严重疼痛]）和四项与治疗相关的风险（周围神经病变、严重副作用、轻至中度恶心、轻至中度皮肤反应）。采用狄利克雷回归来估计平均偏好权重。计算了边际效用以及患者为换取其他属性降低10分或降低10%而愿意接受的ORR降低幅度。
共招募了100名患者，他们自行完成了调查和MDT。患者的平均年龄为64.9岁（标准差7.6），54%为女性，38%为白人。所有纳入的治疗属性对偏好均有统计学显著影响。ORR的变化影响最大，其次是癌症相关疼痛和治疗相关风险。患者愿意接受ORR降低8.4%以将疼痛水平降低10分，或接受ORR降低7.8%以将周围神经病变风险降低10%。对于严重副作用、轻至中度恶心或皮肤反应降低10%，患者分别愿意接受ORR降低5.5%、3.7%或3.4%。
在所测试的属性中，ORR的变化对患者最为重要。患者在治疗属性之间进行了权衡，这表明为改善其他属性（如降低癌症相关疼痛或治疗相关不良事件的风险），较低的ORR可能是可以接受的。
