FGF23 and Hypophosphatemic Rickets/Osteomalacia

Curr Osteoporos Rep. 2021 Dec;19(6):669-675. doi: 10.1007/s11914-021-00709-4. Epub 2021 Nov 10.

Abstract

Purpose of review: X-linked hypophosphatemia and tumor-induced osteomalacia are diseases characterized by hypophosphatemia with impaired proximal tubular phosphate reabsorption. Complete resection of responsible tumors is the first-line therapy for patients with tumor-induced osteomalacia. In contrast, phosphate and active vitamin D have been used for patients with X-linked hypophosphatemia and inoperable ones with tumor-induced osteomalacia. The purpose of this review is to summarize the pathogenesis of these diseases and discuss about the new treatment.

Recent findings: Excessive FGF23 production has been shown to underline several kinds of hypophosphatemic rickets/osteomalacia including X-linked hypophosphatemia and tumor-induced osteomalacia. Burosumab, an anti-FGF23 monoclonal antibody, was approved for clinical use, while the indications of burosumab are different depending on countries. The inhibition of excessive FGF23 activity has been approved as a new therapy for several kinds of hypophosphatemic diseases. Further studies are necessary to clarify the long-term effects and safety of burosumab.

Keywords: Burosumab; FGF23; Hypophosphatemia; Osteomalacia; Rickets.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Familial Hypophosphatemic Rickets / drug therapy*
  • Familial Hypophosphatemic Rickets / genetics*
  • Fibroblast Growth Factor-23 / genetics*
  • Genetic Diseases, X-Linked / drug therapy
  • Genetic Diseases, X-Linked / genetics
  • Humans
  • Mice
  • Osteomalacia / drug therapy*
  • Osteomalacia / genetics*
  • Paraneoplastic Syndromes / drug therapy*
  • Paraneoplastic Syndromes / genetics*

Substances

  • Antibodies, Monoclonal, Humanized
  • FGF23 protein, human
  • Fibroblast Growth Factor-23
  • burosumab

Supplementary concepts

  • Oncogenic osteomalacia