Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzyme replacement therapy improves health outcomes for adults with late-onset Pompe disease (LOPD). PROPEL was a Phase 3, double-blind, randomized controlled trial, which evaluated cipaglucosidase alfa plus miglustat, vs alglucosidase alfa plus placebo in 123 adult patients with LOPD (clinicaltrials.gov: NCT03729362).