Abstract：
BACKGROUND: Hydroxyurea (HU) is a commonly used first-line treatment in patients with polycythemia vera (PV). However, approximately 15%-24% of PV patients report intolerance and resistance to HU.
METHODS: This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients.
RESULTS: In the 350 enrolled patients, 70% were >60 years old. Most patients (59.4%) had received ≥1 phlebotomy in the 12 months prior to the first dose of ruxolitinib. Overall, 68.2% of patients achieved hematocrit control with 92.3% patients having hematocrit <45% and 35.4% achieved hematologic remission at month 24. 85.1% of patients had no phlebotomies during the study. Treatment-related adverse events were reported in 54.3% of patients and the most common event was anemia (22.6%). Of the 10 reported deaths, two were suspected to be study drug-related.
CONCLUSIONS: This study demonstrates that ruxolitinib treatment in PV maintains durable hematocrit control with a decrease in the number of phlebotomies in the majority of patients and was generally well tolerated.
METHODS: This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients.
RESULTS: In the 350 enrolled patients, 70% were >60 years old. Most patients (59.4%) had received ≥1 phlebotomy in the 12 months prior to the first dose of ruxolitinib. Overall, 68.2% of patients achieved hematocrit control with 92.3% patients having hematocrit <45% and 35.4% achieved hematologic remission at month 24. 85.1% of patients had no phlebotomies during the study. Treatment-related adverse events were reported in 54.3% of patients and the most common event was anemia (22.6%). Of the 10 reported deaths, two were suspected to be study drug-related.
CONCLUSIONS: This study demonstrates that ruxolitinib treatment in PV maintains durable hematocrit control with a decrease in the number of phlebotomies in the majority of patients and was generally well tolerated.
摘要：
背景：羟基脲（HU）是真性红细胞增多症（PV）患者常用的一线治疗方法。然而,约15%-24%的PV患者报告对HU不耐受和耐药。
方法：第四阶段，欧洲,真实世界,观察性研究评估了鲁索利替尼在对HU耐药和/或不耐受的PV患者中的疗效和安全性，24个月的随访。主要目的是描述PV患者的概况和疾病负担。
结果：在350名入选患者中，70%为60岁以上。大多数患者（59.4％）在首次服用鲁索替尼之前的12个月内接受了≥1次静脉切开术。总的来说,68.2％的患者实现了血细胞比容控制，92.3％的患者血细胞比容<45％，35.4％的患者在第24个月时实现了血液学缓解。85.1%的患者在研究期间没有静脉造血。54.3%的患者报告了治疗相关的不良事件，最常见的事件是贫血（22.6%）。在报告的10例死亡中，其中两人被怀疑与研究药物有关.
结论：这项研究表明，鲁索利替尼治疗肺静脉肺静脉维持了持久的血细胞比容控制，大多数患者的静脉切除术数量减少，并且总体上耐受性良好。
方法：第四阶段，欧洲,真实世界,观察性研究评估了鲁索利替尼在对HU耐药和/或不耐受的PV患者中的疗效和安全性，24个月的随访。主要目的是描述PV患者的概况和疾病负担。
结果：在350名入选患者中，70%为60岁以上。大多数患者（59.4％）在首次服用鲁索替尼之前的12个月内接受了≥1次静脉切开术。总的来说,68.2％的患者实现了血细胞比容控制，92.3％的患者血细胞比容<45％，35.4％的患者在第24个月时实现了血液学缓解。85.1%的患者在研究期间没有静脉造血。54.3%的患者报告了治疗相关的不良事件，最常见的事件是贫血（22.6%）。在报告的10例死亡中，其中两人被怀疑与研究药物有关.
结论：这项研究表明，鲁索利替尼治疗肺静脉肺静脉维持了持久的血细胞比容控制，大多数患者的静脉切除术数量减少，并且总体上耐受性良好。
