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

Kristl G ClaeysHani KushlafSyed RazaNoemi HummelSimon ShohetIan KeyzorAgnieszka KopiecRyan GrahamBrian FoxBenedikt Schoser

Published in: Orphanet journal of rare diseases (2024)

Our analysis shows that MCID depends on the chosen method and disease severity. The findings suggest that applying a single MCID to all patients can be misleading; consequently, a range of possible MCIDs should be considered. This may also be highly relevant for other neuromuscular diseases. This study provides a range of 6MWD MCIDs for LOPD, with lower MCIDs for more severe patients.

Keyphrases

late onset
end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
early onset
peritoneal dialysis
smoking cessation