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Mycophenolate mofetil for primary focal segmental glomerulosclerosis: systematic review
Emily W Y Lau1, Polly H X Ma, Xinyin Wu
1The Jockey Club School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong.
Mycophenolate mofetil (MMF) shows promise for primary focal segmental glomerulosclerosis (FSGS) in uncontrolled studies, but lacks strong evidence from randomized controlled trials (RCTs). Further research is needed to confirm its efficacy and safety.
Area of Science:
- Nephrology
- Immunosuppression
- Glomerular Diseases
Background:
- Primary focal segmental glomerulosclerosis (FSGS) treatments like corticosteroids and cyclosporine have limitations.
- Mycophenolate mofetil (MMF) is explored as an add-on therapy for FSGS.
- Existing treatments for FSGS are not universally effective and can cause side effects.
Purpose of the Study:
- To systematically review the efficacy and safety of MMF in treating primary FSGS.
- To evaluate MMF's role as monotherapy or add-on treatment for FSGS.
Main Methods:
- Systematic review of controlled and uncontrolled clinical trials.
- Inclusion of studies from nine electronic databases and four clinical trial registries.
- Kidney failure identified as the primary outcome measure.
Main Results:
- Randomized controlled trials (RCTs) found MMF not superior to cyclosporine or cyclophosphamide for kidney preservation with corticosteroid baseline.
- Uncontrolled studies suggested potential benefits of MMF monotherapy and add-on therapy in preventing kidney failure.
- MMF was generally well-tolerated, with mild side effects like gastrointestinal issues and infections.
Conclusions:
- Favorable outcomes in uncontrolled FSGS studies using MMF require cautious interpretation due to small sample sizes and lack of randomization.
- Current RCT evidence does not support MMF as a primary alternative to established immunosuppressants for FSGS.
- Further rigorous evaluation of MMF's role in FSGS treatment is warranted.
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