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在奥地利医疗体系中,与仅采用放血疗法相比,聚乙二醇干扰素α-2b治疗低危真性红细胞增多症患者的成本效用分析。

Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system.

作者信息

Walter Evelyn, Torelli Francesca, Barbui Tiziano

机构信息

IPF Institute for Pharmaeconomic Research, Vienna, Austria.

AOP Orphan Pharmaceuticals, Vienna, Austria.

出版信息

Ann Hematol. 2025 Jan;104(1):219-229. doi: 10.1007/s00277-025-06229-w. Epub 2025 Jan 31.

DOI:10.1007/s00277-025-06229-w
PMID:39888353
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11868240/
Abstract

Treatment of polycythemia vera (PV) aims to maintain hematocrit on target to reduce risk of thrombotic complications, while preventing disease progression to myelofibrosis (MF) and acute myeloid leukemia (AML). This analysis evaluated cost-effectiveness of adding ropeginterferon alfa-2b (ropegIFNα) to phlebotomy in patients with low-risk PV (those younger than 60 years without prior thrombosis), compared to phlebotomy alone. We combined a 12-month decision tree with a semi-Markov cohort model comparing ropegIFNα to the standard treatment from the Austrian healthcare system perspective over 30 years. Outcomes were quality adjusted life years (QALYs), costs, and incremental cost-utility ratio (ICUR). Model inputs were obtained from the phase 2 Low-PV study, additional published literature and from Austrian-specific cost databases. One-way and probabilistic sensitivity analyses (SA) assessed the robustness of findings. RopegIFNα led to 1,43 higher QALYs and 50.960 EUR overall higher costs compared to phlebotomy alone, with an ICUR of 35.525 EUR/QALY. Thrombosis, MF, and AML costs decreased for the ropegIFNα group by 12%, 30% and 16% respectively, due to the delayed complications onset and disease progression. In the one-way SA, ropegIFNα costs and discount rates had the greatest impact on results. The probabilistic SA showed a 100% probability of cost-effectiveness at willingness-to-pay threshold aligned to the Austrian GDP per capita. RopegIFNα is a cost-effective treatment option for patients with low-risk PV. These findings suggest that early treatment with ropegIFNα could ensure optimal resource allocation by preventing costly thrombotic events and progression to MF whilst increasing patient quality of life.

摘要

真性红细胞增多症(PV)的治疗旨在将血细胞比容维持在目标水平,以降低血栓形成并发症的风险,同时预防疾病进展为骨髓纤维化(MF)和急性髓系白血病(AML)。本分析评估了在低风险PV患者(年龄小于60岁且无既往血栓形成)中,与单纯放血疗法相比,添加聚乙二醇化干扰素α-2b(ropegIFNα)至放血疗法的成本效益。我们结合了一个12个月的决策树和一个半马尔可夫队列模型,从奥地利医疗保健系统的角度比较了ropegIFNα与标准治疗方案在30年中的情况。结果指标为质量调整生命年(QALYs)、成本和增量成本效用比(ICUR)。模型输入数据来自2期低风险PV研究、其他已发表文献以及奥地利特定的成本数据库。单向和概率敏感性分析(SA)评估了研究结果的稳健性。与单纯放血疗法相比,ropegIFNα导致QALYs提高1.43,总体成本高出50960欧元,ICUR为35525欧元/QALY。由于并发症发作和疾病进展延迟,ropegIFNα组的血栓形成、MF和AML成本分别降低了12%、30%和16%。在单向敏感性分析中,ropegIFNα成本和贴现率对结果影响最大。概率敏感性分析显示,在与奥地利人均国内生产总值一致的支付意愿阈值下,成本效益概率为100%。对于低风险PV患者,ropegIFNα是一种具有成本效益的治疗选择。这些发现表明,早期使用ropegIFNα治疗可通过预防代价高昂的血栓形成事件和疾病进展为MF,同时提高患者生活质量,确保最佳资源分配。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/9c8cf154b28c/277_2025_6229_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/0ddcd43e86d0/277_2025_6229_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/eb47fcfbe0c8/277_2025_6229_Fig2_HTML.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/688aed869b21/277_2025_6229_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/9c8cf154b28c/277_2025_6229_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/0ddcd43e86d0/277_2025_6229_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/eb47fcfbe0c8/277_2025_6229_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/000b3d6c1e92/277_2025_6229_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/688aed869b21/277_2025_6229_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6e1/11868240/9c8cf154b28c/277_2025_6229_Fig5_HTML.jpg

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本文引用的文献

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Contemporary Challenges in Polycythemia Vera Management From the Perspective of Patients and Physicians.从患者和医生的角度看原发性骨髓纤维化治疗的当代挑战。
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Ropeginterferon versus Standard Therapy for Low-Risk Patients with Polycythemia Vera.
罗特西普干扰素与标准疗法治疗低危真性红细胞增多症患者的比较。
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The impact of thrombosis on probabilities of death and disease progression in polycythemia vera: a multistate transition analysis of 1,545 patients.血栓形成对真性红细胞增多症患者死亡和疾病进展概率的影响:1545 例患者的多状态转移分析。
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Event-free survival in patients with polycythemia vera treated with ropeginterferon alfa-2b versus best available treatment.接受罗培戈干扰素α-2b治疗与最佳可用治疗的真性红细胞增多症患者的无事件生存期
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