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在常规临床实践中用罗米司亭治疗原发免疫性血小板减少症(ITP):来自英国 ITP 注册研究的回顾性研究。

Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry.

机构信息

Barts and The London School of Medicine and Dentistry, London, UK.

Amgen Limited, London, UK.

出版信息

Eur J Haematol. 2019 May;102(5):416-423. doi: 10.1111/ejh.13221. Epub 2019 Mar 7.

Abstract

BACKGROUND

Romiplostim is a thrombopoietin-mimetic peptibody for adult refractory chronic immune thrombocytopenia (ITP). We aimed to describe ITP patients receiving romiplostim, platelet counts and romiplostim usage in UK clinical practice.

METHODS

This was a retrospective cohort study of patients in the UKITP Registry who received romiplostim between October 2009 and January 2015, including data up to 6 months before romiplostim initiation through follow-up.

RESULTS

Of 1440 patients in the UKITP Registry, 118 adults with primary ITP were eligible. Before romiplostim, 22% had splenectomy, 12% received platelet transfusion, 97% received ≥ 1 different ITP medication and 77% received ≥ 3. Most patients (73%) initiated romiplostim ≥ 1 year after ITP diagnosis (chronic phase). The mean duration of romiplostim treatment was 5.7 (SE 0.9) months, and the median was 1.4 months (IQR: 0.2, 6.5). Mean platelet count before romiplostim was 38 × 10 /L, rising to 103 × 10 /L within 1 month, and remaining 50-150 × 10 /L through up to 3 years of follow-up. After romiplostim, 4% of patients had splenectomy, 6% received platelet transfusion, and 57% received just one ITP medication other than romiplostim.

CONCLUSION

The study provides valuable insights into the real-world use of romiplostim in primary ITP in routine practice and highlighted the timing of romiplostim initiation at different ITP disease phases.

摘要

背景

罗米司亭是一种用于治疗成人难治性慢性免疫性血小板减少症(ITP)的血小板生成素模拟肽结合抗体。我们旨在描述在英国临床实践中接受罗米司亭治疗的 ITP 患者、血小板计数和罗米司亭的使用情况。

方法

这是一项回顾性队列研究,纳入了 UKITP 登记处中自 2009 年 10 月至 2015 年 1 月期间接受罗米司亭治疗的患者,包括罗米司亭治疗开始前 6 个月至随访期间的数据。

结果

在 UKITP 登记处的 1440 名患者中,符合条件的 118 名原发性 ITP 成年患者。在接受罗米司亭治疗之前,22%的患者接受过脾切除术,12%的患者接受过血小板输注,97%的患者接受过≥1 种不同的 ITP 药物治疗,77%的患者接受过≥3 种。大多数患者(73%)在 ITP 诊断后≥1 年开始接受罗米司亭治疗(慢性期)。罗米司亭治疗的平均持续时间为 5.7(SE 0.9)个月,中位数为 1.4 个月(IQR:0.2,6.5)。接受罗米司亭治疗前的平均血小板计数为 38×10 /L,治疗后 1 个月内升至 103×10 /L,在 3 年的随访期间,血小板计数保持在 50-150×10 /L。接受罗米司亭治疗后,4%的患者接受了脾切除术,6%的患者接受了血小板输注,57%的患者除了罗米司亭外,仅接受了一种其他 ITP 药物治疗。

结论

该研究提供了有关罗米司亭在常规实践中治疗原发性 ITP 的真实世界使用的有价值的见解,并强调了在不同 ITP 疾病阶段开始使用罗米司亭的时机。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f93c/6850028/32c11fd41d13/EJH-102-416-g001.jpg

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