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Usefulness of mycophenolate mofetil in Indian patients with C3 glomerulopathy
Joyita Bharati1, Karalanglin Tiewsoh2, Ashwani Kumar3
1Department of Nephrology, Post Graduate Institute of Medical Education and Research, Chandigarh, India.
Background:
C3 glomerulopathy (C3G) is a heterogeneous disease caused by alternative complement pathway abnormalities without any standardized treatment. An immunosuppressive agent, mycophenolate mofetil (MMF), has been recently shown to be useful in treating C3G, mainly in studies from the west. We report the clinical outcome of 17 Indian C3G patients treated with MMF with or without steroids.
Methods:
The clinical and histology details of the C3G patients treated with MMF for at least 6 months with a follow-up of at least 12 months were retrieved from the medical records of our center.
Results:
The median serum creatinine and proteinuria at presentation were 0.8 mg/dL and 3.7 g/day, respectively, with the majority (88.2%) presenting as nephrotic syndrome. The mean dose of MMF was 1.65 (±0.56) g/day, and the median duration of MMF therapy was 18 months. Two-thirds (64%) of the patients responded to the treatment, with complete remission in 4 (23%) and partial remission in 7 (41%) (median time: 9 months). Three patients progressed to end-stage renal disease (ESRD) on follow-up. Of the three patients, one (33%) had an initial response in proteinuria to MMF but did not respond after a relapse and subsequently progressed to ESRD and two (67%) other patients were nonresponsive to MMF from the start of the therapy.
Conclusion:
Despite a small sample size and lack of a control arm, this study describes the effectiveness of MMF in treating C3G patients from Asia and forms a basis for future randomized trials.
Insights
Mycophenolate mofetil (MMF) shows promise in treating C3 glomerulopathy (C3G), a rare kidney disease. This study found that MMF treatment led to remission in two-thirds of Indian patients with C3G.
Area of Science:
- Nephrology
- Immunology
- Complement System
Background:
- C3 glomerulopathy (C3G) is a rare kidney disease characterized by alternative complement pathway dysregulation.
- Currently, no standardized treatment exists for C3G, presenting a significant unmet medical need.
- Mycophenolate mofetil (MMF), an immunosuppressant, has shown potential in Western studies for C3G management.
Purpose of the Study:
- To evaluate the clinical outcomes of C3 glomerulopathy (C3G) patients treated with mycophenolate mofetil (MMF) in an Indian population.
- To assess the efficacy and safety of MMF, with or without steroids, in managing C3G.
Main Methods:
- Retrospective analysis of clinical and histological data from 17 Indian C3G patients treated with MMF for at least six months.
- Follow-up duration was a minimum of 12 months.
- Data included baseline serum creatinine, proteinuria, and treatment response to MMF.
Main Results:
- The majority of patients (88.2%) presented with nephrotic syndrome, with median proteinuria of 3.7 g/day.
- Two-thirds (64%) of patients responded to MMF therapy, achieving complete remission in 23% and partial remission in 41%.
- Three patients progressed to end-stage renal disease (ESRD), with two being non-responsive to MMF from the outset.
Conclusions:
- Mycophenolate mofetil (MMF) demonstrates effectiveness in treating C3 glomerulopathy (C3G) in an Asian cohort, supporting its use in this population.
- This study provides a foundation for larger, randomized controlled trials to further establish MMF's role in C3G management.
- The findings highlight the potential of MMF as a therapeutic option for C3G patients, particularly in regions where standardized treatments are lacking.
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