Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa-Reference-Cited by-同舟云学术

Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa

Published:2023-12-27 Issue: Volume: Page:1-6
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Boentert Matthias¹²,Campana Emmanuelle Salort³,Attarian Shahram³,Diaz-Manera Jordi⁴,Dimachkie Mazen M.⁵,Periquet Magali⁶,Thibault Nathan⁷,Miossec Patrick⁸,Zhou Tianyue⁷,Berger Kenneth I.⁹

Affiliation:

1. Department of Neurology and Institute of Translational Neurology, Münster University Hospital, Münster, Germany

2. Department of Medicine, UKM-Marienhospital Steinfurt, Steinfurt, Germany

3. Referral Centre for Neuromuscular Diseases and ALS, Hôpital La Timone, Marseille, France

4. Newcastle University Centre for Life, Newcastleupon Tyne, UK

5. University of Kansas Medical Center, Department of Neurology, Kansas City, KS, USA

6. Sanofi, Berlin, Germany

7. Sanofi, Cambridge, MA, USA

8. Sanofi, Paris, France

9. NYU Grossman School of Medicine, New York, NY, USA

Abstract

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).

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

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