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Objectives

Timed walking tests in clinical studies of certain rare muscular disorders are important efficacy measures appraised in health technology assessments (HTA). We explored whether the six-minute walk distance (6MWD) was associated with various patient-reported outcomes (PROs), including quality of life, in Pompe Disease (PD)–a rare muscular disorder.

Methods

Linear regression between 6MWD and PROs with and without adjusting for baseline covariates was performed using patient-level data from the PROPEL trial (n= 123 adult late-onset PD patients), examining 6MWD change from baseline (CFBL) to Week 52, and at intermediate visits. PROs included EuroQoL-5 dimensions-5 levels (EQ-5D-5L) index value, EQ-5D-5L visual analogue scale (VAS), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical function (PPF), PROMIS Fatigue (PF) and the Rasch-built Pompe …