PDF(Pubmed)
Abstract：
Ropeginterferon-alfa2b (ropegIFNα2b) is a long-acting IFN formulation with broad FDA/EMA approval as a therapy of polycythemia vera (PV) with no symptomatic splenomegaly. There is currently lack of information on the real-world patient selection, including the impact of local reimbursement policies, and drug management, particularly: type/timing of screening and follow-up tests; absolute/relative contraindications to therapy; ropegIFNα2b dose and combinations with hydroxyurea. As a sub-analysis of the PV-ARC retrospective study (NCT06134102), we here report our monocenter experience with ropegIFNα2b in the period from January 2021, corresponding to drug availability outside clinical trial, and December 2023. Among the 149 patients with EMA/FDA indication, only 55 (36.9%) met the local reimbursement criteria and 18 (12.1%) received ropegIFNα2b. Thanks to appropriate screening, relative/absolute contraindications to ropegIFNα2b were detected and managed in a multidisciplinary manner. Efficacy and safety of ropegIFNα2b was confirmed, with 3 cases of early molecular response. General use of low ropegIFNα2b dose, with frequent need for hydroxyurea combinations, was noted. This real-world experience suggests a significant impact of local regulations on drug prescription and the need for greater real-world data collection on ropegIFNα2b in PV patients. Also, it describes appropriate multidisciplinary screening and monitoring procedures during ropegIFNα2b therapy.
摘要：
Ropeg干扰素-alfa2b（ropegIFNα2b）是一种长效IFN制剂，已获得FDA/EMA的广泛批准，可用于治疗真性红细胞增多症（PV），无症状性脾肿大。目前缺乏有关真实世界患者选择的信息，包括当地报销政策的影响，和药物管理，特别是：筛查和随访试验的类型/时机；治疗的绝对/相对禁忌症；ropegIFNα2b剂量和与羟基脲的组合。作为PV-ARC回顾性研究的子分析(NCT06134102)，我们在这里报告我们在2021年1月期间使用ropegIFNα2b的单一中心经验，对应于临床试验之外的药物可用性，2023年12月。在149例EMA/FDA适应症患者中，只有55人(36.9%)符合当地报销标准,18人(12.1%)获得ropegIFNα2b.多亏了适当的筛选，对ropegIFNα2b的相对/绝对禁忌症进行了多学科检测和管理.RopegIFNα2b的疗效和安全性得到证实，3例早期分子反应。一般使用低ropegIFNα2b剂量，经常需要羟基脲组合，已注意到。这种现实世界的经验表明，当地法规对药物处方产生了重大影响，并且需要对PV患者的ropegIFNα2b进行更多的现实世界数据收集。此外，它描述了在ropegIFNα2b治疗期间适当的多学科筛查和监测程序。
