Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia

Frontiers in Pediatrics
Silvia Martín RamosF Santos

Abstract

X-linked hypophosphatemia (XLH) causes significant burden in pediatric patients in spite of maintained treatment with phosphate supplements and vitamin D derivatives. Administration of burosumab has shown promising results in clinical trial but studies assessing its effect in the everyday practice are missing. With this aim, we analyzed the response to one-year treatment with burosumab, injected subcutaneously at 0.8 mg/kg every 2 weeks, in five children (three females) aged from 6 to 16 years, with genetically confirmed XLH. Patients were being treated with phosphate and vitamin D analogs until the beginning of burosumab treatment. In all children, burosumab administration led to normalization of serum phosphate in association with marked increase of tubular reabsorption of phosphate and reduction of elevated serum alkaline phosphatase levels. Baseline height of patients, from -3.56 to -0.46 SD, increased in the three prepubertal children (+0.84, +0.89, and +0.16 SD) during burosumab treatment. Growth improvement was associated with reduction in body mass index (-1.75, -1.47, and -0.17 SD, respectively), suggesting a salutary effect of burosumab on physical activity and body composition. Burosumab was well-tolerated, mild loca...Continue Reading

References

Dec 2, 2010·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Miroslav ZivičnjakUNKNOWN Hypophosphatemic Rickets Study Group of Arbeitsgemeinschaft für Pädiatrische Endokrinologie and Gesellschaft für Pädiatrisch
May 4, 2011·Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research·Thomas O CarpenterKarl L Insogna
Dec 15, 2011·Journal of Bone and Mineral Metabolism·Thomas O Carpenter
Nov 28, 2012·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Fernando SantosFlor A Ordoñez
Feb 20, 2014·Endocrine Connections·Agnès LinglartPol Harvengt
Dec 11, 2014·European Journal of Pediatrics·Vrinda SaraffWolfgang Högler
Jan 29, 2017·Reviews in Endocrine & Metabolic Disorders·R FuenteF Santos
Oct 24, 2017·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Nadine MeyerhoffUNKNOWN Hypophosphatemic Rickets Study Group of the “Deutsche Gesellschaft für Kinderendokrinologie und -diabetologie” and “Gesellsc
May 24, 2018·The New England Journal of Medicine·Thomas O CarpenterAnthony A Portale
Nov 21, 2018·Pediatric Clinics of North America·Martin Bitzan, Paul R Goodyer
Nov 22, 2018·Drugs & Therapy Perspectives : for Rational Drug Selection and Use·Katherine A Lyseng-Williamson
May 10, 2019·Nature Reviews. Nephrology·Dieter HaffnerAgnès Linglart
Jul 2, 2019·Journal of the Endocrine Society·Alison SkrinarJavier San Martin

❮ Previous
Next ❯

Citations

Sep 16, 2021·Journal of Pediatric Endocrinology & Metabolism : JPEM·Ayfer AlikasifogluMehmet Alikasifoglu

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.