Autosomal dominant hypophosphatemic rickets: a case report of two sisters with a novel FGF-23 mutation.

Mumbach, Giselle; Florenzano, Pablo; Quinteros, Juan; Ávila, Claudia; Zanchetta, María Belén · Osteoporos Int · 2025

case_report · Level V

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Abstract

Autosomal dominant hypophosphatemic rickets (ADHR) is an exceptionally rare condition with fewer than 50 cases reported in the literature Int J Environ Res Public Health 18(16):8771, 2021. We present the cases of two sisters who experienced late-onset ADHR with severe clinical manifestations. Genetic analysis revealed a previously unreported mutation in the FGF-23 gene (chr12:4.370.559 G > T), likely responsible for their condition. These cases highlight the diagnostic challenges, emphasizing the critical need for genetic analysis in all patients suspected of having Tumor-Induced Osteomalacia (TIO) when the tumor remains unidentified, particularly in the presence of iron deficiency anemia.

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