目的:目前针对原发性血小板增多症(ET)患者的指南根据血栓危险分层模型推荐不同的治疗方法。然而,这些建议可能不适用于某些真实临床环境下的患者.因此,我们进行了一项回顾性真实世界验证研究.
方法:比较接受不同治疗方法的初始ET患者的无血栓生存(TFS)。ET患者通过三种代表性风险模型进行分层,传统的,ET血栓形成的国际预后评分(IPSET-血栓形成),并修改了IPSET血栓形成。治疗决定主要由个别医生做出,考虑到患者的偏好和背景。
结果:共纳入179例ET患者,在26例患者中观察到血栓事件。与单独的CRT相比,接受细胞减灭术(CRT)和抗血小板治疗(APT)组合的所有风险模型的高风险患者的TFS明显更长。在通过IPSET血栓形成分层的中危患者中也看到了类似的结果。相比之下,在所有风险模型的极低和低风险患者中,TFS不受添加CRT的影响,表明单独观察或APT是这些患者的适当治疗方法。
结论:我们证明,目前的指南为现实世界临床环境下的日本ET患者提供了最佳的治疗方法。
OBJECTIVE: Current
guidelines for essential thrombocythemia (ET) patients recommend different treatment approaches based on thrombosis risk stratification models. However, these recommendations may not be applicable to some patients under real clinical settings. Therefore, we carried out a retrospective real-world validation study.
METHODS: Thrombosis-free survival (TFS) was compared between treatment naïve ET patients receiving different treatment approaches. ET patients were stratified by three representative risk models, the conventional, the International Prognostic Score for thrombosis in ET (IPSET-thrombosis), and revised IPSET-thrombosis. Treatment decisions were largely made by individual physicians, taking into account patient preferences and backgrounds.
RESULTS: A total of 179 ET patients were included, and thrombotic events were observed in 26 patients. TFS was significantly longer in high-risk patients of all risk models receiving a combination of cytoreductive therapy (CRT) and antiplatelet therapy (APT) compared to CRT alone. Similar results were seen in intermediate-risk patients stratified by IPSET-thrombosis. In contrast, in very low- and low-risk patients of all risk models, TFS was not affected by addition of CRT, indicating that observation or APT alone is an appropriate treatment approach for these patients.
CONCLUSIONS: We demonstrate that current
guidelines provide optimal treatment approaches for Japanese ET patients under real-world clinical settings.