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

doi:10.1016/j.jval.2023.03.2304

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 + Show 9 more

https://doi.org/10.1016/j.jval.2023.03.2304

Copy DOI

Journal: Value in Health	Publication Date: Jun 1, 2023
Citations: 3

Affiliation: Sanofi (United States), IQVIA (United States), Sanofi (France), Sanofi (Germany), University of Münster, Erasmus MC

#Late-Onset Pompe Disease #For Forced Vital Capacity + Show 8 more

Abstract
Full-Text PDF
Similar Papers

Abstract

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.

Full Text

Paper version not known

Open DOI Link

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call

Similar Papers

Paper Title

Journal

Date

Author

View more papers

More From: Value in Health

Paper Title

Journal

Date

Author

View more papers

Disclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.