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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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-01-01
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| Series: | Frontiers in Endocrinology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fendo.2022.1034580/full |
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| author | M. Zulf Mughal M. Zulf Mughal Giampiero I. Baroncelli Carmen de Lucas-Collantes Agnès Linglart Agnès Linglart Andrea Magnolato Adalbert Raimann Adalbert Raimann Fernando Santos Dirk Schnabel Nick Shaw Ola Nilsson Ola Nilsson |
| author_facet | M. Zulf Mughal M. Zulf Mughal Giampiero I. Baroncelli Carmen de Lucas-Collantes Agnès Linglart Agnès Linglart Andrea Magnolato Adalbert Raimann Adalbert Raimann Fernando Santos Dirk Schnabel Nick Shaw Ola Nilsson Ola Nilsson |
| author_sort | M. Zulf Mughal |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-04-10T18:58:21Z |
| format | Article |
| id | doaj.art-cf31ca10d592471fad91e3725e089c67 |
| institution | Directory Open Access Journal |
| issn | 1664-2392 |
| language | English |
| last_indexed | 2024-04-10T18:58:21Z |
| publishDate | 2023-01-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Endocrinology |
| spelling | doaj.art-cf31ca10d592471fad91e3725e089c672023-01-31T14:12:00ZengFrontiers Media S.A.Frontiers in Endocrinology1664-23922023-01-011310.3389/fendo.2022.10345801034580Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countriesM. Zulf Mughal0M. Zulf Mughal1Giampiero I. Baroncelli2Carmen de Lucas-Collantes3Agnès Linglart4Agnès Linglart5Andrea Magnolato6Adalbert Raimann7Adalbert Raimann8Fernando Santos9Dirk Schnabel10Nick Shaw11Ola Nilsson12Ola Nilsson13Department of Paediatric Endocrinology and Metabolic Bone Diseases, Royal Manchester Children’s Hospital, Manchester, United KingdomThe Faculty of Biology, Medicine, and Health, University of Manchester, Manchester, United KingdomDivision of Pediatrics, Endocrine Unit, ERN-BOND Representative, Department of Obstetrics, Gynecology and Pediatrics, University-Hospital, Pisa, ItalyServicio Nefrología, Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid, Madrid, SpainAP-HP, Endocrinology and Diabetes for Children, Reference Center for Rare Disorders of Calcium and Phosphate Metabolism, Filière OSCAR, Bicêtre Paris Saclay Hospital, Paris, FrancePlatform of Expertise for Rare Disorders, INSERM, Physiologie et Physiopathologie Endocriniennes, Paris Saclay University, Paris, FranceDepartment of Pediatrics, Institute for Maternal and Child Health – IRCCS “Burlo Garofolo”, Trieste, ItalyDepartment of Pediatrics and Adolescent Medicine, Division of Pediatric Pulmonology, Allergology and Endocrinology, Medical University of Vienna, Vienna, AustriaVienna Bone and Growth Center, Vienna, Austria0Hospital Universitario Central de Asturias (HUCA), University of Oviedo, Oviedo, Spain1Center for Chronic Sick Children, Pediatric Endocrinology, Charitè, University Medicine, Berlin, Germany2Department of Endocrinology and Diabetes, Birmingham Women’s and Children’s NHS Foundation Trust, Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom3Division of Pediatric Endocrinology, Department of Women’s and Children’s Health , Karolinska Institutet and University Hospital, Stockholm, Sweden4School of Medical Sciences, Department of Pediatrics, Örebro University and University Hospital, Örebro, SwedenGiven 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.https://www.frontiersin.org/articles/10.3389/fendo.2022.1034580/fullXLHricketsburosumabchildrenadolescentsgrowth plate closure |
| spellingShingle | M. Zulf Mughal M. Zulf Mughal Giampiero I. Baroncelli Carmen de Lucas-Collantes Agnès Linglart Agnès Linglart Andrea Magnolato Adalbert Raimann Adalbert Raimann Fernando Santos Dirk Schnabel Nick Shaw Ola Nilsson Ola Nilsson Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries Frontiers in Endocrinology XLH rickets burosumab children adolescents growth plate closure |
| 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 | XLH rickets burosumab children adolescents growth plate closure |
| url | https://www.frontiersin.org/articles/10.3389/fendo.2022.1034580/full |
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