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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.
Background:
Current treatments for primary focal segmental glomerulosclerosis (FSGS), including corticosteroids and cyclosporine, are not satisfactory for all patients and may induce significant side effects. Antidotal benefits of mycophenolate mofetil (MMF) as an add-on to these immunosuppressive therapies have been reported. This review aims to systematically summarize the efficacy and safety of MMF as a treatment for primary FSGS.
Method:
Controlled and uncontrolled clinical trials evaluating the use of MMF in primary FSGS patients were identified from nine electronic databases and four clinical trial registries. Kidney failure was selected as the primary outcome.
Results:
Three randomized controlled trials (RCT) and 18 uncontrolled pre-post studies were included. Results from RCTs revealed that MMF is no more effective than cyclosporine or cyclophosphamide for promoting kidney function preservation when corticosteroid is used as baseline treatment. One underpowered RCT reported that MMF provides no extra benefit on top of prednisolone, but the result is unlikely to be reliable. Amongst the small, uncontrolled pre-post studies, three of them used MMF as monotherapy, two of which reported successful prevention of kidney failure in all patients. The remaining 15 uncontrolled studies used MMF as add-on therapy and 11 reported kidney failure as an outcome. Amongst them, eight reported no patients developed kidney failure. MMF was generally well tolerated with mild adverse effects, including abdominal discomfort, diarrhea and infections.
Conclusions:
MMF tended to show beneficial effects in uncontrolled studies which recruited patients with resistance to routine treatments, but such favorable results have only been reported in small, uncontrolled trials. No RCT results suggested that MMF was a good alternative to cyclosporine or cyclophosphamide. The role of MMF as an add-on to current therapies, or as monotherapy, should further be evaluated.
Insights
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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