Improvement of Fibrous Dysplasia After Burosumab Therapy in a Pediatric Patient with McCune-Albright Syndrome: A Case Report.

CASE: Burosumab is a novel drug developed to treat hereditary fibroblast growth factor 23 (FGF23)-related disorders. We report the case of an 11-year-old girl with McCune-Albright syndrome (MAS) who sustained hypophosphatemia due to excess FGF23 and multiple bone lesions of fibrous dysplasia (FD). Burosumab therapy markedly improved not only the biochemical parameters but also the radiographic appearance of the FD lesions and clinical symptoms.

CONCLUSION

This is the first report to demonstrate that burosumab is effective in improving FD lesions in a patient with MAS.