Alglucosidase alfa demonstrates effectiveness and safety in Chinese patients with late-onset Pompe disease: A multi-center prospective study - PubMed
6 hours ago
- #Clinical trial
- #Enzyme replacement therapy
- #Pompe disease
- Alglucosidase alfa shows effectiveness and safety in Chinese patients with late-onset Pompe disease (LOPD).
- A 52-week multi-center prospective study involved 41 LOPD patients receiving Alglucosidase alfa infusions (20 mg/kg every 2 weeks).
- Primary endpoints: significant improvement in six-minute walk test (6MWT) by 43.6 m (P = 0.0017), but forced vital capacity (FVC) improvement was not statistically significant.
- Secondary endpoints: significant improvements in manual muscle test (MMT), maximal inspiratory/expiratory pressure (MIP/MEP), and Quick motor function test (QMFT) scores.
- Health-related quality of life (SF-12) physical component score (PCS) improved significantly, but mental component score (MCS) did not.
- 78% of participants experienced mild to moderate treatment-emergent adverse events (TEAEs), all resolved without long-term effects.
- Conclusion: Alglucosidase alfa has a positive benefit-risk profile for Chinese LOPD patients.