Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series
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By
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Zelei He
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Yuanyuan Wu
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Hong Yang
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Yijuan Li
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Ling Xu
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June 17, 2026
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Clinical Scorecard: Clinical Efficacy of Agalsidase Beta (Fabrazyme) in Chinese Patients with Fabry Disease and Proteinuria: A Case Series Analysis
At a Glance
| Category | Detail |
|---|
| Condition | |
| Key Mechanisms | |
| Target Population | Chinese adult males with classic Fabry disease and proteinuria. |
| Care Setting | |
Key Highlights
- Two male patients with classic Fabry disease treated with agalsidase beta for 4 years.
- Significant reduction in plasma Lyso-Gb3 levels observed (70.6% and 68.5%).
- Proteinuria improved in both patients with ongoing RAAS inhibition.
- No infusion-related adverse events reported.
- Family cascade screening recommended for early diagnosis.
Guideline-Based Recommendations
Diagnosis
Management
Monitoring & Follow-up
- Regular assessment of renal function and proteinuria every 3-6 months.
- Monitoring of plasma Lyso-Gb3 levels every 6 months.
Risks
Patient & Prescribing Data
Agalsidase beta therapy led to stabilization of renal and cardiac function, with noted improvements in symptoms.
Clinical Best Practices
- Utilize standardized protocols for clinical assessments.
- Ensure consistent biomarker measurements for longitudinal studies.
- Implement family cascade screening for early diagnosis.
- Provide patient education to enhance treatment adherence.
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