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Objectives <p>The&#xa0;primary objective was to evaluate the efficacy of mycophenolate mofetil (MMF), assessed by stable remission rates, in pediatric&#xa0;frequently relapsing or steroid-dependent nephrotic syndrome (FRNS/SDNS).&#xa0;Secondary objectives&#xa0;included assessing treatment failure, relapse rates, adverse effects, and sustained remission following MMF discontinuation.</p> Methods <p>This cohort enrolled children aged &lt; 18&#xa0;years with FRNS/SDNS initiated on MMF between 2010 and 2024. Longitudinal outcomes were noted and analyzed.</p> Results <p>A total of 101 children with FRNS/SDNS had a median (q1, q3) age of 5.5 (4, 8.8) years at MMF initiation. In the year preceding MMF initiation, children experienced 3.6 (1.3) relapses and a cumulative steroid dose of 5033 (4297, 5969) mg/m<sup>2</sup>. At 12&#xa0;months of MMF therapy, 81 (80.1%) children were in stable remission (33 in sustained remission, 48 with infrequent relapses), 12 (11.8%) had frequent relapses, and 8 (7.9%) had treatment failure. At a median follow-up of 24 (18, 36) months, 66 (65.3%) children were in stable remission (29 in sustained remission, 37 with infrequent relapses), 16 (15.8%) had frequent relapses, and 19 (18.8%) had treatment failure. Relapse rates reduced by 82% with a relative relapse rate of&#xa0;0.18&#xa0;(95% CI 0.15–0.22) relapses/person-year. Cumulative steroid dose decreased by 57.5% in the first year and 91.5% in the second year among those in stable remission. Adverse effects included three serious infections and one persistent thrombocytopenia. The median relapse-free survival was 5 (3, 9) months. The median follow-up post-discontinuation of MMF (<i>n</i> = 34) was 2.1 (0.1, 4.3) years; 15 (44.1%) maintained remission at 1&#xa0;year.</p> Conclusion <p>MMF is an efficacious steroid-sparing agent for pediatric FRNS/SDNS with minimal residual effect.</p>

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A Longitudinal Study of Clinical Outcomes and Efficacy of Mycophenolate Mofetil in Pediatric Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome

  • 期刊论文

  • 发表日期：

    2026年5月5日

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  • Ramesh Abhay,
  • Bobbity Deepthi,
  • Sudarsan Krishnasamy,
  • Sriram Krishnamurthy

摘要

Objectives

The primary objective was to evaluate the efficacy of mycophenolate mofetil (MMF), assessed by stable remission rates, in pediatric frequently relapsing or steroid-dependent nephrotic syndrome (FRNS/SDNS). Secondary objectives included assessing treatment failure, relapse rates, adverse effects, and sustained remission following MMF discontinuation.

Methods

This cohort enrolled children aged < 18 years with FRNS/SDNS initiated on MMF between 2010 and 2024. Longitudinal outcomes were noted and analyzed.

Results

A total of 101 children with FRNS/SDNS had a median (q1, q3) age of 5.5 (4, 8.8) years at MMF initiation. In the year preceding MMF initiation, children experienced 3.6 (1.3) relapses and a cumulative steroid dose of 5033 (4297, 5969) mg/m2. At 12 months of MMF therapy, 81 (80.1%) children were in stable remission (33 in sustained remission, 48 with infrequent relapses), 12 (11.8%) had frequent relapses, and 8 (7.9%) had treatment failure. At a median follow-up of 24 (18, 36) months, 66 (65.3%) children were in stable remission (29 in sustained remission, 37 with infrequent relapses), 16 (15.8%) had frequent relapses, and 19 (18.8%) had treatment failure. Relapse rates reduced by 82% with a relative relapse rate of 0.18 (95% CI 0.15–0.22) relapses/person-year. Cumulative steroid dose decreased by 57.5% in the first year and 91.5% in the second year among those in stable remission. Adverse effects included three serious infections and one persistent thrombocytopenia. The median relapse-free survival was 5 (3, 9) months. The median follow-up post-discontinuation of MMF (n = 34) was 2.1 (0.1, 4.3) years; 15 (44.1%) maintained remission at 1 year.

Conclusion

MMF is an efficacious steroid-sparing agent for pediatric FRNS/SDNS with minimal residual effect.