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Published on: November 8, 2015

Treatment of childhood nephrotic syndrome with long-term, low-dose tacrolimus

Margret E Bock1, Richard A Cohn, Farah N Ali

  • 1Division of Kidney Diseases, Ann, Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL 60618, USA. mbock@luriechildrens.org

Clinical Nephrology
|February 6, 2013
PubMed

Insights

Low-dose, long-term tacrolimus (TAC) effectively induces and sustains remission in children with steroid-resistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS). This treatment shows high remission rates, even when other agents have failed.

Area of Science:

  • Pediatric Nephrology
  • Immunosuppressive Therapy
  • Glomerular Diseases

Background:

  • Steroid-resistant (SR) and steroid-dependent (SD) nephrotic syndrome (NS) in children present significant treatment challenges.
  • Limited literature exists on the use of tacrolimus (TAC) for maintenance therapy in pediatric NS.
  • Calcineurin inhibitors like TAC offer potential therapeutic options for refractory NS cases.

Purpose of the Study:

  • To evaluate the efficacy and safety of low-dose, long-term tacrolimus (TAC) in children with SD/SR NS.
  • To assess TAC's ability to induce and maintain remission in this challenging patient population.
  • To compare remission rates in treatment-naïve patients versus those previously treated with other agents.

Main Methods:

  • Retrospective analysis of 40 pediatric patients with NS treated with TAC from 1999 to 2009.
  • TAC treatment duration ranged from 3 to 80 months (median 25.2 months).
  • Patient diagnoses included focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), IgM nephropathy, and membranoproliferative glomerulonephritis (MPGN).

Main Results:

  • Complete remission was achieved by 26%, 48%, and 29% of patients after 1, 2, and 3 years on TAC, respectively.
  • Overall complete or partial remission rates reached 85%, 100%, and 86% at 1, 2, and 3 years (p < 0.05).
  • Median time to remission was 41 days; FSGS and SR disease were associated with lower remission likelihood (p < 0.05).

Conclusions:

  • Low-dose, long-term TAC treatment is associated with high rates of sustained remission in children with SR/SD NS.
  • TAC demonstrates efficacy even in patients who failed previous second-line therapies.
  • TAC represents a viable therapeutic option for managing difficult-to-treat pediatric nephrotic syndrome.
Abstract

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