Tolley Health Economics, Buxton, UK.
Eur J Health Econ. 2013 Oct;14(5):749-59. doi: 10.1007/s10198-012-0419-2. Epub 2012 Sep 1.
In the United Kingdom (UK), chronic lymphocytic leukaemia (CLL) makes up 40 % of all leukaemias in patients over 65 years. The study objective was to obtain societal preferences in the UK for "progression-free" and "progressive" states of late-stage CLL, refractory to current first and second line regimens. Preferences were also obtained for selected treatment-related adverse events (AEs).
A utility elicitation study, using the time trade-off (TTO) method, was conducted by face-to-face interviews with 110 subjects for a baseline disease state (before treatment), three primary disease states [progression-free survival (PFS) and treatment responder, PFS and treatment non-responder and disease progression], and 4 AE sub-states (PFS responder with thrombocytopenia, neutropenia, and infection, and PFS non-responder with infection). TTO scores were converted into utility values, and disutilities were calculated for AEs. Visual analogue scale (VAS) scores were obtained.
The primary disease state mean TTO utility scores were: baseline: 0.549; PFS response: 0.671; PFS non-response: 0.394; and progression: 0.214. The mean TTO utility (disutility) scores for the AEs were: PFS response with thrombocytopenia, 0.563 (-0.108), neutropenia, 0.508 (-0.163), and infection, 0.476 (-0.195); PFS non-response with infection, 0.333 (-0.061). The VAS results were in line with the TTO results.
The utility was higher for the PFS state than baseline, but decreased below baseline in non-response and disease progression states. AEs had an impact on utility within the PFS response state. The severe infection AE had a greater impact on utilities for the responding to treatment state compared to the non-responder state.
在英国(UK),慢性淋巴细胞白血病(CLL)占 65 岁以上患者所有白血病的 40%。本研究的目的是获得英国社会对晚期 CLL 无进展和进展状态的偏好,这些状态对目前的一线和二线方案均无反应。还对选定的与治疗相关的不良事件(AE)获得了偏好。
使用时间权衡(TTO)方法进行了一项效用 elicitation 研究,通过 110 名患者的面对面访谈进行,针对基线疾病状态(治疗前)、三个主要疾病状态[无进展生存(PFS)和治疗应答者、PFS 和治疗无应答者以及疾病进展]和 4 个 AE 子状态(PFS 应答者伴血小板减少、中性粒细胞减少和感染,以及 PFS 无应答者伴感染)。TTO 评分转换为效用值,并计算 AE 的不效用值。还获得了视觉模拟量表(VAS)评分。
主要疾病状态的平均 TTO 效用评分分别为:基线:0.549;PFS 反应:0.671;PFS 无反应:0.394;以及进展:0.214。AE 的平均 TTO 效用(不效用)评分分别为:PFS 反应伴血小板减少症,0.563(-0.108),中性粒细胞减少症,0.508(-0.163),和感染,0.476(-0.195);PFS 无反应伴感染,0.333(-0.061)。VAS 结果与 TTO 结果一致。
与基线相比,PFS 状态的效用更高,但在无反应和疾病进展状态下低于基线。AE 在 PFS 反应状态下对效用有影响。与无反应状态相比,严重感染 AE 对治疗反应状态的效用影响更大。