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Burosumab in management of X-linked hypophosphataemia: a retrospective cohort study of growth and serum phosphate levels

Walker, Emma Yi Xiu; Lindsay, Timothy Alexander James; Allgrove, Jeremy; Marlais, Matko; Bockenhauer, Detlef; Hayes, Wesley; (2023) Burosumab in management of X-linked hypophosphataemia: a retrospective cohort study of growth and serum phosphate levels. Archives of Disease in Childhood 10.1136/archdischild-2022-324962. (In press). Green open access

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Abstract

BACKGROUND: Burosumab, an antifibroblast growth factor 23 monoclonal antibody, improves rickets severity, symptoms and growth in children with X-linked hypophosphataemia (XLH) followed up to 64 weeks in clinical trials. International dosing guidance recommends targeting normal serum phosphate concentration; however, some children may not achieve this despite maximal dosing. This study compares clinical outcomes in children with XLH on long-term burosumab treatment who achieved normal phosphate versus those who did not. METHODS: Single-centre retrospective review of a large paediatric cohort with XLH treated with burosumab. We evaluated growth and biochemical markers of bone health in those who did compared with those who did not achieve normal plasma phosphate concentration. RESULTS: Fifty-five children with XLH with median age of 11.7 (IQR 6.8-15.5) years were included. 27 (49%) had low plasma phosphate concentration, and 27 (49%) had normal phosphate after a median burosumab treatment duration of 3.3 (IQR 2.6-3.7) years. 1 (2%) did not have a recent phosphate level recorded. No difference in growth was found between normal and abnormal phosphate groups (p=0.9). CONCLUSIONS: Young children with XLH experience sustained growth on long-term burosumab treatment, although without normal plasma phosphate concentration in many. Consideration should be made to changing burosumab dosing recommendations to target normalisation of alkaline phosphatase, as opposed to plasma phosphate concentration.

Type: Article
Title: Burosumab in management of X-linked hypophosphataemia: a retrospective cohort study of growth and serum phosphate levels
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1136/archdischild-2022-324962
Publisher version: https://doi.org/10.1136/archdischild-2022-324962
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Endocrinology, genetics, growth, Life Sciences & Biomedicine, nephrology, paediatrics, Pediatrics, Science & Technology
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Renal Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Biology and Cancer Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10165974
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