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Immunosuppressive therapy in the nephrotic syndrome in children
1Department of Paediatrics, Royal Free Hospital, London, UK.
Insights
Corticosteroids effectively treat minimal change nephrotic syndrome (MCNS) in children, reducing mortality. Further research should focus on optimizing corticosteroid delivery to minimize toxicity and improve outcomes for steroid-dependent cases.
Area of Science:
- Pediatric Nephrology
- Clinical Pharmacology
Background:
- Minimal change nephrotic syndrome (MCNS) is a common cause of nephrotic syndrome in children.
- Corticosteroids have been the cornerstone of MCNS treatment for over 30 years, significantly reducing mortality rates to below 5%.
Purpose of the Study:
- To review the efficacy of corticosteroids in inducing remission and preventing relapse in pediatric MCNS.
- To evaluate optimal methods for corticosteroid administration to maximize therapeutic benefit and minimize toxicity.
- To assess alternative immunosuppressive therapies for steroid-dependent MCNS and focal segmental glomerulosclerosis (FSGS).
Main Methods:
- Review of existing literature on corticosteroid treatment for MCNS in children.
- Analysis of outcomes for patients treated with alkylating agents (cyclophosphamide) and cyclosporine A.
- Examination of treatment responses in children with focal segmental glomerulosclerosis (FSGS).
Main Results:
- Corticosteroids are highly effective in inducing remission and preventing relapse in MCNS.
- Cyclophosphamide achieves sustained remission in approximately 50% of children with frequently relapsing, steroid-dependent MCNS.
- Cyclosporine A shows promise as an adjunctive therapy in steroid-responsive cases.
- About 30% of children with FSGS achieve remission with corticosteroids, while many require dialysis or transplantation.
Conclusions:
- Corticosteroid therapy is well-established for pediatric MCNS, with current focus shifting to optimizing administration and minimizing side effects.
- For steroid-dependent MCNS, alkylating agents and potentially cyclosporine A offer valuable treatment options.
- Immunosuppressive therapy is warranted for FSGS to prevent renal function decline, given the limited remission rates with corticosteroids alone.
Abstract:
The high incidence of remission and prevention of relapse of minimal change nephrotic syndrome (MCNS) in children, produced by corticosteroids is reviewed. With the introduction of corticosteroids over 30 years ago and the increased expertise in their use, the mortality rate has been reduced to less than 5%. There is no justification for a clinical trial to test the effect of corticosteroids in inducing remission, but the need remains to evaluate methods of administration in order to achieve therapeutic benefit with minimum toxicity. Children with frequently relapsing, steroid-dependent MCNS will usually enter remission following treatment with an alkylating agent such as cyclophosphamide. In about 50% no further relapse in experienced. The results of recent experience using cyclosporin A immunosuppression suggest a beneficial effect associated with steroid responsiveness. Approximately 30% of children with focal segmental glomerulosclerosis enter remission following treatment with corticosteroids. Some 30% require dialysis and transplantation within 5 years of diagnosis and immunosuppressive therapy to prevent deterioration of renal function is probably justified.