Sugar Elizabeth A, Holbrook Janet T, Kempen John H, Burke Alyce E, Drye Lea T, Thorne Jennifer E, Louis Thomas A, Jabs Douglas A, Altaweel Michael M, Frick Kevin D
Department of Biostatistics, The Johns Hopkins University Bloomberg School of Public Health, Baltimore, Maryland; Department Epidemiology, The Johns Hopkins University Bloomberg School of Public Health, Baltimore, Maryland; Center for Clinical Trials, The Johns Hopkins University Bloomberg School of Public Health, Baltimore, Maryland; Division of Biostatistics and Bioinformatics, The Sidney Kimmel Comprehensive Cancer Center, The Johns Hopkins University School of Medicine, Baltimore, Maryland.
Department Epidemiology, The Johns Hopkins University Bloomberg School of Public Health, Baltimore, Maryland; Center for Clinical Trials, The Johns Hopkins University Bloomberg School of Public Health, Baltimore, Maryland.
Ophthalmology. 2014 Oct;121(10):1855-62. doi: 10.1016/j.ophtha.2014.04.022. Epub 2014 Jun 5.
To evaluate the 3-year incremental cost-effectiveness of fluocinolone acetonide implant versus systemic therapy for the treatment of noninfectious intermediate, posterior, and panuveitis.
Randomized, controlled, clinical trial.
Patients with active or recently active intermediate, posterior, or panuveitis enrolled in the Multicenter Uveitis Steroid Treatment Trial.
Data on cost and health utility during 3 years after randomization were evaluated at 6-month intervals. Analyses were stratified by disease laterality at randomization (31 unilateral vs 224 bilateral) because of the large upfront cost of the implant.
The primary outcome was the incremental cost-effectiveness ratio (ICER) over 3 years: the ratio of the difference in cost (in United States dollars) to the difference in quality-adjusted life-years (QALYs). Costs of medications, surgeries, hospitalizations, and regular procedures (e.g., laboratory monitoring for systemic therapy) were included. We computed QALYs as a weighted average of EQ-5D scores over 3 years of follow-up.
The ICER at 3 years was $297,800/QALY for bilateral disease, driven by the high cost of implant therapy (difference implant - systemic [Δ]: $16,900; P < 0.001) and the modest gains in QALYs (Δ = 0.057; P = 0.22). The probability of the ICER being cost-effective at thresholds of $50,000/QALY and $100,000/QALY was 0.003 and 0.04, respectively. The ICER for unilateral disease was more favorable, namely, $41,200/QALY at 3 years, because of a smaller difference in cost between the 2 therapies (Δ = $5300; P = 0.44) and a larger benefit in QALYs with the implant (Δ = 0.130; P = 0.12). The probability of the ICER being cost-effective at thresholds of $50,000/QALY and $100,000/QALY was 0.53 and 0.74, respectively.
Fluocinolone acetonide implant therapy was reasonably cost-effective compared with systemic therapy for individuals with unilateral intermediate, posterior, or panuveitis but not for those with bilateral disease. These results do not apply to the use of implant therapy when systemic therapy has failed or is contraindicated. Should the duration of implant effect prove to be substantially >3 years or should large changes in therapy pricing occur, the cost-effectiveness of implant versus systemic therapy would need to be reevaluated.
评估曲安奈德植入剂与全身治疗相比，治疗非感染性中间葡萄膜炎、后葡萄膜炎和全葡萄膜炎的3年增量成本效果。
随机对照临床试验。
参加多中心葡萄膜炎类固醇治疗试验的活动性或近期活动性中间葡萄膜炎、后葡萄膜炎或全葡萄膜炎患者。
在随机分组后的3年中，每6个月评估一次成本和健康效用数据。由于植入剂前期成本较高，分析按随机分组时的疾病单侧性进行分层（31例单侧 vs 224例双侧）。
主要结局是3年的增量成本效果比（ICER）：成本差异（以美元计）与质量调整生命年（QALY）差异的比值。包括药物、手术、住院和常规检查（如全身治疗的实验室监测）的费用。我们将QALY计算为随访3年期间EQ-5D评分的加权平均值。
双侧疾病3年的ICER为297,800美元/QALY，这是由植入治疗的高成本（植入剂 - 全身治疗的差异[Δ]：16,900美元；P < 0.001）和QALY的适度增加（Δ = 0.057；P = 0.22）驱动的。在50,000美元/QALY和100,000美元/QALY的阈值下，ICER具有成本效益的概率分别为0.003和0.04。单侧疾病的ICER更有利，即3年时为41,200美元/QALY，因为两种治疗之间的成本差异较小（Δ = 5300美元；P = 0.44），且植入剂在QALY方面的获益更大（Δ = 0.130；P = 0.12）。在50,000美元/QALY和100,000美元/QALY的阈值下，ICER具有成本效益的概率分别为0.53和0.74。
对于单侧中间葡萄膜炎、后葡萄膜炎或全葡萄膜炎患者，曲安奈德植入剂治疗与全身治疗相比具有合理的成本效益，但对于双侧疾病患者则不然。这些结果不适用于全身治疗失败或禁忌时植入治疗的使用。如果植入剂效果的持续时间被证明显著>3年，或者治疗价格发生重大变化，则需要重新评估植入剂与全身治疗的成本效益。
