FGF23-Related Hypophosphataemic Bone Disease

López, Antonio González-Meneses

doi:10.1007/s12325-019-01177-0

Adv Ther

2020

DOI: 10.1007/s12325-019-01177-0

|View full text |Cite

FGF23-Related Hypophosphataemic Bone Disease

Antonio González-Meneses López

Abstract: Metabolic skeletal dysplasias comprise an extensive group of diseases capable of causing changes, usually progressive, in the bone and are due to hereditary disorders in many cases.

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...

Citation Types

Supporting

Mentioning

Contrasting

Year Published

2022

Publication Types

Select...

Article3

Relationship

Self Cite0

Independent3

Authors

Journals

Cited by 3 publications

(1 citation statement)

References 9 publications

(21 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…As phosphate is critical in many biological processes, including musculoskeletal development, FGF23-dependent reductions in serum phosphorus levels lead to abnormal bone mineralization and poor muscle function [ 4 ]. The resulting musculoskeletal disorders comprise a group of related diseases, of which X-linked hypophosphatemia (XLH) is the most common [ 2 , 3 , 5 ]; the causative gene varies but clinical musculoskeletal characteristics of these diseases are similar, manifesting as rickets in children with open growth plates and osteomalacia in adolescents and adults [ 2 , 4 ].…”

mentioning

confidence: 99%

Safety and Efficacy of Burosumab in Pediatric Patients With X-Linked Hypophosphatemia: A Phase 3/4 Open-Label Trial

Namba

Kubota

Muroya

et al. 2022

Journal of the Endocrine Society

View full text Add to dashboard Cite

Objective Burosumab, an anti-fibroblast growth factor 23 antibody, was recently approved for treatment of X-linked hypophosphatemia (XLH). We evaluated burosumab safety and efficacy in pediatric XLH patients. Methods This open-label, phase 3/4 trial of ≤124 weeks’ duration was conducted at four Japanese medical centers. Fifteen children aged 1–12 years with XLH were included. All had previously been treated with phosphorus or vitamin D. Subcutaneous burosumab was administered every 2 weeks, starting with 0.8 mg/kg, and adjusted based on serum phosphorus levels and any safety concerns (maximum 2 mg/kg). Safety assessments included the frequency of treatment-emergent adverse events (TEAEs). Efficacy of burosumab on biochemical markers, clinical markers of rickets, motor function, and growth was also evaluated. Results The average treatment duration was 121.7 weeks. Frequently reported TEAEs were nasopharyngitis (46.7%), dental caries (40.0%), and influenza (33.3%). At baseline, patients had low serum phosphorus concentrations (2.6 ± 0.3 mg/dL) and low-to-normal 1,25-dihydroxyvitamin D (1,25[OH]2D) concentrations (24.7 ± 12.7 pg/mL), which increased with burosumab treatment and were maintained during the study period. Alkaline phosphatase decreased continuously. At baseline, the mean ± standard deviation total Thacher Rickets Severity Score (RSS) was 1.3 ± 1.2, and four patients (26.7%) had an RSS ≥2.0. Mean Radiographic Global Impression of Change and RSS tended to improve, particularly in patients with higher baseline RSS. There was a trend toward increased 6-minute walk test distance. No apparent changes in growth rate were observed. Conclusions Burosumab has a good safety profile and is effective in pediatric patients with XLH.

show abstract

mentioning

confidence: 99%

Safety and Efficacy of Burosumab in Pediatric Patients With X-Linked Hypophosphatemia: A Phase 3/4 Open-Label Trial

Namba

Kubota

Muroya

et al. 2022

Journal of the Endocrine Society

View full text Add to dashboard Cite

show abstract

Burden of disease of X-linked hypophosphatemia in Japanese and Korean patients: a cross-sectional survey

et al. 2022

View full text Add to dashboard Cite

Cellular and Molecular Alterations Underlying Abnormal Bone Growth in X-Linked Hypophosphatemia

Fuente

García-Bengoa

Fernández-Iglesias

et al. 2022

IJMS

View full text Add to dashboard Cite

X-linked hypophosphatemia (XLH), the most common form of hereditary hypophosphatemic rickets, is caused by inactivating mutations of the phosphate-regulating endopeptidase gene (PHEX). XLH is mainly characterized by short stature, bone deformities and rickets, while in hypophosphatemia, normal or low vitamin D levels and low renal phosphate reabsorption are the principal biochemical aspects. The cause of growth impairment in patients with XLH is not completely understood yet, thus making the study of the growth plate (GP) alterations necessary. New treatment strategies targeting FGF23 have shown promising results in normalizing the growth velocity and improving the skeletal effects of XLH patients. However, further studies are necessary to evaluate how this treatment affects the GP as well as its long-term effects and the impact on adult height.

show abstract

scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.

Contact Info

hi@scite.ai

10624 S. Eastern Ave., Ste. A-614

Henderson, NV 89052, USA

Blog Terms and Conditions API Terms Privacy Policy Contact Cookie Preferences Do Not Sell or Share My Personal Information

Made with 💙 for researchers

Part of the Research Solutions Family.

FGF23-Related Hypophosphataemic Bone Disease

Abstract: Metabolic skeletal dysplasias comprise an extensive group of diseases capable of causing changes, usually progressive, in the bone and are due to hereditary disorders in many cases.

Cited by 3 publications

References 9 publications

Safety and Efficacy of Burosumab in Pediatric Patients With X-Linked Hypophosphatemia: A Phase 3/4 Open-Label Trial

Safety and Efficacy of Burosumab in Pediatric Patients With X-Linked Hypophosphatemia: A Phase 3/4 Open-Label Trial

Burden of disease of X-linked hypophosphatemia in Japanese and Korean patients: a cross-sectional survey

Cellular and Molecular Alterations Underlying Abnormal Bone Growth in X-Linked Hypophosphatemia

Contact Info

Product

Resources

About