{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T07:34:51Z","timestamp":1781508891092,"version":"3.54.1"},"reference-count":24,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2022,1,1]],"date-time":"2022-01-01T00:00:00Z","timestamp":1640995200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Chronic Disease"],"published-print":{"date-parts":[[2022,1]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>\n                      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)\n                      <jats:sub>2<\/jats:sub>\n                      D) synthesis. Disease characteristics include rickets, osteomalacia, odontomalacia, and short stature. Historically, treatment has been oral phosphate and 1,25(OH)\n                      <jats:sub>2<\/jats:sub>\n                      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 &gt;1 year.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Data collection started on 24 April 2019. The expected date of the final study report is 31 December 2028, with two interim reports.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Registry identification:<\/jats:title>\n                    <jats:p>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 ).<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/20406223221117471","type":"journal-article","created":{"date-parts":[[2022,9,5]],"date-time":"2022-09-05T03:17:55Z","timestamp":1662347875000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":10,"title":["Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8741-0592","authenticated-orcid":false,"given":"Maria Luisa","family":"Brandi","sequence":"first","affiliation":[{"name":"FIRMO Foundation, Via San Gallo 123, 50100 Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gema","family":"Ariceta","sequence":"additional","affiliation":[{"name":"Department of Pediatric Nephrology, Hospital Universitario Vall d\u2019Hebron, Universidad Aut\u00f3noma de Barcelona, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Signe Sparre","family":"Beck-Nielsen","sequence":"additional","affiliation":[{"name":"Centre for Rare Diseases, Department of Paediatrics, Aarhus University Hospital, Aarhus, Denmark"},{"name":"Department of Clinical Medicine, Aarhus University, Aarhus, Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annemieke M.","family":"Boot","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karine","family":"Briot","sequence":"additional","affiliation":[{"name":"APHP, Department of Rheumatology, Cochin Hospital, Universit\u00e9 de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen","family":"de Lucas Collantes","sequence":"additional","affiliation":[{"name":"Department of Nephrology, Hospital Infantil Universitario Ni\u00f1o Jes\u00fas, Universidad Aut\u00f3noma de Madrid, Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Emma","sequence":"additional","affiliation":[{"name":"Division of Nephrology, Bambino Ges\u00f9 Children\u2019s Hospital IRCCS, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandro","family":"Giannini","sequence":"additional","affiliation":[{"name":"Department of Medicine, Clinica Medica 1, University of Padua, Padua, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dieter","family":"Haffner","sequence":"additional","affiliation":[{"name":"Department of Pediatric Kidney, Liver and Metabolic Diseases, Children\u2019s Hospital, Hannover Medical School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Keen","sequence":"additional","affiliation":[{"name":"Metabolic Unit, Royal National Orthopaedic Hospital NHS Trust, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Levtchenko","sequence":"additional","affiliation":[{"name":"Department of Pediatric Nephrology and Development and Regeneration, University Hospitals Leuven, University of Leuven, Leuven, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Outi","family":"M\u04d3kitie","sequence":"additional","affiliation":[{"name":"Pediatric Endocrinology, Children\u2019s Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ola","family":"Nilsson","sequence":"additional","affiliation":[{"name":"Division of Pediatric Endocrinology, Department of Women\u2019s and Children\u2019s Health, Karolinska Institutet and University Hospital, Stockholm, Sweden"},{"name":"Department of Medical Sciences and Department of Pediatrics, \u00d6rebro University and University Hospital, \u00d6rebro, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dirk","family":"Schnabel","sequence":"additional","affiliation":[{"name":"Center for Chronic Sick Children, Pediatric Endocrinology, Charit\u00e8, University Medicine, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liana","family":"Tripto-Shkolnik","sequence":"additional","affiliation":[{"name":"Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel"},{"name":"Division of Endocrinology, Diabetes and Metabolism, Sheba Medical Center, Tel-Hashomer, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Carola","family":"Zillikens","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Erasmus MC Bone Center \u2013 Erasmus University Medical Center, Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Liu","sequence":"additional","affiliation":[{"name":"Kyowa Kirin International, Marlow, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alina","family":"Tudor","sequence":"additional","affiliation":[{"name":"Kyowa Kirin International, Marlow, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Zulf","family":"Mughal","sequence":"additional","affiliation":[{"name":"Department of Paediatric Endocrinology, Royal Manchester Children\u2019s Hospital, Manchester, UK"},{"name":"Faculty of Biology, Medicine & Health, University of Manchester, Manchester, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,9,5]]},"reference":[{"key":"bibr1-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1507\/endocrj.EJ15-0275"},{"key":"bibr2-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1530\/EJE-15-0515"},{"key":"bibr3-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1210\/clinem\/dgz203"},{"key":"bibr4-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1530\/EJE-08-0818"},{"key":"bibr5-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1038\/s41581-019-0152-5"},{"key":"bibr6-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-019-1014-8"},{"issue":"11","key":"bibr7-20406223221117471","first-page":"4075","volume":"81","author":"Whyte MP","year":"1996","journal-title":"J Clin Endocrinol Metab"},{"key":"bibr8-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-018-0147-6"},{"key":"bibr9-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01434-4"},{"key":"bibr10-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1002\/jbmr.340"},{"key":"bibr11-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1210\/js.2018-00365"},{"key":"bibr12-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1007\/s004310050962"},{"key":"bibr13-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1210\/jc.2003-030036"},{"key":"bibr14-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1007\/s00467-011-2046-z"},{"key":"bibr15-20406223221117471","unstructured":"European Medicines Agency. Crystvita summary of product characteristics, https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/crysvita (2021, accessed 4 August 2021)."},{"key":"bibr16-20406223221117471","unstructured":"European Medicines Agency. New medicine for rare bone disease, https:\/\/www.ema.europa.eu\/en\/news\/new-medicine-rare-bone-disease (2017, accessed 5 August 2021)."},{"key":"bibr17-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1172\/JCI72829"},{"key":"bibr18-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1714641"},{"key":"bibr19-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1007\/s00223-019-00568-3"},{"key":"bibr20-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01872-8"},{"key":"bibr21-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-9-81"},{"key":"bibr22-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.38413"},{"key":"bibr23-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1080\/14740338.2018.1437144"},{"key":"bibr24-20406223221117471","doi-asserted-by":"publisher","DOI":"10.1183\/23120541.00084-2018"}],"container-title":["Therapeutic Advances in Chronic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406223221117471","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/20406223221117471","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406223221117471","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T23:19:13Z","timestamp":1777591153000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/20406223221117471"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,1]]},"references-count":24,"alternative-id":["10.1177\/20406223221117471"],"URL":"https:\/\/doi.org\/10.1177\/20406223221117471","relation":{},"ISSN":["2040-6223","2040-6231"],"issn-type":[{"value":"2040-6223","type":"print"},{"value":"2040-6231","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,1]]},"article-number":"20406223221117471"}}