背景:短肠综合征伴肠衰竭(SBS-IF)是一种罕见但具有破坏性的医学疾病。肠长度的绝对损失迫使患者进入肠胃外支持(PS)依赖性和各种医疗后遗症,导致发病率和死亡率增加。跨学科治疗可能包括有效的治疗,但是昂贵的肠道营养肽teduglutide。
目的:开发了一个时间离散的马尔可夫模型来模拟治疗效果(生命周期成本,在SBS-IF患者中,teduglutide加最佳支持治疗(BSC)的质量调整生命年(QALYs)和生命年(LYs))与单独的BSC相比。
方法:模型的健康状态是围绕PS的天数构建的。使用来自三个数据集的临床数据:(1)奥地利观察性研究(基本病例)(2)汇总观察性队列研究和(3)前瞻性STEPS研究。直接和间接成本来自公布的来源。QALYs,LYs和成本进行了折扣(每年3%)。
结果:在基本情况下,假设teduglutede与每个患者的2,296,311€和10.78QALYs(13.74LYs)的生命周期相关。没有teduglutide与1,236,816€和2.24QALYs(8.57LYs)相关。增量成本效用比(ICUR)为123,945欧元。在合并的临床数据集的情况下,ICUR增加到184,961欧元。如果使用基于STEPS研究的临床数据,ICUR增加到235,612欧元。
结论:Teduglutide治疗SBS-IF患者符合欧洲社会的传统成本效益标准。然而,不同水平的teduglutide功效给计算留下了一定程度的不确定性。
Short bowel syndrome with intestinal failure (SBS-IF) is a rare but devastating medical condition. An absolute loss of bowel length forces the patients into parenteral support dependency and a variety of medical sequelae, resulting in increased morbidity and mortality. Interdisciplinary treatment may include therapy with the effective but expensive intestinotrophic peptide teduglutide.
A time-discrete Markov model was developed to simulate the treatment effect [lifetime costs, quality-adjusted life years (QALYs), and life years (LYs)] of teduglutide plus best supportive care compared with best supportive care alone in patients with SBS-IF.
The health status of the model was structured around the number of days on PS. Clinical data from 3 data sets were used: 1) an Austrian observational study (base case), 2) pooled observational cohort studies, and 3) a prospective study of teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects. Direct and indirect costs were derived from published sources. QALYs, LYs, and costs were discounted (3% per annum).
Under the base case assumption,
teduglutide is associated with costs of 2,296,311 € per patient and 10.78 QALYs (13.74 LYs) over a lifetime horizon. No
teduglutide is associated with 1,236,816 € and 2.24 QALYs (8.57 LYs). The incremental cost-utility ratio (ICUR) amounts to 123,945 €. In case of the pooled clinical data set, the ICUR increases to 184,961 €. If clinical data based on the study of
teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects were used, the ICUR increased to 235,612 €.
Teduglutide in treating patients with SBS-IF meets the traditional cost-effectiveness criteria from a European societal perspective. Nevertheless, the varying concentrations of
teduglutide efficacy leave a degree of uncertainty in the calculations.