Cleveland Clinic Taussig Cancer Institute, Cleveland, OH 44106, USA.
PharmaEssentia USA Corporation, Burlington, MA 01803, USA.
J Comp Eff Res. 2023 Sep;12(9):e230066. doi: 10.57264/cer-2023-0066. Epub 2023 Aug 2.
Patients with polycythemia vera (PV), a rare and chronic blood cancer, are at a higher risk for thromboembolic events, progression to myelofibrosis, and leukemic transformation. In 2021, ropeginterferon alfa-2b-njft (BESREMi) was approved in the US to treat adults with PV. The purpose of this study is to estimate the cost-effectiveness of ropeginterferon alfa-2b-njft, used as a first- or second-line treatment, for the treatment of patients with PV in the US. A Markov cohort model was developed from the healthcare system perspective in the United States. Model inputs were informed by the PROUD-PV and CONTINUATION-PV studies and published literature. The model population included both low-risk and high-risk patients with PV. The model compared ropeginterferon alfa-2b-njft used either as first- or second-line versus an alternative treatment pathway of first-line hydroxyurea followed by ruxolitinib. Over the modeled lifetime, ropeginterferon alfa-2b-njft provided an additional 0.4 higher quality-adjusted life years (QALYs) and 0.4 life-years with an added cost of USD60,175, resulting in a cost per QALY of USD141,783. The model was sensitive to treatment costs, the percentage of patients who discontinue hydroxyurea, the percentage of ropeginterferon alfa-2b-njft users who switch to monthly dosing, the percentage of ropeginterferon alfa-2b-njft users as 2nd line treatment, and the treatment response rates. A younger patient age at baseline and a higher percentage of patients with low-risk disease improved the cost-effectiveness of ropeginterferon alfa-2b-njft. Ropeginterferon alfa-2b-njft is a cost-effective treatment option for a broad range of patients with PV, including both low- and high-risk patients and patients with and without prior cytoreductive treatment with hydroxyurea.
真性红细胞增多症（PV）是一种罕见的慢性血液病，患者发生血栓栓塞事件、进展为骨髓纤维化和白血病转化的风险较高。2021 年，罗匹尼罗干扰素 alfa-2b-njft（BESREMi）在美国获批用于治疗 PV 成人患者。本研究旨在评估罗匹尼罗干扰素 alfa-2b-njft 作为一线或二线治疗药物用于治疗美国 PV 患者的成本效果。 该研究从美国医疗保健系统的角度建立了一个 Markov 队列模型。模型输入信息来自 PROUD-PV 和 CONTINUATION-PV 研究以及已发表的文献。模型人群包括低危和高危 PV 患者。模型比较了罗匹尼罗干扰素 alfa-2b-njft 作为一线或二线治疗与一线羟脲后使用鲁索替尼的替代治疗途径。 在模型寿命期内，罗匹尼罗干扰素 alfa-2b-njft 额外提供了 0.4 个更高的质量调整生命年（QALY）和 0.4 个生命年，增加成本为 60175 美元，导致每 QALY 成本为 141783 美元。该模型对治疗成本、停止使用羟脲的患者比例、每月剂量转换的罗匹尼罗干扰素 alfa-2b-njft 用户比例、罗匹尼罗干扰素 alfa-2b-njft 二线治疗的用户比例以及治疗反应率较为敏感。基线时患者年龄较小和低危疾病患者比例较高，提高了罗匹尼罗干扰素 alfa-2b-njft 的成本效果。 罗匹尼罗干扰素 alfa-2b-njft 是一种具有成本效益的治疗选择，适用于广泛的 PV 患者，包括低危和高危患者以及有或没有先前接受羟脲细胞减少治疗的患者。
