Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia : rationale and description
Brandi, Maria Luisa (FIRMO Foundation (Itàlia))
Ariceta Iraola, Gema (Hospital Universitari Vall d'Hebron)
Beck-Nielsen, Signe (Aarhus University Hospital (Aarhus, Dinamarca))
Boot, Annemieke (University of Groningen)
Briot, Karine (Université de Paris)
de Lucas-Collantes, Carmen (Universidad Autónoma de Madrid)
Emma, Francesco (Bambino Gesù Children's Hospital IRCCS (Itàlia))
Giannini, Sandro (University of Padua)
Haffner, Dieter (Children's Hospital (Alemanya))
Keen, Richard (Royal National Orthopaedic Hospital NHS Trust (Regne Unit))
Levtchenko, Elena (University Hospitals Leuven (Bèlgica))
Mӓkitie, Outi (Helsinki University Hospital)
Nilsson, Ola (Karolinska Institutet and University Hospital)
Schnabel, Dirk (Charitè, University Medicine)
Tripto-Shkolnik, Liana (Tel-Aviv University)
Zillikens, M. Carola (Department of Internal Medicine, Erasmus MC Bone Center - Erasmus University Medical Center, Rotterdam (Holanda))
Liu, Jonathan (Kyowa Kirin International (Regne Unit))
Tudor, Alina (Kyowa Kirin International (Regne Unit))
Mughal, M. Zulf (Royal Manchester Children's Hospital)
Universitat Autònoma de Barcelona

Data:	2022
Resum:	X-linked hypophosphataemia (XLH) is a rare, inherited, phosphate-wasting disorder that elevates fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D (1,25(OH)D) synthesis. Disease characteristics include rickets, osteomalacia, odontomalacia, and short stature. Historically, treatment has been oral phosphate and 1,25(OH)D supplements. However, these treatments do not correct the primary pathogenic mechanism or treat all symptoms and can be associated with adverse effects. Burosumab is a recombinant human immunoglobulin G1 monoclonal antibody against FGF23, approved for treating XLH in several geographical regions, including Europe and Israel. Burosumab restores normal serum phosphate levels, minimising the clinical consequences of XLH. Safety data on long-term treatment with burosumab are lacking owing to the rarity of XLH. This post-authorisation safety study (PASS) aims to evaluate the safety outcomes in patients aged >1 year. The PASS is a 10-year retrospective and prospective cohort study utilising data from the International XLH Registry (NCT03193476), which includes standard diagnostic and monitoring practice data at participating centres. The PASS aims to evaluate frequency and severity of safety outcomes, frequency and outcomes of pregnancies in female patients, and safety outcomes in patients with mild to moderate kidney disease at baseline, in children, adolescents and adults treated with burosumab for XLH. It is expected that there will be at least 400 patients who will be administered burosumab. Data collection started on 24 April 2019. The expected date of the final study report is 31 December 2028, with two interim reports. This PASS will provide data on the long-term safety of burosumab treatment for XLH patients and describe safety outcomes for patients receiving burosumab contrasted with those patients receiving other XLH treatments, to help inform the future management of XLH patients. The PASS will be the largest real-world safety study of burosumab. The International XLH Registry is registered with clinicaltrials. gov as NCT03193476 (), and the PASS is registered with the European Union electronic Register of Post-Authorisation Studies as EUPAS32190 ().
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Burosumab ; Patient registry ; Phosphate ; Post-authorisation safety study (PASS) ; Rare bone disease ; Real-world evidence ; X-linked hypophosphataemia (XLH)
Publicat a:	Therapeutic Advances in Chronic Disease, Vol. 13 (september 2022) , ISSN 2040-6231

DOI: 10.1177/20406223221117471
PMID: 36082134

13 p, 587.1 KB

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