Recent advances in fibroblast growth factor 23-related hypophosphatemic disorders.

IF 2.6 3区医学 Q3 ENDOCRINOLOGY & METABOLISM

Current Opinion in Endocrinology & Diabetes and Obesity Pub Date : 2024-08-01 Epub Date: 2024-04-30 DOI:10.1097/MED.0000000000000866

Yuichi Takashi, Daiji Kawanami, Seiji Fukumoto

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引用次数: 0

Abstract

Purpose of review: Fibroblast growth factor 23 (FGF23) is a hormone to reduce blood phosphate concentration. Excessive actions of FGF23 induce FGF23-related hypophosphatemic disorders, such as X-linked hypophosphatemic rickets (XLH) and tumor-induced osteomalacia (TIO). We will summarize recent advances in the diagnosis and treatment of FGF23-related hypophosphatemic disorders.

Recent findings: The measurement of blood FGF23 is useful to make a diagnosis of FGF23-related hypophosphatemic disorders. It was reported that many patients with FGF23-related hypophosphatemic disorders, especially TIO, were misdiagnosed, therefore, it is necessary to enhance the awareness of these diseases. A novel method to inhibit excessive actions of FGF23 by a human monoclonal antibody for FGF23, burosumab, has been approved in several countries. In more long-term observation than clinical trials, burosumab has also been shown to improve biochemical abnormalities and symptoms of rickets/osteomalacia. Following these advances, several registries and consensus recommendations on FGF23-related hypophosphatemic disorders, especially XLH, have been established in each country or region.

Summary: Further long-term effects of burosumab and the precise mechanism of FGF23 overproduction in patients with FGF23-related hypophosphatemic disorders need to be clarified in the future studies.

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成纤维细胞生长因子 23 相关低磷血症的最新进展。

综述目的：成纤维细胞生长因子 23（FGF23）是一种降低血液磷酸盐浓度的激素。FGF23的过量作用会诱发与FGF23相关的低磷血症，如X连锁低磷血症佝偻病（XLH）和肿瘤诱发骨软化症（TIO）。我们将总结诊断和治疗 FGF23 相关性低磷血症的最新进展：测量血液中的FGF23有助于诊断FGF23相关性低磷血症。据报道，许多与FGF23相关的低磷血症（尤其是TIO）患者被误诊，因此有必要加强对这些疾病的认识。一种通过 FGF23 人单克隆抗体（burosumab）抑制 FGF23 过度作用的新方法已在多个国家获得批准。在比临床试验更长期的观察中，burosumab 也被证明可以改善生化异常和佝偻病/骨软化症的症状。在取得这些进展后，各国或各地区建立了多个登记处，并就与 FGF23 相关的低磷血症，尤其是 XLH，提出了共识性建议：小结：布罗索单抗的长期疗效以及FGF23相关性低磷血症患者体内FGF23过度生成的确切机制仍需在今后的研究中进一步明确。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Opinion in Endocrinology & Diabetes and Obesity ENDOCRINOLOGY & METABOLISM-

CiteScore

5.80

自引率

3.10%

发文量

128

审稿时长

6-12 weeks

期刊介绍： Current Opinion in Endocrinology, Diabetes and Obesity delivers a broad-based perspective on the most recent and exciting developments in the field from across the world. Published bimonthly and featuring twelve key topics – including androgens, gastrointestinal hormones, diabetes and the endocrine pancreas, and neuroendocrinology – the journal’s renowned team of guest editors ensure a balanced, expert assessment of the recently published literature in each respective field with insightful editorials and on-the-mark invited reviews.