Hyperoxaluria and systemic oxalosis: Current therapy and future directions

Amy E. Bobrowski, Craig B. Langman*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

26 Scopus citations

Abstract

Excessive urinary oxalate excretion, termed hyperoxaluria, may arise from inherited or acquired diseases. The most severe forms are caused by increased endogenous production of oxalate related to one of several inborn errors of metabolism, termed primary hyperoxaluria. Recurrent kidney stones and progressive medullary nephrocalcinosis lead to the loss of kidney function, requiring dialysis or transplantation, accompanied by systemic oxalate deposition that is termed systemic oxalosis. For most primary hyperoxalurias, accurate diagnosis leads to the use of therapies that include pyridoxine supplementation, urinary crystallisation inhibitors, hydration with enteral fluids and, in the near future, probiotic supplementation or other innovative therapies. These therapies have varying degrees of success, and none represent a cure. Organ transplantation results in reduced patient and organ survival when compared with national statistics. Exciting new approaches under investigation include the restoration of defective enzymatic activity through the use of chemical chaperones and hepatocyte cell transplantation, or recombinant gene therapy for enzyme replacement. Such approaches give hope for a future therapeutic cure for primary hyperoxaluria that includes correction of the underlying genetic defect without exposure to the life-long dangers associated with organ transplantation.

Original languageEnglish (US)
Pages (from-to)1887-1896
Number of pages10
JournalExpert Opinion on Pharmacotherapy
Volume7
Issue number14
DOIs
StatePublished - Oct 1 2006

Keywords

  • Alanine glyoxylate aminotransferase
  • Dialysis
  • Glyoxylate reductase/hydroxypyruvate reductase
  • Hyperoxaluria
  • Inborn errors of metabolism
  • Kidney stones
  • Kidney transplantation
  • Liver transplantation
  • Peroxisomal disorders
  • Probiotics
  • Pyridoxine
  • Systemic oxalosis

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

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