New therapies in steroid-sensitive and steroid-resistant idiopathic nephrotic syndrome

Pediatr Nephrol. 2011 Jun;26(6):881-92. doi: 10.1007/s00467-010-1717-5. Epub 2011 Jan 13.

Abstract

Although many children with idiopathic nephrotic syndrome (INS) respond initially to steroid therapy, repeated courses for patients with relapses often cause significant steroid toxicity. Patients with frequent relapses who develop steroid dependency thus require alternative treatment. The first such options have been considered to be cyclophosphamide or levamisole, although the latter is no longer available in many countries. There is also an increasing body of data indicating that mycophenolic acid (MPA) may be an alternative for these patients. Calcineurin inhibitors (cyclosporine A or tacrolimus) are usually effective and often used after cytotoxic treatment, but long-term treatment with these agents is necessary, raising concerns of a possible accumulation of side effects. Some patients show a tendency to relapse even on such maintenance regimens, and some even have a refractory course that creates a medical dilemma. For this situation, recent data indicate that monoclonal antibodies directed to B-cells (e.g. rituximab) may have some effect and that such drugs may also prove to be a therapeutic option in less complicated cases. Patients that do not respond to steroid treatment need genetic testing and a renal biopsy since focal segmental glomerulosclerosis (FSGS) may be present. Treatment options include pulse methylprednisolone, often in addition to calcineurin inhibitors, mainly in the form of cyclosporine, but tacrolimus has also come into recent favor. Some studies have found cytotoxic treatment, especially intravenous cyclophosphamide, to be effective in steroid resistant nephrotic syndrome, but it seems to be inferior to calcineurin inhibitors. MPA and rituximab have also been used in children with primary FSGS, but the response seems to be inferior to that in patients with steroid sensitive nephrotic syndrome. Taken together, INS in both steroid-sensitive and steroid-resistant patients is a potentially complicated disorder, and despite a wide arsenal of immunological interventions, some patients have a treatment refractory course. Prospective studies or at least standardized treatment for complicated cases is urgently needed.

Publication types

  • Review

MeSH terms

  • Antibiotics, Antineoplastic / therapeutic use
  • Antibodies, Monoclonal, Murine-Derived / therapeutic use
  • Child
  • Drug Substitution / methods*
  • Drug Therapy, Combination
  • Glucocorticoids / therapeutic use*
  • Humans
  • Immunologic Factors / therapeutic use
  • Levamisole / therapeutic use
  • Mycophenolic Acid / therapeutic use
  • Nephrotic Syndrome / congenital*
  • Nephrotic Syndrome / drug therapy
  • Recurrence
  • Rituximab

Substances

  • Antibiotics, Antineoplastic
  • Antibodies, Monoclonal, Murine-Derived
  • Glucocorticoids
  • Immunologic Factors
  • Levamisole
  • Rituximab
  • Mycophenolic Acid

Supplementary concepts

  • Nephrotic syndrome, idiopathic, steroid-resistant