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生物制剂治疗对疾病修饰抗风湿药物反应不佳的多关节病程幼年特发性关节炎患者的成本效益分析。

Cost-effectiveness of biologics in polyarticular-course juvenile idiopathic arthritis patients unresponsive to disease-modifying antirheumatic drugs.

机构信息

The Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.

出版信息

Arthritis Care Res (Hoboken). 2011 Jan;63(1):111-9. doi: 10.1002/acr.20337.

Abstract

OBJECTIVE

Juvenile idiopathic arthritis (JIA) is the most common chronic pediatric rheumatic disease and can have long-term effects leading to disability in adulthood. Biologics are a new class of drugs increasingly used to treat JIA. The primary study objective was to determine the incremental costs of biologics per additional responder compared to conventional treatment (methotrexate).

METHODS

A separate decision model was created for etanercept, infliximab, adalimumab, and abatacept. The study population consisted of polyarticular-course JIA patients with a prior inadequate response or intolerance to disease-modifying antirheumatic drugs (DMARDs). The effectiveness measure was the proportion of patients who had a treatment response at 1 year according to the American College of Rheumatology (ACR) Pediatric 30 (Pedi 30) improvement criteria. Direct and indirect costs were calculated in 2008 Canadian dollars. Incremental cost-effectiveness ratios and 95% confidence intervals (95% CIs) were calculated for each biologic agent using probabilistic sensitivity analyses.

RESULTS

The additional costs per additional ACR Pedi 30 responder at 1 year were $26,061 (95% CI $17,070, $41,834), $46,711 (95% CI $30,042, $75,787), $16,204 (95% CI $11,393, $22,608), and $31,209 (95% CI $16,659, $66,220) for etanercept, adalimumab, abatacept, and infliximab, respectively.

CONCLUSION

Biologics are more effective than methotrexate in achieving a short-term response in JIA patients with prior inadequate responses to DMARDs; however, this comes at a high annual cost. Adequate long-term data with respect to both safety and effectiveness are not currently available, nor are utility estimates. Such data will be important to estimate value for money for treating JIA with biologic drugs over the long term.

摘要

目的

幼年特发性关节炎(JIA)是最常见的慢性儿科风湿性疾病,可导致成年后残疾等长期影响。生物制剂是一种越来越多地用于治疗 JIA 的新药。主要研究目的是确定与传统治疗(甲氨蝶呤)相比,每种生物制剂每增加一个应答者的增量成本。

方法

为依那西普、英夫利昔单抗、阿达木单抗和阿巴西普分别建立了单独的决策模型。研究人群包括多关节型 JIA 患者,这些患者先前对疾病修饰抗风湿药物(DMARDs)治疗反应不足或不耐受。疗效测量标准是根据美国风湿病学会(ACR)儿科 30 项(Pedi 30)改善标准,1 年内有治疗反应的患者比例。以 2008 年加拿大元计算直接和间接成本。使用概率敏感性分析计算每个生物制剂的增量成本-效果比和 95%置信区间(95%CI)。

结果

1 年内每增加一个 ACR Pedi 30 应答者的额外成本分别为依那西普 26061 加元(95%CI 17070 加元,41834 加元)、英夫利昔单抗 46711 加元(95%CI 30042 加元,75787 加元)、阿达木单抗 16204 加元(95%CI 11393 加元,22608 加元)和阿巴西普 31209 加元(95%CI 16659 加元,66220 加元)。

结论

与甲氨蝶呤相比,生物制剂在治疗对 DMARDs 治疗反应不足的 JIA 患者时更能在短期内取得疗效,但成本较高。目前尚无关于安全性和有效性的充分长期数据,也没有效用估计。此类数据对于长期使用生物药物治疗 JIA 的成本效益具有重要意义。

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