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Papers of the Week


2021 May 10


Bone

Burosumab treatment for fibrous dysplasia.

Authors

Gladding A, Szymczuk V, Auble BA, Boyce AM
Bone. 2021 May 10:116004.
PMID: 33984553.

Abstract

Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a rare mosaic disorder of Gα activation. Fibroblast Growth Factor 23 (FGF23)-mediated hypophosphatemia is a feature of FD/MAS that has been associated with poor skeletal outcomes. Standard therapy includes oral phosphorus and vitamin D analogs; however, treatment is limited by potential adverse renal and gastrointestinal effects. Burosumab is a monoclonal antibody to FGF23 approved to treat patients with X-linked hypophosphatemia and tumor-induced osteomalacia. There is currently no safety or efficacy data to support burosumab use in patients with FD/MAS.