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头颈癌患者健康状态效用测量的比较

Comparison of Health State Utility Measures in Patients With Head and Neck Cancer.

作者信息

Noel Christopher W, Lee Daniel J, Kong Qin, Xu Wei, Simpson Colleen, Brown Dale, Gilbert Ralph W, Gullane Patrick J, Irish Jonathan C, Huang Shao Hui, O'Sullivan Brian, Goldstein David P, de Almeida John R

机构信息

Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.

Department of Biostatistics, University Health Network, Toronto, Ontario, Canada3Department of Statistics and Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada.

出版信息

JAMA Otolaryngol Head Neck Surg. 2015 Aug;141(8):696-703. doi: 10.1001/jamaoto.2015.1314.

Abstract

IMPORTANCE

Accurate measurement of health state utilities (HU) is the cornerstone for cost-utility analyses and the valuation of quality of life for given health states. Current indirect methods of HU derivation lack face validity for patients with head and neck cancer. The appropriateness of these measures compared with direct methods, such as the standard gamble (SG), time trade-off (TTO), and visual analog scale (VAS), have not been assessed in this patient population.

OBJECTIVE

To assess the convergent and construct validities of 5 different HU derivation methods in patients with head and neck cancer.

DESIGN, SETTING, AND PARTICIPANTS: In a cross-sectional study, we recruited 100 consecutive patients with squamous cell carcinoma of the upper aerodigestive tract treated in the outpatient surgical oncology clinics of the Princess Margaret Cancer Centre from August 1 through October 31, 2014. We enrolled patients with a minimum of 3 months of follow-up after completion of treatment and no evidence of recurrent or metastatic disease. Participants completed SG, TTO, and VAS exercises, the EuroQoL instrument (EQ-5D), and the Health Utilities Index Mark 3 (HUI3) questionnaire. Data analysis was performed November 1 through December 15, 2014.

EXPOSURES

Head and neck cancer and HU measures.

MAIN OUTCOMES AND MEASURES

We assessed convergent validity of the 5 HU instruments through Spearman rank order correlation assessment. We determined construct validity through a priori hypotheses relating HU scores with clinical indexes of disease severity.

RESULTS

The SG and TTO measures generated higher mean (SD) utility scores (0.91 [0.17] and 0.94 [0.14], respectively) than the VAS, EQ-5D, and HUI3 (0.76 [0.19], 0.82 [0.18], and 0.75 [025], respectively) (P < .001). The maximum score of 1.0 was reported in 60 of 99 cases (61%) for the SG and 75 of 99 cases (76%) for the TTO (a significant ceiling effect), in contrast to 5 of 99 cases (5%) for the VAS, 29 of 99 cases (29%) for the EQ-5D, and 6 of 99 cases (6%) for the HUI3. The VAS showed strong correlations with the EQ-5D (ρ = 0.63 [P < .001]) and HUI3 (ρ = 0.50 [P < .001]), and the HUI3 strongly correlated with the EQ-5D (ρ = 0.67 [P < .001]), whereas the SG and TTO generally correlated poorly with other HU measures (ρ range, 0.19-0.29) and with one another (ρ = 0.21 [P < .001]). The VAS, EQ-5D, and HUI3 were able to discriminate between participants who underwent salvage surgery compared with those who underwent primary surgery (mean [SD] utility scores, 0.48 [0.13] vs 0.76 [0.20] [P = .006], 0.62 [0.17] vs 0.83 [0.19] [P = .004], and 0.37 [0.29] vs 0.78 [0.22] [P = .004], respectively). Mean EQ-5D utility scores monotonically increased over time since completion of treatment (0.26 [P = .01]). The HUI3 yielded lower utility values for participants with laryngeal cancer (mean [SD], 0.59 [0.29]). The SG and TTO measures frequently generated utility scores that contradicted our hypothesized expectations.

CONCLUSIONS AND RELEVANCE

Indirect HU measures may be more reflective of the health status of patients with head and neck cancer than direct measures. Current instruments lack face validity for attributes germane to this population.

摘要

重要性

准确测量健康状态效用值(HU)是成本效用分析以及特定健康状态下生活质量评估的基石。目前推导HU的间接方法对于头颈癌患者缺乏表面效度。与直接方法(如标准博弈法(SG)、时间权衡法(TTO)和视觉模拟量表(VAS))相比,这些测量方法在该患者群体中的适用性尚未得到评估。

目的

评估5种不同的HU推导方法在头颈癌患者中的收敛效度和结构效度。

设计、设置和参与者:在一项横断面研究中,我们于2014年8月1日至10月31日在玛格丽特公主癌症中心的门诊外科肿瘤诊所连续招募了100例上消化道鳞状细胞癌患者。我们纳入了治疗完成后至少随访3个月且无复发或转移疾病证据的患者。参与者完成了SG、TTO和VAS测试、欧洲生活质量量表(EQ - 5D)以及健康效用指数Mark 3(HUI3)问卷。数据分析于2014年11月1日至12月15日进行。

暴露因素

头颈癌和HU测量方法。

主要结局和测量指标

我们通过Spearman等级相关评估来评估5种HU工具的收敛效度。我们通过将HU得分与疾病严重程度的临床指标相关的先验假设来确定结构效度。

结果

SG和TTO测量产生的平均(标准差)效用得分(分别为0.91 [0.17]和0.94 [0.14])高于VAS、EQ - 5D和HUI3(分别为0.76 [0.19]、0.82 [0.18]和0.75 [0.25])(P <.001)。在99例病例中,SG有60例(61%)、TTO有75例(76%)报告了最高得分1.0(存在显著的天花板效应),相比之下,VAS有5例(5%)、EQ - 5D有29例(29%)、HUI3有6例(6%)。VAS与EQ - 5D(ρ = 0.63 [P <.001])和HUI3(ρ = 0.50 [P <.001])显示出强相关性,HUI3与EQ - 5D(ρ = 0.67 [P <.001])也显示出强相关性,而SG和TTO通常与其他HU测量方法的相关性较差(ρ范围为0.19 - 0.29),且彼此之间的相关性也较差(ρ = 0.21 [P <.001])。VAS、EQ - 5D和HUI3能够区分接受挽救性手术的参与者与接受初次手术的参与者(平均[标准差]效用得分分别为0.48 [0.13]对0.76 [0.20] [P =.006]、0.62 [0.17]对0.83 [0.19] [P =.004]、0.37 [0.29]对0.78 [0.22] [P =.004])。自治疗完成后,平均EQ - 5D效用得分随时间单调增加(0.26 [P =.01])。HUI3对头颈癌患者产生的效用值较低(平均[标准差],0.59 [0.29])。SG和TTO测量经常产生与我们假设预期相矛盾的效用得分。

结论与相关性

间接HU测量方法可能比直接测量方法更能反映头颈癌患者的健康状况。目前的工具对于该人群相关的属性缺乏表面效度。

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