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血小板生成素受体激动剂在复发/难治性免疫性血小板减少症中的比较利用和疗效。

Comparative Utilization and Efficacy of Thrombopoietin Receptor Agonists in Relapsed/Refractory Immune Thrombocytopenia.

机构信息

Atrium Health, Specialty Pharmacy Service, Charlotte, NC; and.

Department of Pharmacy, Atrium Health, Levine Cancer Institute, Concord, NC.

出版信息

Am J Ther. 2021 Jan 8;28(5):e525-e530. doi: 10.1097/MJT.0000000000001335.

Abstract

BACKGROUND

The thrombopoietin (TPO) agonists, eltrombopag and romiplostim, stimulate the production of platelets and offer an effective treatment option in relapsed/refractory immune thrombocytopenia (ITP). Recently published 2019 ITP guidelines recommend the TPO agonists as second-line therapy following corticosteroids; however, little data offer insights into comparative efficacy and tolerability.

STUDY QUESTION

Is there a difference in the efficacy between romiplostim and eltrombopag in relapsed/refractory ITP?

STUDY DESIGN

We conducted a single-center, retrospective chart review of patients with ITP treated with romiplostim or eltrombopag.

MEASURES AND OUTCOMES

The primary objective was a sustained platelet response, defined as platelets greater than 50,000/μL in more than 66% of clinic visits over a 6-month period. Secondary objectives sought to evaluate response to and tolerability of TPO agonists.

RESULTS

The study included 107 consecutive patients, 67 (63%) on romiplostim and 40 (37%) on eltrombopag. Previous corticosteroids and rituximab were used in 95% and 50% of patients, respectively. There was no difference identified in platelet responses between the TPO-RAs, 72% romiplostim versus 65% eltrombopag (P = 0.520). In addition, no differences were identified in secondary measures of response.

CONCLUSIONS

In our experience with romiplostim and eltrombopag for ITP, we did not identify a difference in the efficacy of these agents. Further larger and prospective evaluations should be considered.

摘要

背景

血小板生成素(TPO)激动剂艾曲泊帕和罗米司亭可刺激血小板生成,为复发/难治性免疫性血小板减少症(ITP)提供了有效的治疗选择。最近发布的 2019 年 ITP 指南建议将 TPO 激动剂作为皮质类固醇治疗后的二线治疗方法;然而,很少有数据提供关于疗效和耐受性的比较。

研究问题

罗米司亭和艾曲泊帕在复发/难治性 ITP 中的疗效是否存在差异?

研究设计

我们对接受罗米司亭或艾曲泊帕治疗的 ITP 患者进行了一项单中心、回顾性图表审查。

测量和结果

主要目标是持续的血小板反应,定义为在 6 个月的时间内,超过 66%的就诊时血小板大于 50,000/μL。次要目标旨在评估 TPO 激动剂的反应和耐受性。

结果

该研究纳入了 107 例连续患者,67 例(63%)接受罗米司亭治疗,40 例(37%)接受艾曲泊帕治疗。分别有 95%和 50%的患者之前使用过皮质类固醇和利妥昔单抗。TPO-RAs 之间的血小板反应没有差异,罗米司亭为 72%,艾曲泊帕为 65%(P=0.520)。此外,在次要反应测量中也未发现差异。

结论

在我们使用罗米司亭和艾曲泊帕治疗 ITP 的经验中,我们没有发现这些药物的疗效存在差异。应进一步考虑更大规模和前瞻性的评估。

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