Importance of early treatment and quantitative evaluation of enzyme replacement therapy for Pompe disease: alglucosidase alfa post-marketing surveillance additional analysis

Abstract

We conducted an additional analysis using the data from the post-marketing surveillance of Alglucosidase alfa for Pompe disease. We aimed to investigate the changes in the percentage of predicted forced vital capacity (%FVC) and the changes in the distance of the 6-min walk test (6MWT) by overall improvement and to investigate the %FVC change by the duration from symptom onset to survey registration (shorter/longer groups) using a linear mixed model. Thirty-seven and eighteen survey participants had %FVC and 6MWT data available, respectively; of the patients whose overall improvement was rated as “relatively improved,” %FVC and 6MWT worsened in 71.4% and 66.7%, respectively. The %FVC at the survey registration estimated using a linear mixed model was significantly higher in the shorter group than in the longer group (P = 0.0413). The estimated slope of %FVC was significantly lower in the shorter group than in the longer group (P = 0.0051). These results suggest the importance of early treatment initiation and quantitative evaluation of each symptom.