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 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2024-01-01 DOI:10.3233/JND-230175

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）。

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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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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.