Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description
Autor (es)
Brandi, Maria Luisa; Ariceta, Gema; Beck-Nielsen, Signe Sparre; Boot, Annemieke M.; Briot, Karine; Lucas Collantes, María del Carmen de; Emma, Francesco; Giannini, Sandro; Haffner, Dieter; Keen, Richard; Levtchenko, Elena; Mӓkitie, Outi; Nilsson, Ola; Schnabel, Dirk; Tripto-Shkolnik, Liana; Zillikens, M. Carola; Liu, Jonathan; Tudor, Alina; Mughal, M. ZulfEntidad
UAM. Departamento de MedicinaEditor
Sage PublicationsFecha de edición
2022-09-05Cita
10.1177/20406223221117471
Therapeutic Advances in Chronic Disease 13 (2022): 1-13
ISSN
2040-6231 (online); 2040-6223 (print)DOI
10.1177/20406223221117471Versión del editor
https://doi.org/10.1177/20406223221117471Materias
burosumab; patient registry; phosphate; post-authorisation safety study (PASS); rare bone disease; real-world evidence; X-linked hypophosphataemia (XLH); MedicinaDerechos
© The Author(s), 2022Resumen
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
Lista de ficheros
Google Scholar:Brandi, Maria Luisa
-
Ariceta, Gema
-
Beck-Nielsen, Signe Sparre
-
Boot, Annemieke M.
-
Briot, Karine
-
Lucas Collantes, María del Carmen de
-
Emma, Francesco
-
Giannini, Sandro
-
Haffner, Dieter
-
Keen, Richard
-
Levtchenko, Elena
-
Mӓkitie, Outi
-
Nilsson, Ola
-
Schnabel, Dirk
-
Tripto-Shkolnik, Liana
-
Zillikens, M. Carola
-
Liu, Jonathan
-
Tudor, Alina
-
Mughal, M. Zulf
Lista de colecciones del ítem
Registros relacionados
Mostrando ítems relacionados por título, autor, creador y materia.
-
The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data
Ariceta, Gema; Beck-Nielsen, Signe Sparre; Boot, Annemieke M.; Brandi, Maria Luisa; Briot, Karine; Lucas Collantes, María del Carmen de; Emma, Francesco; Giannini, Sandro; Hafner, Dieter; Keen, Richard; Levtchenko, Elena; Outi, Mäkitie; Mughal, M. Zulf; Nilsson, Ola; Schnabe, Dirk; Tripto-Shkolnik, Liana; Liu, Jonathan; Williams, Angela; Wood, Sue; Carola Zillikens, M.
2023-09-27 -
Fermi observations of γ-ray emission from the moon
Abdo, Aous A.; Ackermann, Markus; Ajello, Marco; Atwoo, W. B.; Baldini, Luca; Ballet, Jean; Barbiellini, Guido; Bastieri, Denis; Bechtol, Keith C.; Bellazzini, Ronaldo; Berenji, Bijan; Blandford, Roger D.; Bonamente, Emanuele; Borgland, AW W.; Bottacini, Eugenio; Bouvier, Aurelien; Bregeon, Johan; Brigida, Monica; Bruel, Ph; Buehler, Rolf; Buson, S.; Caliandro, G. Andrea; Cameron, Robert A.; Caraveo, Patrizia A.; Casandjian, Jean Marc; Cecchi, Claudia; Charles, E.; Chekhtman, Alexandre; Chiang, James; Ciprini, Stefano; Claus, Richard; Cohen-Tanugi, Johann; Conrad, Jan; Cutini, Sara; D'Ammando, Filippo; De Angelis, Alessandro D.; De Palma, Francesco; Dermer, Charles D.; Digel, Seth W.; Do Couto E Silva, Eduardo; Drell, PS S.; Drlica-Wagner, Alex; Dubois, Richard; Favuzzi, C.; Fegan, S. J.; Focke, Warren B.; Fortin, Pascal D.; Fukazawa, Yasushi; Funk, Stefan; Fusco, P. G.; Gargano, Fabio; Gehrels, Neil A.; Germani, Stefano; Giglietto, Nicola; Giommi, Paolo; Giordano, Francesco; Giroletti, Marcello; Glanzman, T.; Godfrey, Gary L.; Gómez-Vargas, Germán A.; Grenier, Isabelle A.; Grove, J. Eric; Guiriec, Sylvain; Hadasch, Daniela; Hays, Elizabeth; Hill, Adam B.; Horan, Deirdre; Hou, X.; Hughes, Richard E.; Iafrate, Giulia; Jackson, Miranda S.; Jóhannesson, Gudlaugur; Johnson, Arne S.; Kamae, Tsuneyoshi; Katagiri, Hideaki; Kataoka, Jun; Knödlseder, Jürgen; Kuss, Michael W.; Lande, Joshua; Larsson, Stefan; Latronico, Luca; Lemoine-Goumard, Marianne; Longo, Francesco; Loparco, Francesco; Lott, Benoît; Lovellette, Michael N.; Lubrano, Pasquale; Mazziotta, Mario Nicola; McEnery, Julie E.; Méhault, Jérémie; Michelson, Peter F.; Mitthumsiri, W.; Mizuno, Tsunefumi; Moiseev, Alexander A.; Monte, Christian Federico Perez; Monzani, Maria Elena; Morselli, Aldo; Moskalenko, Igor V.; Murgia, Simona; Naumann-Godó, Melitta; Nolan, Patrick L.; Norris, Jay P.; Nuss, Eric; Ohno, Masanori; Ohsugi, Takashi; Okumura, Akira; Omodei, Nicola; Orienti, Monica; Orlando, Elena; Ormes, Jonathan F.; Ozaki, Masanobu; Paneque, David; Panetta, Julian H.; Parent, Damien; Pesce-Rollins, Melissa; Pierbattista, Marco; Piron, Frédéric; Pivato, G.; Poon, H.; Porter, Troy A.; Prokhorov, Dmitrii A.; Rainò, Silvia; Rando, Riccardo; Razzano, Massimiliano; Razzaque, Soebur; Reimer, Anita; Reimer, Olaf L.; Reposeur, Th; Rochester, Leon S.; Roth, Markus; Sadrozinski, Hartmut F W; Sanchez, David A.; Sbarra, Ca; Schalk, Terry L.; Sgrò, Carmelo; Share, Gerald H.; Siskind, Eric J.; Spandre, Gloria; Spinelli, P. F.; Stawarz., Łukasz; Takahashi, Hiromitsu; Tanaka, Takaaki; Thayer, J. G.; Thayer, J. B.; Thompson, David J.; Tibaldo, Luigi; Tinivella, M.; Torres, Diego F.; Tosti, Gino; Troja, Eleonora; Uchiyama, Yasunobu; Usher, T. L.; Vandenbroucke, Justin A.; Vasileiou, Vlasios; Vianello, Giacomo; Vítale, Vincenzo; Waite, A. P.; Wang, Peng; Winer, Brian L.; Wood, Dan L.; Wood, Kent S.; Yang, Zongchang; Zimmer, S.
2012-10-20