{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T03:48:28Z","timestamp":1780372108288,"version":"3.54.1"},"reference-count":31,"publisher":"The Endocrine Society","issue":"1","license":[{"start":{"date-parts":[[2023,7,27]],"date-time":"2023-07-27T00:00:00Z","timestamp":1690416000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,12,21]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context<\/jats:title>\n                  <jats:p>Burosumab is approved for the treatment of X-linked hypophosphatemia (XLH).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>To assess the efficacy and safety of burosumab in XLH patients, we conducted a systematic review and meta-analysis.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>We searched PubMed, the Cochrane Library, Embase, ClinicalTrials.gov, and Web of Science for studies on the use of burosumab in patients with XLH. Meta-analysis of randomized controlled trials (RCTs) and single-arm trials (SATs) was done to explore burosumab treatment on the efficacy and safety of XLH.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Of the 8 eligible articles, 5 were from RCTs and 3 were from SATs. Compared with the control group in RCTs, serum phosphorus level was significantly increased in the burosumab group (0.52 mg\/dL, 95% CI 0.24-0.80 mg\/dL). A meta-analysis of the burosumab arms in all trials revealed significant increase in serum phosphorus levels (0.78 mg\/dL, 95% CI 0.61-0.96 mg\/dL), TmP\/GFR (0.86 mg\/dL, 95% CI 0.60-1.12 mg\/dL), and 1,25-dihydroxyvitamin D level (13.23 pg\/mL, 95% CI 4.82-21.64 pg\/mL) as well. Changes in secondary events also validated the effects of burosumab treatment. Compared with the control group, in RCTs, the safety profile of burosumab is not much different from the control group. Data of the single-arm combined group demonstrated the incidence of any treatment emergency adverse event (TEAE) and the related TEAE rate were high, but the severity of most adverse events is mild to moderate, and the rate of serious TEAE is low.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>This study suggests that burosumab can be an option for patients with XLH and did not significantly increase the incidence of adverse events.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/clinem\/dgad440","type":"journal-article","created":{"date-parts":[[2023,7,27]],"date-time":"2023-07-27T08:41:52Z","timestamp":1690447312000},"page":"293-302","source":"Crossref","is-referenced-by-count":10,"title":["Efficacy and Safety of Burosumab in X-linked Hypophosphatemia"],"prefix":"10.1210","volume":"109","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4325-7928","authenticated-orcid":false,"given":"Sen","family":"Wang","sequence":"first","affiliation":[{"name":"Department of Geriatrics, The First Affiliated Hospital of Chongqing Medical University , Chongqing 400016 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoqin","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Geriatrics, The First Affiliated Hospital of Chongqing Medical University , Chongqing 400016 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miao","family":"He","sequence":"additional","affiliation":[{"name":"Department of Neurology, The Ninth Clinical Medical College Affiliated to Shanxi Medical University , Taiyuan 030000 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuanfen","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Geriatrics, The First Affiliated Hospital of Chongqing Medical University , Chongqing 400016 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Minghui","family":"Xiao","sequence":"additional","affiliation":[{"name":"Department of Geriatrics, The First Affiliated Hospital of Chongqing Medical University , Chongqing 400016 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9365-5265","authenticated-orcid":false,"given":"Houxun","family":"Ma","sequence":"additional","affiliation":[{"name":"Department of Geriatrics, The First Affiliated Hospital of Chongqing Medical University , Chongqing 400016 , China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2023,7,27]]},"reference":[{"issue":"7","key":"2023122122192800300_dgad440-B1","doi-asserted-by":"crossref","first-page":"1381","DOI":"10.1002\/jbmr.340","article-title":"A clinician's Guide to X-linked hypophosphatemia","volume":"26","author":"Carpenter","year":"2011","journal-title":"J Bone Miner Res"},{"issue":"1","key":"2023122122192800300_dgad440-B2","doi-asserted-by":"crossref","first-page":"17101","DOI":"10.1038\/nrdp.2017.101","article-title":"Rickets","volume":"3","author":"Carpenter","year":"2017","journal-title":"Nat Rev Dis Primers"},{"issue":"2","key":"2023122122192800300_dgad440-B3","doi-asserted-by":"crossref","first-page":"770","DOI":"10.1007\/s12325-019-01193-0","article-title":"Exploring the burden of X-linked hypophosphataemia: an opportunistic qualitative study of patient statements generated during a technology appraisal","volume":"37","author":"Ferizovi\u0107","year":"2020","journal-title":"Adv Ther"},{"issue":"10189","key":"2023122122192800300_dgad440-B4","doi-asserted-by":"crossref","first-page":"2416","DOI":"10.1016\/S0140-6736(19)30654-3","article-title":"Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial","volume":"393","author":"Imel","year":"2019","journal-title":"Lancet"},{"issue":"8","key":"2023122122192800300_dgad440-B5","doi-asserted-by":"crossref","first-page":"3144","DOI":"10.1210\/jc.2006-0021","article-title":"Dietary phosphorus regulates serum fibroblast growth factor-23 concentrations in healthy men","volume":"91","author":"Antoniucci","year":"2006","journal-title":"J Clin Endocrinol Metab"},{"issue":"4","key":"2023122122192800300_dgad440-B6","doi-asserted-by":"crossref","first-page":"1846","DOI":"10.1210\/jc.2009-1671","article-title":"Treatment of X-linked hypophosphatemia with calcitriol and phosphate increases circulating fibroblast growth factor 23 concentrations","volume":"95","author":"Imel","year":"2010","journal-title":"J Clin Endocrinol Metab"},{"issue":"4","key":"2023122122192800300_dgad440-B7","doi-asserted-by":"crossref","first-page":"1587","DOI":"10.1172\/JCI72829","article-title":"Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia","volume":"124","author":"Carpenter","year":"2014","journal-title":"J Clin Invest"},{"issue":"7","key":"2023122122192800300_dgad440-B8","doi-asserted-by":"crossref","first-page":"2565","DOI":"10.1210\/jc.2015-1551","article-title":"Prolonged correction of Serum phosphorus in adults with X-linked hypophosphatemia using monthly doses of KRN23","volume":"100","author":"Imel","year":"2015","journal-title":"J Clin Endocrinol Metab"},{"issue":"6","key":"2023122122192800300_dgad440-B9","doi-asserted-by":"crossref","first-page":"707","DOI":"10.1007\/s40265-018-0905-7","article-title":"Burosumab: first global approval","volume":"78","author":"Lamb","year":"2018","journal-title":"Drugs"},{"key":"2023122122192800300_dgad440-B10","doi-asserted-by":"crossref","first-page":"103420","DOI":"10.1016\/j.drugpo.2021.103420","article-title":"The impact of alcohol taxation changes on unrecorded alcohol consumption: A review and recommendations","volume":"99","author":"Rehm","year":"2022","journal-title":"Int J Drug Policy"},{"issue":"jul21 1","key":"2023122122192800300_dgad440-B11","doi-asserted-by":"crossref","first-page":"b2535","DOI":"10.1136\/bmj.b2535","article-title":"Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement","volume":"339","author":"Moher","year":"2009","journal-title":"BMJ"},{"key":"2023122122192800300_dgad440-B12","author":"Ma"},{"issue":"oct18 2","key":"2023122122192800300_dgad440-B13","doi-asserted-by":"crossref","first-page":"d5928","DOI":"10.1136\/bmj.d5928","article-title":"The Cochrane Collaboration's Tool for assessing risk of bias in randomised trials","volume":"343","author":"Higgins","year":"2011","journal-title":"BMJ"},{"issue":"9","key":"2023122122192800300_dgad440-B14","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1046\/j.1445-2197.2003.02748.x","article-title":"Methodological index for non-randomized studies (minors): development and validation of a new instrument","volume":"73","author":"Slim","year":"2003","journal-title":"ANZ J Surg"},{"issue":"3","key":"2023122122192800300_dgad440-B15","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1007\/s00223-019-00568-3","article-title":"Continued beneficial effects of burosumab in adults with X-linked hypophosphatemia: results from a 24-week treatment continuation period after a 24-week double-blind placebo-controlled period","volume":"105","author":"Portale","year":"2019","journal-title":"Calcif Tissue Int"},{"issue":"3","key":"2023122122192800300_dgad440-B16","doi-asserted-by":"crossref","first-page":"e001714","DOI":"10.1136\/rmdopen-2021-001714","article-title":"Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension","volume":"7","author":"Briot","year":"2021","journal-title":"RMD Open"},{"issue":"3","key":"2023122122192800300_dgad440-B17","doi-asserted-by":"crossref","first-page":"813","DOI":"10.1210\/clinem\/dgab729","article-title":"Sustained efficacy and safety of burosumab, a monoclonal antibody to FGF23, in children with X-linked hypophosphatemia","volume":"107","author":"Linglart","year":"2022","journal-title":"J Clin Endocrinol Metab"},{"issue":"21","key":"2023122122192800300_dgad440-B18","doi-asserted-by":"crossref","first-page":"1987","DOI":"10.1056\/NEJMoa1714641","article-title":"Burosumab therapy in children with X-linked hypophosphatemia","volume":"378","author":"Carpenter","year":"2018","journal-title":"N Engl J Med"},{"issue":"12","key":"2023122122192800300_dgad440-B19","doi-asserted-by":"crossref","first-page":"2183","DOI":"10.1002\/jbmr.3843","article-title":"Burosumab improved histomorphometric measures of osteomalacia in adults with X-linked hypophosphatemia: a phase 3, single-arm, international trial","volume":"34","author":"Insogna","year":"2019","journal-title":"J Bone Miner Res"},{"issue":"3","key":"2023122122192800300_dgad440-B20","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1016\/S2213-8587(18)30338-3","article-title":"Efficacy and safety of burosumab in children aged 1-4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial","volume":"7","author":"Whyte","year":"2019","journal-title":"Lancet Diabetes Endocrinol"},{"key":"2023122122192800300_dgad440-B21","doi-asserted-by":"crossref","first-page":"48","DOI":"10.3389\/fped.2020.00048","article-title":"Positive response to one-year treatment with burosumab in pediatric patients with X-linked hypophosphatemia","volume":"8","author":"Mart\u00edn Ramos","year":"2020","journal-title":"Front Pediatr"},{"issue":"3","key":"2023122122192800300_dgad440-B22","first-page":"529","article-title":"Rickets resistant to vitamin D therapy","volume":"54","author":"Albright","year":"1937","journal-title":"Am J Dis Child"},{"issue":"2","key":"2023122122192800300_dgad440-B23","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1038\/ng1095-130","article-title":"A gene (PEX) with homologies to endopeptidases is mutated in patients with X-linked hypophosphatemic rickets","volume":"11","author":"The HYP Consortium","year":"1995","journal-title":"Nat Genet"},{"issue":"4","key":"2023122122192800300_dgad440-B24","doi-asserted-by":"crossref","first-page":"469","DOI":"10.1016\/S0022-3476(64)80336-X","article-title":"Use of phosphate salts as an adjunct to vitamin d in the treatment of hypophosphatemic vitamin D refractory rickets","volume":"64","author":"West","year":"1964","journal-title":"J Pediatr"},{"issue":"18","key":"2023122122192800300_dgad440-B25","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1056\/NEJM198010303031802","article-title":"Bone response to phosphate salts, ergocalciferol, and calcitriol in hypophosphatemic vitamin D-resistant rickets","volume":"303","author":"Glorieux","year":"1980","journal-title":"N Engl J Med"},{"issue":"6","key":"2023122122192800300_dgad440-B26","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1002\/jbmr.5650070602","article-title":"X-linked hypophosphatemic rickets: a study (with literature review) of linear growth response to calcitriol and phosphate therapy","volume":"7","author":"Petersen","year":"1992","journal-title":"J Bone Miner Res"},{"issue":"1","key":"2023122122192800300_dgad440-B27","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.rdc.2012.03.005","article-title":"Assessment and interpretation of circulating 25-hydroxyvitamin D and 1,25-dihydroxyvitamin D in the clinical environment","volume":"38","author":"Hollis","year":"2012","journal-title":"Rheum Dis Clin North Am"},{"issue":"1","key":"2023122122192800300_dgad440-B28","doi-asserted-by":"crossref","first-page":"R13","DOI":"10.1530\/EC-13-0103","article-title":"Therapeutic management of hypophosphatemic rickets from infancy to adulthood","volume":"3","author":"Linglart","year":"2014","journal-title":"Endocr Connect"},{"issue":"6","key":"2023122122192800300_dgad440-B29","first-page":"1514","article-title":"Tertiary hyperparathyroidism during high phosphate therapy of familial hypophosphatemic rickets","volume":"75","author":"Rivkees","year":"1992","journal-title":"J Clin Endocrinol Metab"},{"issue":"5","key":"2023122122192800300_dgad440-B30","doi-asserted-by":"crossref","first-page":"762","DOI":"10.1111\/jpc.15976","article-title":"Clinical practice guidelines for paediatric X-linked hypophosphataemia in the era of burosumab","volume":"58","author":"Sandy","year":"2022","journal-title":"J Paediatr Child Health"},{"key":"2023122122192800300_dgad440-B31","doi-asserted-by":"crossref","first-page":"338","DOI":"10.3389\/fendo.2020.00338","article-title":"Clinical evidence for the benefits of burosumab therapy for X-linked hypophosphatemia (XLH) and other conditions in adults and children","volume":"11","author":"Schindeler","year":"2020","journal-title":"Front Endocrinol (Lausanne)"}],"container-title":["The Journal of Clinical Endocrinology &amp; Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/jcem\/advance-article-pdf\/doi\/10.1210\/clinem\/dgad440\/51096118\/dgad440.pdf","content-type":"application\/pdf","content-version":"am","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/jcem\/article-pdf\/109\/1\/293\/54731421\/dgad440.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/jcem\/article-pdf\/109\/1\/293\/54731421\/dgad440.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,12,21]],"date-time":"2023-12-21T22:20:26Z","timestamp":1703197226000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/jcem\/article\/109\/1\/293\/7231988"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,7,27]]},"references-count":31,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2023,7,27]]},"published-print":{"date-parts":[[2023,12,21]]}},"URL":"https:\/\/doi.org\/10.1210\/clinem\/dgad440","relation":{},"ISSN":["0021-972X","1945-7197"],"issn-type":[{"value":"0021-972X","type":"print"},{"value":"1945-7197","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2024,1]]},"published":{"date-parts":[[2023,7,27]]}}}