CO168 Defining Clinically Meaningful Thresholds (CMT) for Forced Vital Capacity (FVC) and Six-Minute Walk Test (6MWT) in Patients with Late-Onset Pompe Disease (LOPD)

K.I. Berger,C. Ivanescu,J. Msihid, M. Periquet,A. Hamed,K. An Haack,T. Zhou,M. Boentert, M.E. Kruijshaar,N.A.M.E. van der Beek, L. Pollissard

Value in Health（2023）

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摘要

Interpretation of clinical trial results is limited without a defined CMT for within-patient change or between-group differences of study endpoints. Pooled data (n =100) from COMET trial (NCT02782741) comparing avalglucosidase alfa (AVA) vs. alglucosidase alfa (ALG) in LOPD was used to estimate within-patient and between-group CMTs for FVC (%predicted) and 6MWT.

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关键词

clinically meaningful thresholds,forced vital capacity,cmt,six-minute,late-onset

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