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免疫性血小板减少症患者的疾病管理——德国一项具有代表性的回顾性调查结果。

Disease management of patients with immune thrombocytopenia-results of a representative retrospective survey in Germany.

机构信息

Department of Hematology, Cellular Therapy and Hemostaseology, University Hospital Leipzig, Leipzig, Germany.

Lübecker onkologische Schwerpunktpraxis, Lübeck, Germany.

出版信息

Ann Hematol. 2020 Sep;99(9):2085-2093. doi: 10.1007/s00277-020-04173-5. Epub 2020 Jul 25.

DOI:10.1007/s00277-020-04173-5
PMID:32710167
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7419449/
Abstract

Clinical research has resulted in an improvement of treatment options for patients with immune thrombocytopenia (ITP) over the last years. However, only few data exist on the real-life management of patients with ITP. To expand the knowledge, a multicenter, national survey was undertaken in 26 hematology practices distributed all over Germany. All patients with a diagnosis of ITP were documented using questionnaires, irrespective of the diagnosis date over a period of 2 years. Overall, data of 1023 patients were evaluated with 56% of patients being older than 60 years. Seventy-nine percent of the patients had chronic (> 12 months), 16% persistent (> 3-12 months), and 5% newly diagnosed (0-3 months) ITP. In 61% of cases, the disease lasted 3 or more years before survey documentation started. Main strategies applied as first-line therapy consisted of steroids in 45% and a "watch and wait" approach in 41% of patients. During second- and third-line strategies, treatment with steroids decreased (36% and 28%, respectively), while treatment modalities such as TPO-RAs increased (19% and 26%, respectively). As expected, patients with a low platelet count and thus a higher risk for bleeding and mortality received treatment (esp. steroids) more frequently during first line than those with a higher platelet count. Up to a third of patients were treated with steroids for more than a year. Overall, our study provides a cross-section overview about the current therapeutic treatment landscape in German ITP patients. The results will help to improve therapeutic management of ITP patients.

摘要

近年来,临床研究为免疫性血小板减少症 (ITP) 患者的治疗选择带来了改善。然而,关于 ITP 患者的实际治疗管理,仅有少量数据。为了扩展这方面的知识,我们在德国 26 家血液科诊所进行了一项多中心、全国性的调查。在为期 2 年的时间里,使用问卷对所有确诊为 ITP 的患者进行了记录,无论其诊断日期如何。总体上,评估了 1023 名患者的数据,其中 56%的患者年龄大于 60 岁。79%的患者为慢性 (>12 个月),16%为持续性 (>3-12 个月),5%为新诊断 (0-3 个月) ITP。在开始调查记录之前,61%的病例已经持续了 3 年或更长时间。作为一线治疗的主要策略包括 45%的患者使用类固醇和 41%的患者采用“观察等待”方法。在二线和三线治疗策略中,类固醇的治疗减少(分别为 36%和 28%),而 TPO-RAs 等治疗方法的应用增加(分别为 19%和 26%)。正如预期的那样,血小板计数较低(因此出血和死亡率风险较高)的患者在一线治疗中比血小板计数较高的患者更频繁地接受治疗(尤其是类固醇)。多达三分之一的患者接受类固醇治疗超过一年。总的来说,我们的研究提供了德国 ITP 患者当前治疗状况的横断面概述。这些结果将有助于改善 ITP 患者的治疗管理。

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免疫性血小板减少症中血小板生成素受体激动剂的二线治疗:基于意大利德尔菲法的共识推荐
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Understanding how older age drives decision-making and outcome in Immune Thrombocytopenia. A single centre study on 465 adult patients.
了解年龄如何影响免疫性血小板减少症患者的决策和预后。一项针对 465 例成年患者的单中心研究。
Br J Haematol. 2019 Feb;184(3):424-430. doi: 10.1111/bjh.15668. Epub 2018 Nov 8.
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Eltrombopag versus romiplostim in treatment of adult patients with immune thrombocytopenia: A systematic review incorporating an indirect-comparison meta-analysis.依替巴肽与罗米司亭治疗成人免疫性血小板减少症的疗效比较:一项系统评价及间接比较荟萃分析。
PLoS One. 2018 Jun 1;13(6):e0198504. doi: 10.1371/journal.pone.0198504. eCollection 2018.
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