{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T00:28:46Z","timestamp":1786580926215,"version":"build-2736575974"},"reference-count":47,"publisher":"The Endocrine Society","issue":"9","license":[{"start":{"date-parts":[[2026,3,31]],"date-time":"2026-03-31T00:00:00Z","timestamp":1774915200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004095","name":"Kyowa Kirin Co., Ltd","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004095","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Kyowa Kirin International plc"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,8,13]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Context<\/jats:title>\n                    <jats:p>X-linked hypophosphatemia (XLH) is a rare, genetic, progressive, lifelong disorder manifest by impaired growth and disproportionate short stature. Burosumab, a monoclonal antibody against fibroblast growth factor 23, is approved for treating patients with XLH.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To understand the impact of burosumab treatment on growth in a real-world setting.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>Interim data from 3 ongoing, real-world observational studies (NCT03651505, NCT03193476, and NCT03745521), were unified into a single study (APEX).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Setting<\/jats:title>\n                    <jats:p>Outpatient clinics.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Patients<\/jats:title>\n                    <jats:p>Children aged 2-17 years with XLH.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Intervention(s)<\/jats:title>\n                    <jats:p>Subcutaneous burosumab vs oral phosphate salts and active vitamin D or no treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Main Outcome Measure(s)<\/jats:title>\n                    <jats:p>Retrospective and prospective height data from children enrolled in APEX were analyzed to provide long-term estimation of the impact of treatment on growth. Growth and height were estimated by a mixed regression model with separate models for children and adolescents.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>In total, 641 participants were analyzed (402 female) with 498 burosumab-treated and 143 burosumab na\u00efve. Median (interquartile range [IQR]) enrollment ages were 8 (5-12) years for burosumab-treated and 10 (4-14) years for burosumab-na\u00efve participants. Burosumab-treated participants experienced improved growth over burosumab-na\u00efve participants (additional growth velocity: 0.085 Z-score\/year for children [P &amp;lt; .0001]; 0.121 Z-score\/year for adolescents [P &amp;lt; .0001]). Modeling predicted greater adult height among burosumab-treated participants.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Burosumab had a robust, positive association with improved growth outcomes in male and female children and adolescents with XLH. Modeling based on a median of 3.3 years of follow-up predicted burosumab can support improvement of growth that will likely result in greater adult height.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1210\/clinem\/dgag136","type":"journal-article","created":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T11:32:03Z","timestamp":1774870323000},"page":"2513-2524","source":"Crossref","is-referenced-by-count":0,"title":["Real-world impact of treatment on growth in children with X-linked hypophosphatemia"],"prefix":"10.1210","volume":"111","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3610-3469","authenticated-orcid":false,"given":"Seiji","family":"Fukumoto","sequence":"first","affiliation":[{"name":"Department of Medicine, Tamaki-Aozora Hospital , Tokushima 779-3125 ,","place":["Japan"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9601-7813","authenticated-orcid":false,"given":"Dieter","family":"Haffner","sequence":"additional","affiliation":[{"name":"Department for Pediatric Kidney, Liver, Metabolic and Neurological Diseases, Hannover Medical School , 30625 Hannover ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik A","family":"Imel","sequence":"additional","affiliation":[{"name":"Departments of Medicine and Pediatrics, Indiana University School of Medicine , Indianapolis, IN 46202 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6517-8825","authenticated-orcid":false,"given":"Keiichi","family":"Ozono","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Center for Promoting Treatment of Intractable Diseases, ISEIKAI International General Hospital , Osaka 530-0052 ,","place":["Japan"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9309-6248","authenticated-orcid":false,"given":"Maria Luisa","family":"Brandi","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, University Vita-Salute San Raffaele , 20132 Milan ,","place":["Italy"]},{"name":"FIRMO Foundation , 50129 Florence ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haruka","family":"Ishii","sequence":"additional","affiliation":[{"name":"Medical Affairs Department, Kyowa Kirin Co. 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