Outcome of severe steroid-dependent nephrotic syndrome treated with mycophenolate mofetil
Sushmita Banerjee1, Amitava Pahari, Jayati Sengupta
1Institute of Child Health, 9 Greek Church Row Extension, Kolkata, India. asban444@gmail.com
Background:
Mycophenolate mofetil (MMF) is used as a steroid-sparing agent in pediatric nephrotic syndrome (NS). However, data about its long-term efficacy and safety is limited.
Methods:
We report the long-term outcome of MMF therapy in 46 NS patients who remained steroid dependent (SD) despite previous treatment with levamisole and cyclophosphamide.
Results:
After 1 year of MMF initiation, 32 (70 %) patients had reduced steroid requirement: 12 with decreased threshold dose and 20 were able to stop steroids. At follow-up of mean 3.56 (standard deviation + 1.76) years, 25 (54 %) children required no further alternative immunosuppression (IS), having infrequent or no relapses, of which 14 stopped MMF after a mean 2.4 (standard deviation + 0.9) years; 11 are continuing on MMF for a median of 2.25 years (range 1.33-7.75 years). One patient had a psoriasis flare, and MMF was stopped. No other patient required permanent drug withdrawal due to side effects. The outcome of patients who did not require further alternate IS was significantly better than those who did, with 56 % vs. 10.5 %, respectively, being off regular medications at last follow-up.
Conclusions:
We conclude that MMF therapy is safe in the long term and allows >50 % of severe SDNS patients to avoid further toxic IS.
Insights
Mycophenolate mofetil (MMF) offers long-term steroid-sparing benefits for pediatric nephrotic syndrome (NS) patients. Over 50% of severe steroid-dependent NS cases avoided further immunosuppression with MMF therapy.
Area of Science:
- Pediatric Nephrology
- Immunosuppressive Therapy
Background:
- Mycophenolate mofetil (MMF) is utilized as a steroid-sparing agent in pediatric nephrotic syndrome (NS).
- Limited data exists on the long-term efficacy and safety of MMF in this population.
Purpose of the Study:
- To evaluate the long-term outcomes of Mycophenolate mofetil (MMF) therapy in pediatric patients with steroid-dependent nephrotic syndrome (SDNS).
Main Methods:
- A cohort of 46 pediatric NS patients, steroid-dependent despite prior levamisole and cyclophosphamide treatment, received MMF.
- Long-term outcomes, including steroid requirements, relapse rates, and adverse events, were assessed.
Main Results:
- Within one year, 70% of patients showed reduced steroid needs, with 20 achieving complete steroid cessation.
- At a mean follow-up of 3.56 years, 54% of children no longer required alternative immunosuppression (IS), experiencing infrequent relapses.
- One patient experienced a psoriasis flare; no other permanent drug withdrawals due to side effects were noted.
Conclusions:
- Long-term Mycophenolate mofetil (MMF) therapy is safe for pediatric nephrotic syndrome (NS) patients.
- MMF enables over 50% of severe steroid-dependent NS patients to avoid additional toxic immunosuppressive (IS) agents.
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