Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.

ABSTRACT:

Background

ADVANCE (NCT01526785) presented an opportunity to obtain a more nuanced understanding of motor function changes in treatment-experienced children with Pompe disease receiving 4000L-production-scale alglucosidase alfa for 52 weeks.

Objective

To estimate minimal detectable change (MDC) and effect size on Gross Motor Function Measure-88 (GMFM-88) after 52 weeks of 4000L alglucosidase alfa (complete data N =  90).

Methods

The GMFM-88 mean total % score changes, MDC, and effect size were analyzed post hoc by Pompe Motor Function Level at enrollment, age groups at enrollment, and fraction of life on pre-study 160L-production-scale alglucosidase alfa.

Results

Overall, participants aged < 2 years surpassed MDC at Week 52 (change [mean±standard deviation]

SUBMITTER: Duong T 

PROVIDER: S-EPMC9697057 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature