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FGF23 and Hypophosphatemic Rickets/Osteomalacia

  • Epidemiology and Pathophysiology (J Compston and M Rothman, Section Editors)
  • Published:
Current Osteoporosis Reports Aims and scope Submit manuscript

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.

Summary

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.

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This work was supported in part by Grant-in-Aid for Young Scientists 18K15980 (JSPS) (to Y.T.) and Grant-in-Aid for Scientific Research 19H03676 (to S.F.) both from the Japan Society for the Promotion of Sciences.

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  1. Department of Endocrinology and Diabetes Mellitus, Fukuoka University School of Medicine, Fukuoka, Japan

    Yuichi Takashi & Daiji Kawanami

  2. Department of Molecular Endocrinology, Fujii Memorial Institute of Medical Sciences, Institute of Advanced Medical Sciences, Tokushima University, 3-18-15 Kuramoto-cho, Tokushima, 770-8503, Japan

    Seiji Fukumoto

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  1. Yuichi Takashi
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Correspondence to Seiji Fukumoto.

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Department of Molecular Endocrinology in Tokushima University is supported by Kyyowa Kirin Co., Ltd.

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Takashi, Y., Kawanami, D. & Fukumoto, S. FGF23 and Hypophosphatemic Rickets/Osteomalacia. Curr Osteoporos Rep 19, 669–675 (2021). https://doi.org/10.1007/s11914-021-00709-4

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