COMET 试验中对强制生命容量的事后非参数分析表明,Avalglucosidase Alfa 与 Alglucosidase Alfa 相比更具优势。

IF 4.3 3区 材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC
Matthias Boentert, Emmanuelle Salort Campana, Shahram Attarian, Jordi Diaz-Manera, Mazen M Dimachkie, Magali Periquet, Nathan Thibault, Patrick Miossec, Tianyue Zhou, Kenneth I Berger
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引用次数: 0

摘要

在对晚发型庞贝病患者进行的 COMET 试验中,观察到阿瓦糖苷酶α(AVA)与阿糖苷酶α(ALGLU)相比,直立用力肺活量(FVC)预测百分比有更大的改善(估计治疗差异:2.43%)。预先指定的混合模型重复测量(MMRM)分析表明,AVA 不具有劣效性(P = 0.0074),并有微弱的优越性(P = 0.063;95% CI:-0.13-4.99)。我们在两项事后分析中报告了 AVA 的优越性,这些分析考虑到了基线时 FVC 低且患有严重慢性阻塞性肺病的极端离群者:MMRM 剔除了异常值(P = 0.013),并对所有数据进行了非参数分析和重复测量协方差分析(P = 0.019)。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa.

In the COMET trial of patients with late-onset Pompe disease, greater improvement in upright forced vital capacity (FVC) % predicted was observed with avalglucosidase alfa (AVA) vs alglucosidase alfa (ALGLU) (estimated treatment difference: 2.43%). The pre-specified mixed model repeated measures (MMRM) analysis demonstrated non-inferiority of AVA (P = 0.0074) and narrowly missed superiority (P = 0.063; 95% CI: -0.13-4.99). We report superiority of AVA in two post-hoc analyses that account for an extreme outlier participant with low FVC and severe chronic obstructive pulmonary disease at baseline: MMRM excluding the outlier (P = 0.013) and non-parametric analysis of all data with repeated measures analysis of covariance (P = 0.019).

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CiteScore
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