慢性淋巴细胞白血病治疗结局的人群偏好值：一项横断面效用研究。
Population preference values for treatment outcomes in chronic lymphocytic leukaemia: a cross-sectional utility study.
机构信息
Oxford Outcomes, Bethesda, MD, USA.
出版信息
Health Qual Life Outcomes. 2010 May 18;8:50. doi: 10.1186/1477-7525-8-50.
BACKGROUND
Given that treatments for chronic lymphocytic leukaemia (CLL) are palliative rather than curative, evaluating the patient-perceived impacts of therapy is critical. To date, no utility (preference) studies from the general public or patient perspective have been conducted in CLL. The objective of this study was to measure preferences for health states associated with CLL treatment.
METHODS
This was a cross-sectional study of 89 members of the general population in the UK (England and Scotland). Using standard gamble, each participant valued four health states describing response status, six describing treatment-related toxicities based on Common Toxicity Criteria, and two describing line of treatment. The health states incorporated standardized descriptions of treatment response (symptoms have "improved," "stabilized," or "gotten worse"), swollen glands, impact on daily activities, fatigue, appetite, and night sweats. Utility estimates ranged from 0.0, reflecting dead, to 1.0, reflecting full health.
RESULTS
Complete response (CR) was the most preferred health state (mean utility, 0.91), followed by partial response (PR), 0.84; no change (NC), 0.78; and progressive disease (PD), 0.68. Among the toxicity states, grade I/II nausea and nausea/vomiting had the smallest utility decrements (both were -0.05), and grade III/IV pneumonia had the greatest decrement (-0.20). The utility decrements obtained for toxicity states can be subtracted from utilities for CR, PR, NC, and PD, as appropriate. The utilities for second- and third-line treatments, which are attempted when symptoms worsen, were 0.71 and 0.65, respectively. No significant differences in utilities were observed by age, sex, or knowledge/experience with leukaemia.
CONCLUSIONS
This study reports UK population utilities for a universal set of CLL health states that incorporate intended treatment response and unintended toxicities. These utilities can be applied in future cost-effectiveness analyses of CLL treatment.
背景
鉴于慢性淋巴细胞白血病（CLL）的治疗方法是姑息性的而非治愈性的，因此评估患者对治疗的感知影响至关重要。迄今为止，尚未从普通人群或患者角度对 CLL 进行任何效用（偏好）研究。本研究旨在衡量与 CLL 治疗相关的健康状况的偏好。
方法
这是一项针对英国（英格兰和苏格兰）普通人群中的 89 名成员的横断面研究。使用标准赌博法，每位参与者对描述治疗反应状态的四个健康状态，描述基于常见毒性标准的六种治疗相关毒性的状态，以及描述两种治疗线的状态进行了评估。健康状态纳入了标准化的治疗反应描述（症状“改善”、“稳定”或“恶化”）、腺体肿大、对日常活动的影响、疲劳、食欲和盗汗。效用估计值范围从 0.0（反映死亡）到 1.0（反映完全健康）。
结果
完全缓解（CR）是最受欢迎的健康状态（平均效用为 0.91），其次是部分缓解（PR），为 0.84；无变化（NC），为 0.78；进行性疾病（PD），为 0.68。在毒性状态中，I/II 级恶心和恶心/呕吐的效用降低最小（均为-0.05），而 III/IV 级肺炎的效用降低最大（-0.20）。毒性状态的效用降低可以从 CR、PR、NC 和 PD 中适当扣除。当症状恶化时尝试的二线和三线治疗的效用分别为 0.71 和 0.65。年龄、性别或对白血病的了解/经验并未导致效用存在显著差异。
结论
本研究报告了英国人群对一套普遍的 CLL 健康状态的效用，这些状态纳入了预期的治疗反应和意外的毒性。这些效用可应用于未来对 CLL 治疗的成本效益分析。
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