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真实世界中 cladribine 片剂治疗美国复发性多发性硬化症患者的模式和疗效。

Real-world treatment patterns and effectiveness of cladribine tablets in patients with relapsing forms of multiple sclerosis in the United States.

机构信息

Analysis Group, Boston, MA, USA.

Analysis Group, Los Angeles, CA, USA.

出版信息

Mult Scler Relat Disord. 2023 Nov;79:105052. doi: 10.1016/j.msard.2023.105052. Epub 2023 Oct 6.

Abstract

BACKGROUND

Real-world evidence on the use of cladribine tablets (CladT) for relapsing forms of multiple sclerosis (RMS) in the United States is emerging. The objective of this study was to assess the real-world treatment patterns and effectiveness of CladT in RMS.

METHODS

Adults with RMS initiating CladT were selected from the Symphony Integrated Dataverse. Baseline and follow-up periods were the 12 months before and 24 months after CladT initiation (index date). Switching to another disease-modifying therapy (DMT) and number of CladT courses were described during follow-up. Annualized relapse rate (ARR), MS disease severity, Expanded Disability Status Scale-Derived Disability Indicators (EDSS-DDI), corticosteroid use, and healthcare resource utilization (HRU) were described during Years 1 and 2 of follow-up and compared with baseline.

RESULTS

A total of 539 CladT-treated patients were included (mean age: 49.9 years; 77.6 % female). Over the 2-year follow-up, 91 % and 59 % of patients had one and two CladT courses, respectively, and 7 % of patients had evidence of switching to another DMT. ARR, MS disease severity score, and corticosteroid use decreased significantly during follow-up compared with baseline, while EDSS-DDI remained stable. All-cause and MS-related HRU decreased during follow-up.

CONCLUSION

CladT-treated patients with RMS had low switch rates, reduced ARR, disease severity, corticosteroid use, and HRU.

摘要

背景

关于克拉屈滨片剂(CladT)在美国用于治疗多发性硬化症(RMS)复发型的真实世界证据正在出现。本研究的目的是评估 RMS 患者使用克拉屈滨的真实世界治疗模式和效果。

方法

从 Symphony Integrated Dataverse 中选择开始使用克拉屈滨的 RMS 成年患者。基线和随访期分别为克拉屈滨起始前的 12 个月和起始后的 24 个月(索引日期)。在随访期间描述了转换为另一种疾病修正疗法(DMT)和克拉屈滨疗程的数量。在随访的第 1 年和第 2 年描述了每年复发率(ARR)、多发性硬化症疾病严重程度、扩展残疾状况量表残疾指标(EDSS-DDI)、皮质类固醇的使用和医疗保健资源利用(HRU),并与基线进行了比较。

结果

共纳入 539 例接受克拉屈滨治疗的患者(平均年龄:49.9 岁;77.6%为女性)。在 2 年的随访中,91%和 59%的患者分别接受了 1 个和 2 个克拉屈滨疗程,7%的患者有转换为另一种 DMT 的证据。与基线相比,ARR、多发性硬化症严重程度评分和皮质类固醇的使用在随访期间显著降低,而 EDSS-DDI 保持稳定。在随访期间,所有原因和与 MS 相关的 HRU 均减少。

结论

接受克拉屈滨治疗的 RMS 患者转换率低,ARR、疾病严重程度、皮质类固醇使用和 HRU 降低。

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