Summary

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• Myozyme (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency).
• This summary is based on the review of three systematic review(s)/meta-analysis(es). ^[1-3]
• Infantile-Onset Pompe Disease (IOPD): Alglucosidase alfa was associated with an improvement in Left Ventricular (LV) mass, with a mean change of 131.3 g/m² (95% CI: 81.02, 181.59) over 48.3 months. The hazard ratio (HR) for time to start ventilation (TSV) was 0.21 (95% CI: 0.12, 0.36), indicating delayed need for ventilation. The HR for survival was 0.10 (95% CI: 0.05, 0.19), suggesting a survival benefit.
• Late-Onset Pompe Disease (LOPD): Alglucosidase alfa resulted in an improvement in the 6-Minute Walking Test (6MWT) by 35.7 meters (95% CI: 7.78, 63.75) over 32.5 months. The physical domain of the SF-36 QOL questionnaire showed a mean improvement of 1.96 (95% CI: 0.33, 3.59). Time on ventilation (TOV) was reduced by -2.64 hours (95% CI: -5.28, 0.00).
• Adverse events (AEs) after enzyme replacement therapy (ERT) for both Infantile-Onset Pompe Disease (IOPD) and Late-Onset Pompe Disease (LOPD) were generally mild in most cases.
• No significant safety concerns or severe adverse effects were highlighted for specific population types or subgroups.
• There is no population types or subgroups information available in the reviewed studies.