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Current Osteoporosis Reports

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01-06-2021 | Rickets | Pediatrics (C Munns and F Rauch, Section Editors)

Burosumab for Pediatric X-Linked Hypophosphatemia

Author: Erik A. Imel

Published in: Current Osteoporosis Reports | Issue 3/2021

Abstract

Purpose of Review

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.

Recent Findings

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.

Summary

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.

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Title: Burosumab for Pediatric X-Linked Hypophosphatemia
Author: Erik A. Imel
Publication date: 01-06-2021
Publisher: Springer US
Keywords: Rickets
Enthesopathy
Published in: Current Osteoporosis Reports / Issue 3/2021
Print ISSN: 1544-1873
Electronic ISSN: 1544-2241
DOI: https://doi.org/10.1007/s11914-021-00669-9

At a glance: The STEP trials

Springer Medicine

Burosumab for Pediatric X-Linked Hypophosphatemia

Abstract

Purpose of Review

Recent Findings

Summary

At a glance: The STEP trials

Springer Medicine

Abstract

Purpose of Review

Recent Findings

Summary

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