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Post-authorisation ...
Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia : rationale and description
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- Brandi, Maria Luisa (författare)
- FIRMO Foundation, Florence, Italy
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- Ariceta, Gema (författare)
- Department of Pediatric Nephrology, Hospital Universitario Vall d'Hebron, Universidad Autónoma de Barcelona, Barcelona, Spain
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- Beck-Nielsen, Signe Sparre (författare)
- Centre for Rare Diseases, Department of Paediatrics, Aarhus University Hospital, Aarhus, Denmark; Department of Clinical Medicine, Aarhus University, Aarhus, Denmark
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- Boot, Annemieke M. (författare)
- Department of Pediatric Endocrinology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
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- Briot, Karine (författare)
- APHP, Department of Rheumatology, Cochin Hospital, Université de Paris, Paris, France
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- de Lucas Collantes, Carmen (författare)
- Department of Nephrology, Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain
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- Emma, Francesco (författare)
- Division of Nephrology, Bambino Gesù Children's Hospital IRCCS, Rome, Italy
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- Giannini, Sandro (författare)
- Department of Medicine, University of Padua, Padua, Italy
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- Haffner, Dieter (författare)
- Department of Pediatric Kidney, Liver and Metabolic Diseases, Children's Hospital, Hannover Medical School, Hannover, Germany
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- Keen, Richard (författare)
- Metabolic Unit, Royal National Orthopaedic Hospital NHS Trust, London, UK
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- Levtchenko, Elena (författare)
- Department of Pediatric Nephrology and Development and Regeneration, University Hospitals Leuven, University of Leuven, Leuven, Belgium
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- Mӓkitie, Outi (författare)
- Karolinska Institutet
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- Nilsson, Ola, 1970- (författare)
- Karolinska Institutet,Örebro universitet,Institutionen för medicinska vetenskaper,Division of Pediatric Endocrinology, Department of Women's and Children's Health, Karolinska Institutet and University Hospital, Stockholm, Sweden; Department of Medical Sciences and Department of Pediatrics, University Hospital, Örebro, Sweden
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- Schnabel, Dirk (författare)
- Center for Chronic Sick Children, Pediatric Endocrinology, Charitè, University Medicine, Berlin, Germany
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- Tripto-Shkolnik, Liana (författare)
- Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel; Division of Endocrinology, Diabetes and Metabolism, Sheba Medical Center, Tel-Hashomer, Israel
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- Zillikens, M. Carola (författare)
- Department of Internal Medicine, Erasmus MC Bone Center - Erasmus University Medical Center, Rotterdam, The Netherlands
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- Liu, Jonathan (författare)
- Kyowa Kirin International, Marlow, UK
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- Tudor, Alina (författare)
- Kyowa Kirin International, Marlow, UK
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- Mughal, M. Zulf (författare)
- Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK; Faculty of Biology, Medicine & Health, University of Manchester, Manchester, UK
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(creator_code:org_t)
- 2022-09-05
- 2022
- Engelska.
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Ingår i: Therapeutic advances in chronic disease. - : Sage Publications. - 2040-6223 .- 2040-6231. ; 13
- Relaterad länk:
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https://doi.org/10.1...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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http://kipublication...
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Abstract
Ämnesord
Stäng
- Background: 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)2D) synthesis. Disease characteristics include rickets, osteomalacia, odontomalacia, and short stature. Historically, treatment has been oral phosphate and 1,25(OH)2D 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.Methods: 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.Results: Data collection started on 24 April 2019. The expected date of the final study report is 31 December 2028, with two interim reports.Conclusion: 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.Registry identification: The International XLH Registry is registered with clinicaltrials.gov as NCT03193476 (https://clinicaltrials.gov/ct2/show/NCT03193476), and the PASS is registered with the European Union electronic Register of Post-Authorisation Studies as EUPAS32190 (http://www.encepp.eu/encepp/viewResource.htm?id=32191).
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Urologi och njurmedicin (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Urology and Nephrology (hsv//eng)
Nyckelord
- X-linked hypophosphataemia (XLH)
- burosumab
- patient registry
- phosphate
- post-authorisation safety study (PASS)
- rare bone disease
- real-world evidence
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
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Till lärosätets databas
- Av författaren/redakt...
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Brandi, Maria Lu ...
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Ariceta, Gema
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Beck-Nielsen, Si ...
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Boot, Annemieke ...
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Briot, Karine
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de Lucas Collant ...
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visa fler...
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Emma, Francesco
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Giannini, Sandro
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Haffner, Dieter
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Keen, Richard
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Levtchenko, Elen ...
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Mӓkitie, Outi
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Nilsson, Ola, 19 ...
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Schnabel, Dirk
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Tripto-Shkolnik, ...
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Zillikens, M. Ca ...
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Liu, Jonathan
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Tudor, Alina
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Mughal, M. Zulf
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- MEDICIN OCH HÄLSOVETENSKAP
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Therapeutic adva ...
- Av lärosätet
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Örebro universitet
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Karolinska Institutet