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Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries

Given the relatively recent introduction of burosumab in the management of X-linked hypophosphatemia (XLH), there is limited real-world data to guide its use in clinical practice. As a group of European physicians experienced with burosumab treatment in clinical practice, we convened with the object...

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Autores principales: Mughal, M. Zulf, Baroncelli, Giampiero I., de Lucas-Collantes, Carmen, Linglart, Agnès, Magnolato, Andrea, Raimann, Adalbert, Santos, Fernando, Schnabel, Dirk, Shaw, Nick, Nilsson, Ola
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9928183/
https://www.ncbi.nlm.nih.gov/pubmed/36798486
http://dx.doi.org/10.3389/fendo.2022.1034580
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author Mughal, M. Zulf
Baroncelli, Giampiero I.
de Lucas-Collantes, Carmen
Linglart, Agnès
Magnolato, Andrea
Raimann, Adalbert
Santos, Fernando
Schnabel, Dirk
Shaw, Nick
Nilsson, Ola
author_facet Mughal, M. Zulf
Baroncelli, Giampiero I.
de Lucas-Collantes, Carmen
Linglart, Agnès
Magnolato, Andrea
Raimann, Adalbert
Santos, Fernando
Schnabel, Dirk
Shaw, Nick
Nilsson, Ola
author_sort Mughal, M. Zulf
collection PubMed
description Given the relatively recent introduction of burosumab in the management of X-linked hypophosphatemia (XLH), there is limited real-world data to guide its use in clinical practice. As a group of European physicians experienced with burosumab treatment in clinical practice, we convened with the objective of sharing these practice-based insights on the use of burosumab in children and adolescents with XLH. We attended two virtual meetings, then discussed key questions via Within3, a virtual online platform. Points of discussion related to patient selection criteria, burosumab starting dose, dose titration and treatment monitoring. Our discussions revealed that criteria for selecting children with XLH varied across Europe from all children above 1 year to only children with overt rickets despite conventional treatment being eligible. We initiated burosumab dosing according to guidance in the Summary of Product Characteristics, an international consensus statement from 2019 and local country guidelines. Dose titration was primarily guided by serum phosphate levels, with some centers also using the ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate (TmP/GFR). We monitored response to burosumab treatment clinically (growth, deformities, bone pain and physical functioning), radiologically (rickets and deformities) and biochemically (serum phosphate, alkaline phosphatase, 1,25-dihydroxyvitamin D, 25-hydroxyvitamin D, urine calcium-creatinine ratio and TmP/GFR). Key suggestions made by our group were initiation of burosumab treatment in children as early as possible, from the age of 1 year, particularly in those with profound rickets, and a need for clinical studies on continuation of burosumab throughout adolescence and into adulthood.
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spelling pubmed-99281832023-02-15 Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries Mughal, M. Zulf Baroncelli, Giampiero I. de Lucas-Collantes, Carmen Linglart, Agnès Magnolato, Andrea Raimann, Adalbert Santos, Fernando Schnabel, Dirk Shaw, Nick Nilsson, Ola Front Endocrinol (Lausanne) Endocrinology Given the relatively recent introduction of burosumab in the management of X-linked hypophosphatemia (XLH), there is limited real-world data to guide its use in clinical practice. As a group of European physicians experienced with burosumab treatment in clinical practice, we convened with the objective of sharing these practice-based insights on the use of burosumab in children and adolescents with XLH. We attended two virtual meetings, then discussed key questions via Within3, a virtual online platform. Points of discussion related to patient selection criteria, burosumab starting dose, dose titration and treatment monitoring. Our discussions revealed that criteria for selecting children with XLH varied across Europe from all children above 1 year to only children with overt rickets despite conventional treatment being eligible. We initiated burosumab dosing according to guidance in the Summary of Product Characteristics, an international consensus statement from 2019 and local country guidelines. Dose titration was primarily guided by serum phosphate levels, with some centers also using the ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate (TmP/GFR). We monitored response to burosumab treatment clinically (growth, deformities, bone pain and physical functioning), radiologically (rickets and deformities) and biochemically (serum phosphate, alkaline phosphatase, 1,25-dihydroxyvitamin D, 25-hydroxyvitamin D, urine calcium-creatinine ratio and TmP/GFR). Key suggestions made by our group were initiation of burosumab treatment in children as early as possible, from the age of 1 year, particularly in those with profound rickets, and a need for clinical studies on continuation of burosumab throughout adolescence and into adulthood. Frontiers Media S.A. 2023-01-31 /pmc/articles/PMC9928183/ /pubmed/36798486 http://dx.doi.org/10.3389/fendo.2022.1034580 Text en Copyright © 2023 Mughal, Baroncelli, de Lucas-Collantes, Linglart, Magnolato, Raimann, Santos, Schnabel, Shaw and Nilsson https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Endocrinology
Mughal, M. Zulf
Baroncelli, Giampiero I.
de Lucas-Collantes, Carmen
Linglart, Agnès
Magnolato, Andrea
Raimann, Adalbert
Santos, Fernando
Schnabel, Dirk
Shaw, Nick
Nilsson, Ola
Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title_full Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title_fullStr Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title_full_unstemmed Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title_short Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries
title_sort burosumab for x-linked hypophosphatemia in children and adolescents: opinion based on early experience in seven european countries
topic Endocrinology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9928183/
https://www.ncbi.nlm.nih.gov/pubmed/36798486
http://dx.doi.org/10.3389/fendo.2022.1034580
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