PURPOSE To investigate the cost effectiveness of adalimumab in combination with methotrexate, compared with methotrexate alone, for the management of uveitis associated with juvenile idiopathic arthritis (JIA). DESIGN A cost-utility analysis based on a clinical trial and decision analytic model. PARTICIPANTS Children and adolescents 2 to 18 years of age with persistently active uveitis associated with JIA, despite optimized methotrexate treatment for at least 12 weeks. METHODS The SYCAMORE (Randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis) trial (identifier, ISRCTN10065623) of methotrexate (up to 25 mg weekly) with or without fortnightly administered adalimumab (20 or 40 mg, according to body weight) provided data on resource use (based on patient self-report and electronic records) and health utilities (from the Health Utilities Index questionnaire). Surgical event rates and long-term outcomes were based on data from a 10-year longitudinal cohort. A Markov model was used to extrapolate the effects of treatment based on visual impairment. MAIN OUTCOME MEASURES Medical costs to the National Health Service in the United Kingdom, utility of defined health states, quality-adjusted life-years (QALYs), and incremental cost per QALY. RESULTS Adalimumab in combination with methotrexate resulted in additional costs of £39 316, with a 0.30 QALY gain compared with methotrexate alone, resulting in an incremental cost-effectiveness ratio of £129 025 per QALY gained. The probability of cost effectiveness at a threshold of £30 000 per QALY was less than 1%. Based on a threshold analysis, a price reduction of 84% would be necessary for adalimumab to be cost effective. CONCLUSIONS Adalimumab is clinically effective in uveitis associated with JIA; however, its cost effectiveness is not demonstrated compared with methotrexate alone in the United Kingdom setting.

中文翻译：

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阿达木单抗治疗幼年特发性关节炎相关葡萄膜炎的成本效益分析。

目的 研究与单独使用甲氨蝶呤相比，阿达木单抗联合甲氨蝶呤治疗幼年特发性关节炎 (JIA) 相关葡萄膜炎的成本效益。设计基于临床试验和决策分析模型的成本效用分析。参与者 2 至 18 岁的儿童和青少年，尽管经过至少 12 周的优化甲氨蝶呤治疗，但仍患有与 JIA 相关的持续活动性葡萄膜炎。方法 SYCAMORE（关于阿达木单抗联合 MethOtRExate 治疗幼年特发性关节炎相关葡萄膜炎的临床有效性、安全性和成本效益的随机对照试验）试验（标识符，ISRCTN10065623）甲氨蝶呤（每周最多 25 毫克）有或没有每两周给药一次阿达木单抗（20 或 40 mg，根据体重）提供了关于资源使用（基于患者自我报告和电子记录）和健康公用事业（来自健康公用事业指数问卷）的数据。手术事件发生率和长期结果基于 10 年纵向队列的数据。马尔可夫模型被用来推断基于视力障碍的治疗效果。主要结果测量英国国家卫生服务的医疗费用、定义的健康状态的效用、质量调整生命年 (QALY) 和每个 QALY 的增量成本。结果 与单独使用甲氨蝶呤相比，阿达木单抗联合甲氨蝶呤导致额外成本 39 316 英镑，QALY 增加 0.30，每增加一个 QALY，成本效益比增加 129 025 英镑。每个 QALY 30 000 英镑阈值的成本效益概率小于 1%。根据阈值分析，阿达木单抗需要降价 84% 才能具有成本效益。结论 阿达木单抗对与 JIA 相关的葡萄膜炎临床有效；然而，与英国单独使用甲氨蝶呤相比，其成本效益并未得到证实。