Clinical Efficacy of Agalsidase Beta in Chinese Patients with Fabry Disease
Overview
This report presents the clinical outcomes of two Chinese male patients with Fabry disease treated with agalsidase beta. Both patients showed significant reductions in plasma Lyso-Gb3 levels and improvements in renal function and proteinuria following enzyme replacement therapy.
Background
Incorporate statistics or references to studies on ERT efficacy in Chinese patients.
Data Highlights
Patient
Mutation
Age at ERT Start
Lyso-Gb3 Reduction
Proteinuria Change
Case 1
GLA c.493G > T
32
70.6% (93.33 to 27.38 ng/mL)
Resolved post-transplant
Case 2
GLA c.1201T > C
37
68.5% (79.17 to 24.93 ng/mL)
2195.52 to 1834.34 mg/24 h
Key Findings
Agalsidase beta was well-tolerated in both patients with no infusion-related adverse events.
Case 1 underwent kidney transplantation after 21 months of ERT, with stabilized renal function post-transplant.
Case 2 showed a significant decrease in Lyso-Gb3 levels and improvement in proteinuria after 4 years of ERT.
Both patients experienced regression of symptoms such as neuropathic pain and gastrointestinal discomfort.
Concomitant RAAS inhibition was necessary for managing proteinuria in both cases.
Clinical Implications
The findings suggest that agalsidase beta can stabilize renal and cardiac function in patients with Fabry disease. Clinicians should consider early initiation of ERT and the importance of managing proteinuria through RAAS inhibition to optimize patient outcomes.
Conclusion
Agalsidase beta demonstrates clinical efficacy in managing Fabry disease in Chinese patients, contributing to improved renal function and symptom relief. Continued monitoring and early intervention are essential for optimal management.
