ECEESPE2025 Poster Presentations Bone and Mineral Metabolism (112 abstracts)
Non-interventional post-authorisation safety study of burosumab in the treatment of children and adolescents with X-linked hypophosphataemia: second interim analysis
Signe Beck-Nielsen 1 , Gema Ariceta 2 , Maria Luisa Brandi 3 , Karine Briot 4 , Carmen de Lucas Collantes 5 , Francesco Emma 6 , Sandro Giannini 7 , Dieter Haffner 8 , Richard Keen 9 , Elena Levtchenko 10 , Ola Nilsson 11 , 12 , Outi Mäkitie 13 , Zulf Mughal 14 , Dirk Schnabel 15 , Liana Tripto-Shkolnik 16,17 , Carola Zillikens 18 , El Mahdi Benchekroun 19 , Eslam El Tahan 20 , Paul Joos-Vandewalle 21 , Nadine Sawoky 22 & Annemieke Boot 23
1Aarhus University Hospital, Aarhus, Denmark; 2University Hospital Vall d’Hebron, Autonomous University of Barcelona, Barcelona, Spain; 3FIRMO Foundation, Florence, Italy; 4AP-HP, Cochin Hospital, Paris, France; 5Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain; 6Bambino Gesù Children’s Hospital IRCCS, Rome, Italy; 7University of Padova, Padova, Italy; 8Hannover Medical School, Hannover, Germany; 9Royal National Orthopaedic Hospital, Stanmore, United Kingdom; 10Amsterdam University Medical Centre, Amsterdam, Netherlands; 11Karolinska Institutet and University Hospital, Stockholm, Sweden; 12Örebro University and University Hospital, Örebro, Sweden; 13University of Helsinki and Helsinki University Hospital, Helsinki, Finland; 14Al Jalila Children’s Speciality Hospital, Dubai, United Arab Emirates; 15Charitè, University Medicine, Berlin, Germany; 16Tel Aviv University, Tel Aviv, Israel; 17Sheba Medical Center, Tel HaShomer, Israel; 18Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands; 19Kyowa Kirin International Plc, Dubai, United Arab Emirates; 20Kyowa Kirin International Plc, Paris, France; 21Kyowa Kirin Co Ltd, Madrid, Spain; 22Kyowa Kirin International Plc, Galashiels, United Kingdom; 23University Medical Center Groningen, University of Groningen, Groningen, Netherlands
JOINT73
Burosumab, a fully human anti-FGF23 antibody is approved for treating X-linked hypophosphataemia (XLH), a rare, genetic, progressive, deforming bone disease. As part of the burosumab risk management plan, a post-authorisation safety study (PASS) was initiated, utilizing International XLH Registry (NCT03193476) data. The PASS is a 10-year multi-centre, non-interventional, observational study. Primary objectives assessed safety outcomes in children and adolescents with XLH, including participants with chronic kidney disease (CKD). The secondary objective compared burosumab and conventional therapy safety outcomes. Of participants enrolled by the 29-Sep-2023 datacut, 340 received burosumab (Safety Analysis Set [SAF]), 91 received conventional therapy (Cohort SAF [CSAF]). Of SAF participants, 51/340 (15. 0%) had CKD. SAF and CSAF adverse event (AE) reporting were solicited and unsolicited, respectively (Table). Hospitalisation data were separate and independent from AE reporting and not necessarily linked to AEs. Most common AEs in the SAF cohort were ‘Pain in extremity, ’ ‘Headache’ and ‘Arthralgia. ’ Of CSAF participants, most common AEs were ‘Pain in extremity, ’ ‘Headache’ and ‘Abdominal pain. ’ No deaths were reported in either cohort. Hospitalisations (inpatient and outpatient) were common in both cohorts; hyperphosphataemia, ectopic mineralisation and elevated parathyroid hormone were rare. The safety profile of burosumab was consistent with previous reports, with no new safety concerns. The most common AEs were typical of paediatric populations or frequent manifestations of XLH. AE frequency in CKD participants was similar compared with SAF participants. Long-term safety outcomes were similar between the SAF and CSAF cohorts.
| SAF participants | |||||
| CKD* (n = 51) | |||||
| n, (%) | Total (n = 340) | Normal: GFR ≥90 mL/min/1. 73 m2[n = 43] | Mild-to-moderate/severe: GFR ≥30–<90 mL/min/1. 73 m2[n = 6] | Severe CKD-to-kidney failure: GFR <30 mL/min/1. 73 m2 [n = 2] | CSAF participants (n = 91) |
| Any AE | 127 (37. 4) | 14 (32. 6) | 3 (50. 0) | 0 | 20 (22) |
| Possibly/probably related to treatment | 54 (15. 9) | 9 (20. 9) | 1 (16. 7) | 5 (5. 5) | |
| Possibly/probably related to burosumab | 49 (14. 4) | 8 (15. 7) | 0 | ||
| Leading to death | 0 | 0 | |||
| Leading to treatment withdrawal | 2 (0. 6) | 1 (1. 1) | |||
| Possibly/probably related to treatment leading to treatment withdrawal | 0 | 0 | 1 (1. 1) | ||
| Any serious AE | 12 (3. 5) | 1 (2. 3) | 0 | ||
| Possibly/probably related to burosumab | 3 (0. 9) | 0 | |||
| Hospitalisations | 244 (71. 8) | 65 (71. 4) | |||
| Hyperphosphataemia | 1 (0. 3) | 0 | |||
| Elevated parathyroid hormone | 7 (2. 1) | 1 (1. 1) | |||
| Ectopic mineralisation | 5 (1. 5)† | 1 (1. 1) | |||
| *Defined as abnormalities of kidney structure or function, present for >3 months. †Nephrocalcinosis. AE, adverse event; CKD chronic kidney disease; CSAF, cohort SAF; GFR, glomerular filtration rate; SAF, safety analysis set. | |||||
Volume 110
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