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Alglucosidase alfa demonstrates effectiveness and safety in Chinese patients with late-onset Pompe disease: A
Duoling Li1, Kexin Jiao2, Huan Yang3
1Department of Neurology, Shandong Key Laboratory of Mitochondrial Medicine and Rare Diseases, Research Institute of Neuromuscular and Neurodegenerative Diseases, Qilu Hospital of Shandong University, Jinan, China.
Insights
Alglucosidase alfa treatment improved ambulation and muscle strength in Chinese patients with late-onset Pompe disease (LOPD). The therapy demonstrated a favorable safety profile over 52 weeks, indicating effectiveness for LOPD management.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Pompe disease is a rare, progressive lysosomal disorder.
- Enzyme replacement therapy (ERT) is a standard treatment for Pompe disease.
- Alglucosidase alfa is approved in China for infantile-onset and late-onset Pompe disease (LOPD).
Purpose of the Study:
- To assess the effectiveness and safety of 52-week Alglucosidase alfa treatment in Chinese LOPD patients.
- Evaluate improvements in ambulation, respiratory function, muscle strength, and quality of life.
- Monitor treatment-emergent adverse events (TEAEs).
Main Methods:
- A multicenter, single-arm, open-label, prospective study.
- 41 eligible LOPD patients received Alglucosidase alfa (20 mg/kg) every 2 weeks for 52 weeks.
- Primary endpoints: 6-minute walk test (6MWT), forced vital capacity (FVC). Secondary endpoints: muscle strength tests, quality of life surveys.
Main Results:
- Significant improvement in 6MWT distance (43.6 m, P=0.0017).
- Significant improvements in Manual Muscle Test (MMT) and Quick Motor Function Test (QMFT) scores (P<0.0001).
- 32 participants (78.0%) experienced mild to moderate TEAEs, resolving without sequelae.
Conclusions:
- Alglucosidase alfa demonstrated significant improvements in motor function for Chinese LOPD patients.
- The treatment showed a positive benefit-risk profile, confirming its safety and effectiveness.
- Alglucosidase alfa is a viable therapeutic option for Chinese LOPD patients.
Objective:
Pompe disease is a rare, progressively debilitating lysosomal disorder. Enzyme replacement therapy (ERT) is an established treatment targeting the underlying enzyme deficiency. Alglucosidase alfa has shown benefits in survival, ambulation, and respiratory function and was approved in China for both infantile-onset (IOPD)and late-onset Pompe disease (LOPD) in 2015.To assess the effectiveness and safety of 52-week Alglucosidase alfa treatment among Chinese patients with LOPD in a multicenter, single arm, open-label, prospective clinical study.
Methods:
Forty-one eligible LOPD patients received Alglucosidase alfa infusions at 20 mg/kg every 2 weeks for 52 weeks. Primary endpoints included the six-minute walk test (6MWT), percentage of predicted forced vital capacity (FVC), and safety profile. Secondary endpoints encompassed manual muscle test (MMT), maximal inspiratory and expiratory pressure (MIP and MEP), Quick motor function test (QMFT) scores, and health-related quality of life (SF-12).
Results:
After 52 weeks, the mean 6MWT distance significantly improved by 43.6 m (P = 0.0017). Although the mean percentage of predicted FVC improved by 2.4%, the difference was not statistically significant (P = 0.1424). MIP and MEP percentages improved, reaching maximal improvement at 38 weeks. Both MMT and QMFT scores demonstrated significant improvements, with increases of 2.5 points (P < 0.0001) and 5.6 points (P < 0.0001), respectively. PCS significantly improved by 3.8 points (P = 0.0039), while MCS improvement was not statistically significant. A total of 32 participants (78.0%) experienced 137 treatment-emergent adverse events (TEAEs). Most of these TEAEs were mild to moderate in severity and resolved without sequelae.
Conclusions:
Alglucosidase alfa demonstrated a positive benefit-risk profile in Chinese LOPD patients, confirming its safety and effectiveness.
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