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阿伐曲泊帕治疗慢性肝病患者血小板减少症的成本效益

Cost- Effectiveness of Avatrombopag for the Treatment of Thrombocytopenia in Patients with Chronic Liver Disease.

作者信息

Mladsi Deirdre, Barnett Christine, Aggarwal Kavita, Vredenburg Michael, Dieterich Douglas, Kim Ray

机构信息

RTI Health Solutions Research, Triangle Park, NC, USA.

Dova Pharmaceuticals, Durham, NC, USA.

出版信息

Clinicoecon Outcomes Res. 2020 Sep 14;12:515-526. doi: 10.2147/CEOR.S262772. eCollection 2020.

Abstract

BACKGROUND AND AIM

Thrombocytopenia is common in people with chronic liver disease, who frequently undergo invasive procedures. To minimize the risk of bleeding, prophylactic platelet transfusions have traditionally been used but carry many risks. The aim of this study was to evaluate the cost-effectiveness of avatrombopag compared with platelet transfusion and lusutrombopag as a treatment for thrombocytopenia in adult patients with chronic liver disease scheduled to undergo a medical procedure.

METHODS

A decision-tree model was developed from a US payer perspective to capture acute events observed in phase 3 global randomized controlled clinical trials and, to support exploratory analyses, potential longer-term complications resulting from a major bleed or thromboembolic event. Treatment costs were taken from publicly available data sources. The interventions were evaluated in the overall trial populations and in subpopulations with higher and lower baseline platelet counts. Results were presented as incremental cost per platelet transfusion avoided. One-way and probabilistic sensitivity analyses were conducted.

RESULTS

In the overall population, avatrombopag reduced the need for platelet transfusions and produced cost-savings compared with platelet transfusion (80% fewer prophylactic platelet transfusions, $4250 lower costs) and lusutrombopag (42% fewer prophylactic platelet transfusions, $5819 lower costs). Similar results were seen in both the higher and lower platelet count subpopulations. The one-way and probabilistic sensitivity analyses found that the use of avatrombopag is cost-saving with the incremental cost-effectiveness ratio in quadrant IV (decreased costs, prophylactic platelet transfusions avoided).

CONCLUSION

The use of avatrombopag is expected to be cost-saving while reducing the need for prophylactic platelet transfusions compared with platelet transfusion and lusutrombopag.

摘要

背景与目的

血小板减少症在慢性肝病患者中很常见,这些患者经常接受侵入性操作。为了将出血风险降至最低,传统上一直使用预防性血小板输注,但存在诸多风险。本研究的目的是评估阿伐曲泊帕与血小板输注及芦曲泊帕相比,作为计划接受医疗程序的成年慢性肝病患者血小板减少症治疗方法的成本效益。

方法

从美国医保支付方的角度开发了一个决策树模型,以捕捉在3期全球随机对照临床试验中观察到的急性事件,并为探索性分析提供支持,即由大出血或血栓栓塞事件导致的潜在长期并发症。治疗成本取自公开可用的数据源。在整个试验人群以及基线血小板计数较高和较低的亚组中对干预措施进行评估。结果以避免每次血小板输注的增量成本表示。进行了单因素和概率敏感性分析。

结果

在总体人群中,与血小板输注(预防性血小板输注减少80%,成本降低4250美元)和芦曲泊帕(预防性血小板输注减少42%,成本降低5819美元)相比,阿伐曲泊帕减少了血小板输注的需求并节省了成本。在血小板计数较高和较低的亚组中均观察到类似结果。单因素和概率敏感性分析发现,使用阿伐曲泊帕具有成本效益,增量成本效益比处于第四象限(成本降低,避免预防性血小板输注)。

结论

与血小板输注和芦曲泊帕相比,使用阿伐曲泊帕预计可节省成本,同时减少预防性血小板输注的需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5540/7500829/05820c2f186f/CEOR-12-515-g0001.jpg

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