Walter Evelyn, Torelli Francesca, Barbui Tiziano
IPF Institute for Pharmaeconomic Research, Vienna, Austria.
AOP Orphan Pharmaceuticals, Vienna, Austria.
Ann Hematol. 2025 Jan;104(1):219-229. doi: 10.1007/s00277-025-06229-w. Epub 2025 Jan 31.
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.
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。