PLATON: use of romiplostim to treat chronic primary immune thrombocytopenia An observational, nonintervention study of real-life practice
PLATON: use of romiplostim to treat chronic primary immune thrombocytopenia An observational, nonintervention study of real-life practice
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DOI：
10.1007/s12254-020-00580-6
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发表时间：
2020-02-25
影响因子：
0.6
通讯作者：
Cernelc, Peter
中科院分区：
文献类型：
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作者：
Mihaylov, Georgi;Skopec, Barbara;Cernelc, Peter
慢性原发性免疫性血小板减少症（ITP）是一种自身免疫性疾病，涉及血小板抗体的形成，导致血小板破坏增加和慢性血小板减少症。此外，血小板生成受损是由于血小板生成素相对缺乏。罗米司亭，一种血小板生成素受体激动剂，在随机对照试验和真实世界观察性研究中使受影响患者的血小板计数正常化。本研究收集了2010年12月至2017年7月期间中欧和东欧（即斯洛伐克、斯洛文尼亚、保加利亚、俄罗斯和捷克共和国）的真实实践数据。这是一项在romiplostim获批适应症范围内开展的双向观察性、非干预性队列研究。分析了100名患者。在开始使用romiplostim之前，98%的患者接受过其他ITP药物治疗，在之前的6个月内，40%的患者发生过出血事件。在ITP诊断后1.92年（中位数）开始使用罗米司亭。中位平均研究剂量为2.62 μ g/kg/周。在romiplostim治疗期间，血小板计数迅速正常化至>50 × 10（9）/L，20%的患者发生出血事件（无3/4级），13%需要脾切除术。在研究结束时，25%的患者处于缓解状态。1例患者发生严重药物不良反应（血栓形成、吞咽困难），无致死性。总之，romiplostim给药、有效性和安全性似乎与临床试验和类似设计的观察性研究中的观察结果相当。
Chronic primary immune thrombocytopenia (ITP) is an autoimmune disease involving the formation of antibodies to thrombocytes, leading to increased platelet destruction and chronic thrombocytopenia. Additionally, impaired platelet production is due to relative thrombopoietin deficiency. Romiplostim, a thrombopoietin receptor agonist, normalized platelet counts in affected patients in randomized controlled trials and real-world observational studies. The present study collected real-world practice data from Central and Eastern Europe, i.e. Slovakia, Slovenia, Bulgaria, Russia, and Czech Republic, between December 2010 and July 2017. This was an ambidirectional observational, noninterventional cohort study within the approved romiplostim indication. One-hundred patients were analyzed. Prior to romiplostim start, 98% had received other ITP medications and, in the prior 6 months, 40% had experienced bleeding events. Romiplostim was started 1.92 years (median) after ITP diagnosis. The median mean on-study dose was 2.62 & x202f;mu g/kg/week. During romiplostim treatment, platelet counts rapidly normalized to >50 & x202f;x 10(9)/L, 20% of patients experienced bleeding events (none grade 3/4), and 13% required splenectomy. At the end of study, 25% of patients were in remission. One patient experienced serious adverse drug reactions (thrombosis, dysphagia), none were fatal. In conclusion, romiplostim dosing, effectiveness and safety in these unselected ITP patients seemed comparable with observations in clinical trials and similarly designed observational studies.