# A Retrospective Cohort Analysis of Short-Term Outcomes of the Mycophenolate Mofetil-Based Triple Regimen for the Treatment of Calcineurin Inhibitor-Resistant Childhood Nephrotic Syndrome: A Single-Center Experience

## TL;DR

This study examines the effectiveness of a triple therapy including mycophenolate mofetil in children with treatment-resistant nephrotic syndrome, showing promising short-term results.

## Contribution

The study provides new evidence for a cost-effective treatment option for calcineurin inhibitor-resistant childhood nephrotic syndrome.

## Key findings

- 65% of children responded to the addition of mycophenolate mofetil to their regimen.
- Half of the responding children achieved complete remission.
- Serum albumin levels improved significantly with the new treatment.

## Abstract

Background

Idiopathic nephrotic syndrome (INS) is a heterogeneous group of disorders. A variable number of children do not respond to the conventional first line of treatment and are at risk of progression and complications of nephrotic syndrome. There is limited literature on the use of a combination of alternative immunosuppressive agents in children with calcineurin inhibitor (CNI)-resistant nephrotic syndrome (NS). This study is designed to review short-term outcomes of children who have failed to respond to CNI with documented adequate levels and preserved renal function.

Methods

Of the 490 registered children receiving CNI, retrospective data were reviewed for 37 patients who received mycophenolate mofetil (MMF) along with conventional CNI and steroids after demonstration of CNI resistance with adequate drug levels. Using the predesigned proforma, clinical, histopathological, and laboratory parameters were collected. Outcomes were classified as complete, partial, and no response.

Results

The median age of the study participants was 10 years (interquartile range (IQR): 7.75-13.2), with a male predominance of 62%. A total of 24 out of 37 (65%) children responded to the addition of MMF, with half of them achieving complete remission (CR). With the addition of MMF to the conventional regimen, the improvement in serum albumin was statistically significant (OR: 9.29, 95% CI: 1.66-51.74; p = 0.011).

Conclusion

This retrospective study demonstrates the short-term efficacy of MMF-based triple regimen in achieving remission in pediatric CNI-resistant nephrotic syndrome and is a cost-effective treatment option. Larger, prospective studies with longer follow-up are, however, required to confirm these findings.