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Childhood nephrotic syndrome--current and future therapies

Larry A Greenbaum1, Rainer Benndorf, William E Smoyer

  • 1Emory University, Atlanta, GA 30322, USA.

Nature Reviews. Nephrology
|June 13, 2012
PubMed

Insights

Corticosteroids are standard for childhood nephrotic syndrome, but some children are resistant. New treatments targeting podocyte dysfunction are needed for steroid-resistant nephrotic syndrome to prevent kidney failure.

Area of Science:

  • Pediatric Nephrology
  • Molecular Medicine
  • Immunology

Background:

  • Corticosteroids significantly improved outcomes for childhood nephrotic syndrome over 50 years ago.
  • A substantial portion of children with nephrotic syndrome are steroid-resistant, risking progression to end-stage renal disease.
  • Current treatments for steroid-resistant nephrotic syndrome include immunosuppressive agents like ciclosporin and tacrolimus.

Purpose of the Study:

  • To review current treatments for childhood nephrotic syndrome.
  • To discuss novel therapeutic pathways targeting podocytes for future treatments.
  • To challenge the traditional immune theory of nephrotic syndrome pathogenesis.

Main Methods:

  • Literature review of current and emerging treatments for childhood nephrotic syndrome.
  • Analysis of evidence implicating podocyte dysfunction in nephrotic syndrome.
  • Discussion of potential molecular targets in podocytes for next-generation therapies.

Main Results:

  • Corticosteroids are the primary treatment, but steroid resistance remains a significant clinical challenge.
  • Emerging evidence suggests podocyte dysfunction, not solely T-cell mediated immunity, underlies nephrotic syndrome.
  • Conventional immunosuppressants like glucocorticoids and ciclosporin directly impact podocyte function.

Conclusions:

  • Novel therapeutic strategies for steroid-resistant nephrotic syndrome should focus on podocyte-targeted therapies.
  • Understanding podocyte biology is crucial for developing next-generation treatments.
  • Future research should explore pathways to restore podocyte integrity and function in pediatric nephrotic syndrome.

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