Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease.

ABSTRACT:

Background

The minimal clinically important difference (MCID) is the smallest change in outcome that physicians or patients would consider meaningful and is relevant when evaluating disease progression or the efficacy of interventions. Studies of patients with late-onset Pompe disease (LOPD) have used the 6-min walk distance (6MWD) as an endpoint to assess motor function. However, an MCID for 6MWD (% predicted and meters) has yet to be established in LOPD. The objective of the study was to derive 6MWD MCID (% predicted and meters) with different analysis methods and for subgroups of different disease severity for LOPD.

Methods

Data from the PROPEL trial were used to calculate 6MWD MCID in the overall PROPEL population and subgroups of baseline severity as assessed by walking

SUBMITTER: Claeys KG 

PROVIDER: S-EPMC11008008 | biostudies-literature | 2024 Apr

REPOSITORIES: biostudies-literature