{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,4]],"date-time":"2026-11-04T23:49:55Z","timestamp":1793836195450,"version":"4.6.1"},"reference-count":35,"publisher":"Oxford University Press (OUP)","issue":"8","license":[{"start":{"date-parts":[[2024,6,10]],"date-time":"2024-06-10T00:00:00Z","timestamp":1717977600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Kyowa Kirin Co., Ltd"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,6,29]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>X-linked hypophosphatemia (XLH) is a genetic disease that results in excessive FGF23, chronic hypophosphatemia, and musculoskeletal abnormalities, with affected patients experiencing symptoms such as bone pain, bone deformity, fracture, and pseudofracture. Burosumab is a fully human monoclonal antibody that binds to FGF23, improving lowered serum 1,25(OH)2D and phosphate levels in patients with XLH. There are insufficient data on the use of burosumab, its safety, and the outcomes of treated patients in a real-world setting. The SUNFLOWER (Study of longitUdinal observatioN For patients with X-Linked hypOphosphatemic rickets\/osteomalacia in collaboration With Asian partnERs) study is an ongoing longitudinal, observational cohort study of patients with XLH in Japan and South Korea. Enrollment occurred between April 2018 and December 2020. This interim analysis compared the background characteristics of patients who received burosumab with those who did not, and assessed improvements in biomarkers, physical and motor function, health-related quality-of-life (HRQOL) and other patient-reported outcome (PRO) measures, as well as the safety of burosumab treatment in 143 Japanese patients from 15 institutions over 6\u00a0mo. The patients had a median [interquartile range] age of 17.5 [11.0, 38.8] yr and 98 (68.5%) were female. Among patients aged &amp;lt;18 and\u2009\u226518\u00a0yr, 40\/73 (54.8%) and 25\/70 (35.7%) received burosumab, respectively. More patients aged \u226518 who received burosumab had bone pain at baseline vs those not treated with burosumab (6\/25, 24.0% vs 2\/45, 4.4%, p=.021). Patients treated with burosumab had improved serum phosphate and 1,25(OH)2D levels; moreover, rickets severity and HRQOL\/PRO measures, such as pain, appeared to improve over 6\u00a0mo of burosumab treatment, and no new safety concerns were identified. This study identified trends in the background characteristics of patients with XLH who receive burosumab in real-world clinical practice. Furthermore, the results support the use of burosumab therapy in real-world settings.<\/jats:p>","DOI":"10.1093\/jbmrpl\/ziae079","type":"journal-article","created":{"date-parts":[[2024,6,10]],"date-time":"2024-06-10T14:39:21Z","timestamp":1718030361000},"source":"Crossref","is-referenced-by-count":15,"title":["Burosumab treatment of X-linked hypophosphatemia patients: interim analysis of the SUNFLOWER longitudinal, observational cohort study"],"prefix":"10.1093","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4069-5508","authenticated-orcid":false,"given":"Toshimi","family":"Michigami","sequence":"first","affiliation":[{"name":"Osaka Women\u2019s and Children\u2019s Hospital, Osaka Prefectural Hospital Organization Department of Bone and Mineral Research, , Osaka 594-1101, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hee Gyung","family":"Kang","sequence":"additional","affiliation":[{"name":"Seoul National University Children\u2019s Hospital Department of Pediatric Nephrology, , Seoul 03080, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3803-4500","authenticated-orcid":false,"given":"Noriyuki","family":"Namba","sequence":"additional","affiliation":[{"name":"Tottori University Division of Pediatrics and Perinatology, Faculty of Medicine, , Tottori 683-8504, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4071-5372","authenticated-orcid":false,"given":"Nobuaki","family":"Ito","sequence":"additional","affiliation":[{"name":"The University of Tokyo Division of Therapeutic Development for Intractable Bone Diseases, Graduate School of Medicine and Faculty of Medicine, , Tokyo 113-0033, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4483-4405","authenticated-orcid":false,"given":"Takuo","family":"Kubota","sequence":"additional","affiliation":[{"name":"Osaka University Department of Pediatrics, Graduate School of Medicine, , Osaka 565-0871, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ayumi","family":"Shintani","sequence":"additional","affiliation":[{"name":"Osaka Metropolitan University Department of Medical Statistics, Graduate School of Medicine, , Osaka 545-8585, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daijiro","family":"Kabata","sequence":"additional","affiliation":[{"name":"Osaka Metropolitan University Department of Medical Statistics, Graduate School of Medicine, , Osaka 545-8585, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masanori","family":"Kanematsu","sequence":"additional","affiliation":[{"name":"Kyowa Kirin Co., Ltd. Medical Affairs Department, , Tokyo 100-0004, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yayoi","family":"Nishida","sequence":"additional","affiliation":[{"name":"Kyowa Kirin Co., Ltd. Medical Affairs Department, , Tokyo 100-0004, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seiji","family":"Fukumoto","sequence":"additional","affiliation":[{"name":"Tamaki-Aozora Hospital Department of Diabetes and Endocrinology, , Tokushima 779-3125, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiichi","family":"Ozono","sequence":"additional","affiliation":[{"name":"ISEIKAI International General Hospital Center for Promoting Treatment of Intractable Diseases, , Osaka 530-0052, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,6,10]]},"reference":[{"issue":"9","key":"2024071614405212500_ref1","doi-asserted-by":"crossref","first-page":"811","DOI":"10.1507\/endocrj.EJ15-0275","article-title":"Nationwide survey of fibroblast growth factor 23 (FGF23)-related hypophosphatemic diseases in Japan: prevalence, biochemical data and treatment","volume":"62","author":"Endo","year":"2015","journal-title":"Endocr J"},{"issue":"3","key":"2024071614405212500_ref2","doi-asserted-by":"crossref","first-page":"491","DOI":"10.1530\/EJE-08-0818","article-title":"Incidence and prevalence of nutritional and hereditary rickets in southern Denmark","volume":"160","author":"Beck-Nielsen","year":"2009","journal-title":"Eur J Endocrinol"},{"issue":"2","key":"2024071614405212500_ref3","doi-asserted-by":"crossref","first-page":"222","DOI":"10.1016\/0002-9343(64)90085-3","article-title":"Vitamin D-resistant rickets. 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