Hazlewood Glen S, Bombardier Claire, Tomlinson George, Thorne Carter, Bykerk Vivian P, Thompson Andrew, Tin Diane, Marshall Deborah A
Department of Medicine, University of Calgary, Calgary, Alberta
Institute of Health, Policy, Management and Evaluation, University of Toronto, Toronto, Ontario.
Rheumatology (Oxford). 2016 Nov;55(11):1959-1968. doi: 10.1093/rheumatology/kew280. Epub 2016 Jul 31.
To quantify the preferences of patients with early RA (ERA) with the benefits and harms of DMARDs.
We assessed patients' preferences using a discrete-choice experiment, an experimentally designed survey to measure trade-offs. Consecutive adult patients with ERA (<2 years since diagnosis) were presented 13 different sets of three treatment options described by eight attributes (clinical outcomes, risks and dosing regimens) and asked to choose one. From patients' responses we estimated the average importance of each attribute and explored preference heterogeneity through latent-class analysis.
A total of 152 patients completed the survey (86% response rate): mean age 52 years, 63% female, disease duration 7.8 months. Treatment benefits (increasing the chance of a major symptom improvement and reducing the chance of serious joint damage) were most important. Of potential adverse events, a small risk of serious infections/possible increased risk of cancer was most important. Patients were willing to accept this risk for a 15% absolute increase in the chance of a major symptom improvement. Patients had an aversion to i.v. therapy, but were relatively indifferent to other dosing regimens. Through latent-class analysis, we identified two patient groups: 54% who were more risk averse, particularly to a possible risk of cancer/infection, and others who were highly benefit-driven.
On average, patients with ERA were risk tolerant, but important differences in preferences were identified. In particular, a subgroup of patients may prefer to avoid treatments with a possible increased risk of cancer/infection if other effective options are available.
量化早期类风湿关节炎（ERA）患者对改善病情抗风湿药（DMARDs）的获益与危害的偏好。
我们采用离散选择实验评估患者的偏好，离散选择实验是一种用于测量权衡取舍的实验性设计调查。向连续纳入的成年ERA患者（诊断后<2年）展示13组不同的三种治疗方案，这些方案由八个属性（临床结局、风险和给药方案）描述，并要求他们选择其一。根据患者的回答，我们估计了每个属性的平均重要性，并通过潜在类别分析探索偏好异质性。
共有152例患者完成了调查（应答率86%）：平均年龄52岁，63%为女性，病程7.8个月。治疗获益（提高主要症状改善的机会和降低严重关节损伤的机会）最为重要。在潜在不良事件中，严重感染的小风险/可能增加的癌症风险最为重要。患者愿意接受这种风险以换取主要症状改善机会绝对增加15%。患者厌恶静脉内治疗，但对其他给药方案相对无差异。通过潜在类别分析，我们确定了两组患者：54%的患者更厌恶风险，尤其是对可能的癌症/感染风险，另一组则高度受获益驱动。
总体而言，ERA患者具有风险耐受性，但偏好存在重要差异。特别是，如果有其他有效选择，一部分患者可能更倾向于避免使用可能增加癌症/感染风险的治疗方法。
