1. [Importance of early treatment and quantitative evaluation of enzyme replacement therapy for Pompe disease: alglucosidase alfa post-marketing surveillance additional analysis].
- Author
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Sunaga Y, Sakashita T, Koga T, Sawada T, Yamane S, and Ikeda M
- Subjects
- Humans, Male, Female, Adult, Middle Aged, Vital Capacity, Adolescent, Time Factors, Child, Treatment Outcome, Young Adult, Aged, Time-to-Treatment, Glycogen Storage Disease Type II drug therapy, Enzyme Replacement Therapy, Product Surveillance, Postmarketing, alpha-Glucosidases administration & dosage, Walk Test
- 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.
- Published
- 2024
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