Burosumab treatment for fibrous dysplasia.
FGF23
Hypophosphatemia
McCune-Albright syndrome
Osteomalacia
Rickets
Journal
Bone
ISSN: 1873-2763
Titre abrégé: Bone
Pays: United States
ID NLM: 8504048
Informations de publication
Date de publication:
09 2021
09 2021
Historique:
received:
17
03
2021
revised:
06
05
2021
accepted:
09
05
2021
pubmed:
14
5
2021
medline:
1
7
2021
entrez:
13
5
2021
Statut:
ppublish
Résumé
Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a rare mosaic disorder of Gα A 7-year-old boy with severe FD/MAS presented with persistent hypophosphatemia and skeletal complications despite conventional treatment with oral phosphate and calcitriol. He was started on burosumab and achieved sustained normalization of serum phosphorus and marked improvement in alkaline phosphatase levels. This was accompanied by an encouraging clinical response, including decreased bone pain, improved muscle strength, and improved ambulation. No adverse effects of burosumab therapy were observed. This is the first reported case of burosumab treatment in a patient with FD/MAS. The encouraging biochemical and clinical response in this patient highlights the need for future studies to explore the safety and efficacy of burosumab in the FD/MAS pediatric population.
Sections du résumé
BACKGROUND
Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a rare mosaic disorder of Gα
CASE DESCRIPTION
A 7-year-old boy with severe FD/MAS presented with persistent hypophosphatemia and skeletal complications despite conventional treatment with oral phosphate and calcitriol. He was started on burosumab and achieved sustained normalization of serum phosphorus and marked improvement in alkaline phosphatase levels. This was accompanied by an encouraging clinical response, including decreased bone pain, improved muscle strength, and improved ambulation. No adverse effects of burosumab therapy were observed.
CONCLUSIONS
This is the first reported case of burosumab treatment in a patient with FD/MAS. The encouraging biochemical and clinical response in this patient highlights the need for future studies to explore the safety and efficacy of burosumab in the FD/MAS pediatric population.
Identifiants
pubmed: 33984553
pii: S8756-3282(21)00166-6
doi: 10.1016/j.bone.2021.116004
pmc: PMC8272883
mid: NIHMS1707689
pii:
doi:
Substances chimiques
Antibodies, Monoclonal
0
Antibodies, Monoclonal, Humanized
0
FGF23 protein, human
0
Fibroblast Growth Factor-23
7Q7P4S7RRE
burosumab
G9WJT6RD29
Types de publication
Case Reports
Journal Article
Research Support, N.I.H., Intramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
116004Subventions
Organisme : Intramural NIH HHS
ID : ZIA DE000758
Pays : United States
Informations de copyright
Published by Elsevier Inc.
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