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Cost-effectiveness of different treat-to-target strategies in rheumatoid arthritis: results from the DREAM registry
BMC Rheumatology Pub Date : 2019-04-29 , DOI: 10.1186/s41927-019-0064-9
Celine J van de Laar 1, 2 , Martijn A H Oude Voshaar 1, 2, 3 , Harald E Vonkeman 1, 2, 3, 4
Affiliation  

Adjusting medication of patients with rheumatoid arthritis (RA) until predefined disease activity targets are met, i.e. Treat-to-Target (T2T), is the currently recommended treatment approach. However, not much is known about long-term cost-effectiveness of different T2T strategies. We model the 5-year costs and effects of a step-up approach (MTX mono - > MTX + csDMARD combination - > Adalimumab - > second anti-TNF) and an initial combination therapy approach (MTX + csDMARD - > MTX + csDMARD higher dose - > anti-TNFs) from the healthcare and societal perspectives, by adapting a previously validated Markov model. We constructed a Markov model in which 3-monthly transitions between DAS28-defined health states of remission (≤2.6), low (2.6 < DAS28 ≤ 3.2), moderate (3.2 < DAS28 ≤ 5.1), and high disease activity (DAS28 > 5.1) were simulated. Modelled patients proceeded to subsequent treatments in case of non-remission at each (3-month) cycle start. In case of remission for two consecutive cycles medication was tapered, until medication-free remission was achieved. Transition probabilities for individual treatment steps were estimated using data of Dutch Rheumatology Monitoring registry Remission Induction Cohort I (step-up) and II (initial combination). Expected costs, utility, and ICER after 5 years were compared between the two strategies. To account for parameter uncertainty, probabilistic sensitivity analysis was employed through Gamma, Normal, and Dirichlet distributions. All utilities, costs, and transition probabilities were replaced by fitted distributions. Over a 5-year timespan, initial combination therapy was less costly and more effective than step-up therapy. Initial combination therapy accrued €16,226.3 and 3.552 QALY vs €20,183.3 and 3.517 QALYs for step-up therapy. This resulted in a negative ICER, indicating that initial combination therapy was both less costly and more effective in terms of utility gained. This can be explained by higher (±5%) remission percentages in initial combination strategy at all time points. More patients in remission generates less healthcare and productivity loss costs and higher utility. Additionally, higher remission percentages caused less bDMARD use in the initial combination strategy, lowering overall costs. Initial combination therapy was found favourable over step-up therapy in the treatment of Rheumatoid Arthritis, when considering cost-effectiveness. Initial combination therapy resulted in more utility at a lower cost over 5 years.

中文翻译:

类风湿性关节炎不同靶向治疗策略的成本效益:来自 DREAM 注册表的结果

目前推荐的治疗方法是调整类风湿性关节炎(RA)患者的用药直至达到预定的疾病活动目标,即达到治疗目标(T2T)。然而,人们对不同 T2T 策略的长期成本效益知之甚少。我们模拟了升压方法(MTX 单 - > MTX + csDMARD 组合 - > 阿达木单抗 - > 第二抗 TNF)和初始联合治疗方法(MTX + csDMARD - > MTX + csDMARD 更高)的 5 年成本和效果剂量 - > 抗肿瘤坏死因子)从医疗保健和社会的角度,通过采用先前验证的马尔可夫模型。我们构建了一个马尔科夫模型,其中 DAS28 定义的健康状态缓解 (≤2.6)、低 (2.6 < DAS28 ≤ 3.2)、中度 (3.2 < DAS28 ≤ 5.1) 和高疾病活动度 (DAS28 > 5) 之间每 3 个月转换一次. 1) 进行了模拟。如果在每个(3 个月)周期开始时未缓解,则建模患者继续进行后续治疗。在连续两个周期缓解的情况下,药物逐渐减少,直到达到无药物缓解。使用荷兰风湿病监测登记缓解诱导队列 I(升压)和 II(初始组合)的数据估计各个治疗步骤的转换概率。比较了两种策略 5 年后的预期成本、效用和 ICER。为了解释参数的不确定性,通过 Gamma、Normal 和 Dirichlet 分布采用概率敏感性分析。所有的效用、成本和转移概率都被拟合分布所取代。超过5年的时间跨度,初始联合治疗比升压治疗成本更低且更有效。初始联合治疗的 QALY 为 16,226.3 欧元和 3.552 欧元,而升压治疗的 QALY 为 20,183.3 欧元和 3.517 欧元。这导致 ICER 为负值,表明初始联合治疗在获得效用方面成本更低且更有效。这可以通过在所有时间点初始组合策略中较高 (±5%) 的缓解百分比来解释。更多处于缓解期的患者会产生更少的医疗保健和生产力损失成本以及更高的效用。此外,较高的缓解百分比导致初始组合策略中 bDMARD 的使用较少,从而降低了总体成本。在考虑成本效益时,发现初始联合疗法在治疗类风湿性关节炎方面优于升压疗法。
更新日期:2019-11-28
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