Burosumab for Pediatric X-Linked Hypophosphatemia.
Burosumab
FGF23
Rickets
X-Linked hypophosphatemia
Journal
Current osteoporosis reports
ISSN: 1544-2241
Titre abrégé: Curr Osteoporos Rep
Pays: United States
ID NLM: 101176492
Informations de publication
Date de publication:
06 2021
06 2021
Historique:
accepted:
12
02
2021
pubmed:
11
5
2021
medline:
8
1
2022
entrez:
10
5
2021
Statut:
ppublish
Résumé
X-Linked hypophosphatemia (XLH) is the most common genetic cause of rickets. This review describes advances in the management of XLH using burosumab which was FDA approved for treating children with XLH in 2018. Elevated FGF23 in XLH leads to systemic hypophosphatemia and several musculoskeletal manifestations, including rachitic bone deformities, impaired growth, dental abscesses, insufficiency fractures, osteoarthritis, and enthesopathy, with lifelong consequences for physical function and quality of life. Burosumab treatment has demonstrated clinical improvement of rickets and growth in children, including during a randomized controlled trial compared with conventional therapy. Burosumab also improved pseudofracture healing in adults. Burosumab led to greater improvement in rickets and growth than conventional therapy. However, many questions remain regarding the impact of burosumab on several outcomes, including final height, nephrocalcinosis, dental disease, enthesopathy, and surgical interventions.
Identifiants
pubmed: 33970403
doi: 10.1007/s11914-021-00669-9
pii: 10.1007/s11914-021-00669-9
pmc: PMC9387050
mid: NIHMS1828490
doi:
Substances chimiques
Antibodies, Monoclonal, Humanized
0
burosumab
G9WJT6RD29
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
271-277Subventions
Organisme : NIAMS NIH HHS
ID : P30 AR072581
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR002529
Pays : United States
Informations de copyright
© 2021. Springer Science+Business Media, LLC, part of Springer Nature.
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