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模拟类风湿关节炎中功能和疾病活动对成本及生活质量的影响。

Modelling the effect of function and disease activity on costs and quality of life in rheumatoid arthritis.

作者信息

Kobelt G, Lindgren P, Lindroth Y, Jacobson L, Eberhardt K

机构信息

Karolinska Institute, Stockholm, Sweden.

出版信息

Rheumatology (Oxford). 2005 Sep;44(9):1169-75. doi: 10.1093/rheumatology/keh703. Epub 2005 Jun 14.

Abstract

OBJECTIVE

When treatments with the potential to change the natural history of a disease are introduced, their longer-term effect on costs and quality of life (utility) has to be estimated using economic models. However, to remain useful tools, models must be updated when new information becomes available. Our earlier models in rheumatoid arthritis (RA) have been based on functional status, but it has recently been shown that disease activity might have an independent effect on utility. The objective of this study was to improve the model by incorporating the effect of a subjective measure of disease severity and activity (global VAS).

METHODS

A Markov model was constructed with five states according to functional status (HAQ), and each state was subdivided according to the VAS (<40 and >40). Disease development (transition probabilities between the states) was taken from a longitudinal cohort study of patients with early RA in Sweden. A recent population-based survey of 616 patients with RA provided data on costs and utilities. The model incorporates the full distribution of costs and utilities from the survey, and long-term projections are made using Monte Carlo simulation.

RESULTS

The global VAS had a highly significant effect on utilities independently of HAQ. For resource consumption, only HAQ was a significant predictor, with the exception of sick leave, which was correlated with the VAS but not with HAQ. Using the cohort distribution from the longitudinal study, expected mean costs per patient over 10 yr were 106 034 euros (s.d. 5091 euros) (1 euro = SEK 9.20) and the expected number of quality-adjusted life years (QALYs) was 5.08 (s.d. 0.09). Patients starting at HAQ <0.6 but with consistently high VAS scores would have expected costs of 102 830 euros and 4.96 QALYs, while patients with low VAS scores would have costs of 81 603 euros and 6.01 QALYs.

CONCLUSION

Our new model incorporates for the first time the effect of a subjective measure of disease severity and activity on both costs and utility, making it a sensitive tool to estimate the cost-effectiveness of disease-modifying treatments. New data on resource consumption indicate a shift to higher direct costs, particularly in early disease, and lower indirect costs in more advanced disease. The large size of the data sets used in this model reduces the uncertainty and makes estimates very stable.

摘要

目的

当引入有可能改变疾病自然史的治疗方法时,必须使用经济模型来估计其对成本和生活质量(效用)的长期影响。然而,为了保持有用的工具,模型必须在有新信息时进行更新。我们早期关于类风湿性关节炎(RA)的模型是基于功能状态,但最近研究表明疾病活动可能对效用有独立影响。本研究的目的是通过纳入疾病严重程度和活动的主观测量指标(整体视觉模拟评分,global VAS)来改进模型。

方法

根据功能状态(健康评估问卷,HAQ)构建了一个具有五个状态的马尔可夫模型,每个状态根据VAS评分(<40和>40)进一步细分。疾病发展(状态之间的转移概率)取自瑞典一项针对早期RA患者的纵向队列研究。最近一项基于人群的对616例RA患者的调查提供了成本和效用数据。该模型纳入了调查中成本和效用的完整分布,并使用蒙特卡罗模拟进行长期预测。

结果

整体VAS对效用有高度显著的独立于HAQ的影响。对于资源消耗,除病假与VAS相关而非与HAQ相关外,只有HAQ是一个显著的预测因素。根据纵向研究的队列分布,每位患者10年的预期平均成本为106034欧元(标准差5091欧元)(1欧元 = 9.20瑞典克朗),预期质量调整生命年(QALY)数为5.08(标准差0.09)。起始HAQ<0.6但VAS评分持续较高的患者预期成本为102830欧元和4.96 QALY,而VAS评分低的患者成本为81603欧元和6.01 QALY。

结论

我们的新模型首次纳入了疾病严重程度和活动的主观测量指标对成本和效用的影响,使其成为估计改善病情治疗成本效益的敏感工具。资源消耗的新数据表明向更高直接成本(特别是在疾病早期)的转变,以及在更晚期疾病中更低的间接成本。本模型中使用的数据集规模较大,减少了不确定性并使估计非常稳定。

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