背景:为了帮助适应溃疡性结肠炎(UC)的复杂治疗环境,我们量化了患者在选择治疗时愿意进行的获益-风险权衡.
方法:患者完成了在线离散选择实验。符合条件的患者诊断为UC≥6个月,年龄≥18岁,居住在法国,德国,意大利,西班牙,或者英国。患者在2种假设的治疗之间进行选择,以确保做出权衡。临床试验数据,文献综述,和患者访谈确定的治疗属性。产生相对属性重要性(RAI)评分和最大可接受风险。以患者为中心的200mg菲尔戈替尼的获益-风险评估作为一个例子,以显示如何使用测量的权衡。
结果:总体而言,631名患者参加;患者的平均年龄为42.2岁,主要为男性(75.3%)。实现和维持临床缓解是患者最重要的因素(RAI32.4%);要实现这一点,患者愿意接受稍高的血栓风险,严重感染,和恶性肿瘤相比,风险较低的治疗方案。患者还重视口腔治疗的便利性,避免使用类固醇,以及上学/工作的能力。以患者为中心的获益-风险评估表明,与安慰剂相比,患者更可能更喜欢Janus激酶1优先抑制剂filgotinib。
结论:实现临床缓解是患者的最高优先治疗。为了实现这一点,患者愿意接受一些风险稍高的治疗方案.患者在获益-风险评估中的选择表明,患者更有可能更喜欢菲戈替尼,而不是安慰剂。
患者愿意接受较高风险的治疗方案,而不是较低风险的治疗方案,以增加实现和维持缓解的机会。以患者为中心的获益-风险评估表明,200mg菲尔戈替尼具有可接受的获益-风险特征。
BACKGROUND: To help navigate the complex treatment landscape of ulcerative colitis (UC), we quantified the benefit-risk trade-offs that patients were willing to make when choosing treatment.
METHODS: Patients completed an online discrete choice experiment. Eligible patients had a UC diagnosis for ≥6 months, were aged ≥18 years, and resided in France, Germany, Italy, Spain, or the UK. Patients chose between 2 hypothetical treatments set up to ensure trade-offs were made. Clinical
trial data, literature review, and patient interviews identified treatment attributes. Relative attribute importance (RAI) scores and maximum acceptable risks were generated. A patient-centric benefit-risk assessment of 200 mg of filgotinib was conducted as an example to show how measured trade-offs can be used.
RESULTS: Overall, 631 patients participated; patients had a mean age of 42.2 years and were predominantly male (75.3%). Achieving and maintaining clinical remission was the most important factor for patients (RAI 32.4%); to achieve this, patients were willing to accept slightly higher risks of blood clots, serious infections, and malignancies compared with lower risk treatment profiles. Patients also valued the convenience of oral treatments, avoiding steroids, and the ability to attend school/work. The patient-centric benefit-risk assessment suggested patients are significantly more likely to prefer Janus kinase 1 preferential inhibitor filgotinib over placebo.
CONCLUSIONS: Achieving clinical remission was the highest treatment priority for patients. To attain this, patients were willing to accept some slightly higher risk treatment profiles. Patient choices in the benefit-risk assessment suggested patients were significantly more likely to prefer filgotinib over placebo.
Patients were willing to accept slightly higher risk treatment profiles over lower risk treatment profiles for an increased chance of achieving and maintaining remission. A patient-centric benefit-risk assessment suggested 200 mg of filgotinib had an acceptable benefit-risk profile.