Available and incoming therapies for idiopathic focal and segmental glomerulosclerosis in adults

J Nephrol. 2018 Feb;31(1):37-45. doi: 10.1007/s40620-017-0402-1. Epub 2017 May 3.

Abstract

Focal and segmental glomerulosclerosis (FSGS) is a histological pattern clinically characterized by nephrotic proteinuria, hypoalbuminemia, edema and dyslipidemia. Approximately 50% of patients progress to end-stage renal disease within 5-10 years, particularly those not responding to the therapies. FSGS pathogenesis is largely unknown and therapy is symptomatic and unspecific. The podocyte is considered as the pathogenetic main target and FSGS is now categorized as a podocytopathy together with minimal change disease, diffuse mesangial proliferation and collapsing glomerulonephritis. This paper provides an overview on the treatment of idiopathic FSGS in adults, citing the latest published trials and the most reliable pathogenetic hypotheses of the disease. A large part of the review then focuses on emerging therapies, specifying for each new drug the assumed mechanism of action and the data available in the literature on the drug's use in experimental animals and humans.

Keywords: Focal and segmental glomerulosclerosis; Nephrotic syndrome; Podocyte; Therapy.

Publication types

  • Review

MeSH terms

  • Animals
  • Cell Proliferation
  • Clinical Decision-Making
  • Drugs, Investigational / adverse effects
  • Drugs, Investigational / therapeutic use*
  • Glomerulosclerosis, Focal Segmental / diagnosis
  • Glomerulosclerosis, Focal Segmental / drug therapy*
  • Glomerulosclerosis, Focal Segmental / epidemiology
  • Glomerulosclerosis, Focal Segmental / physiopathology
  • Humans
  • Plasmapheresis
  • Podocytes / drug effects*
  • Podocytes / pathology
  • Renal Agents / adverse effects
  • Renal Agents / therapeutic use*
  • Risk Factors
  • Stem Cell Transplantation
  • Treatment Outcome

Substances

  • Drugs, Investigational
  • Renal Agents