{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T21:15:29Z","timestamp":1785186929660,"version":"3.55.0"},"reference-count":29,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2022,7,11]],"date-time":"2022-07-11T00:00:00Z","timestamp":1657497600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000272","name":"National Institute for Health Research","doi-asserted-by":"publisher","award":["Academic Clinical Fellowship for NRL"],"award-info":[{"award-number":["Academic Clinical Fellowship for NRL"]}],"id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000853","name":"University of Glasgow","doi-asserted-by":"publisher","award":["Gardiner Lectureship"],"award-info":[{"award-number":["Gardiner Lectureship"]}],"id":[{"id":"10.13039\/501100000853","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014593","name":"Neurocrine Biosciences","doi-asserted-by":"publisher","award":["Unrestricted Education Grant"],"award-info":[{"award-number":["Unrestricted Education Grant"]}],"id":[{"id":"10.13039\/100014593","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"Medical Research Council","doi-asserted-by":"publisher","award":["G1100236"],"award-info":[{"award-number":["G1100236"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Endocrinology"],"published-print":{"date-parts":[[2022,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Congenital adrenal hyperplasia (CAH) requires exogenous steroid replacement. Treatment is commonly monitored by measuring 17\u2010OH progesterone (17OHP) and androstenedione (D4).<\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p>Retrospective cohort study using real\u2010world data to evaluate 17OHP and D4 in relation to hydrocortisone (HC) dose in CAH patients treated in 14 countries.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients<\/jats:title><jats:p>Pseudonymized data from children with 21\u2010hydroxylase deficiency (21OHD) recorded in the International CAH Registry.<\/jats:p><\/jats:sec><jats:sec><jats:title>Measurements<\/jats:title><jats:p>Assessments between January 2000 and October 2020 in patients prescribed HC were reviewed to summarise biomarkers 17OHP and D4 and HC dose. Longitudinal assessment of measures was carried out using linear mixed\u2010effects models (LMEM).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Cohort of 345 patients, 52.2% female, median age 4.3\u2009years (interquartile range: 3.1\u20139.2) were taking a median 11.3\u2009mg\/m<jats:sup>2<\/jats:sup>\/day (8.6\u201314.4) of HC. Median 17OHP was 35.7\u2009nmol\/l (3.0\u2013104.0). Median D4 under 12\u2009years was 0\u2009nmol\/L (0\u20132.0) and above 12\u2009years was 10.5\u2009nmol\/L (3.9\u201321.0). There were significant differences in biomarker values between centres (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.05). Correlation between D4 and 17OHP was good in multiple regression with age (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001, <jats:italic>R<\/jats:italic><jats:sup>2<\/jats:sup>\u2009=\u20090.29).<\/jats:p><jats:p>In longitudinal assessment, 17OHP levels did not change with age, whereas D4 levels increased with age (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001, <jats:italic>R<\/jats:italic><jats:sup>2<\/jats:sup>\u2009=\u20090.08). Neither biomarker varied directly with dose or weight (<jats:italic>p<\/jats:italic>\u2009&gt;\u20090.05). Multivariate LMEM showed HC dose decreasing by 1.0\u2009mg\/m<jats:sup>2<\/jats:sup>\/day for every 1 point increase in weight standard deviation score.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Registry data show large variability in 17OHP and D4 between centres. 17OHP correlates with D4 well when accounting for age. Prescribed HC dose per body surface area\u00a0decreased with weight gain.<\/jats:p><\/jats:sec>","DOI":"10.1111\/cen.14796","type":"journal-article","created":{"date-parts":[[2022,7,4]],"date-time":"2022-07-04T06:31:42Z","timestamp":1656916302000},"page":"551-561","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Analysis of therapy monitoring in the International Congenital Adrenal Hyperplasia Registry"],"prefix":"10.1111","volume":"97","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7686-6172","authenticated-orcid":false,"given":"Neil","family":"Lawrence","sequence":"first","affiliation":[{"name":"Department of Oncology and Metabolism University of Sheffield Sheffield UK"},{"name":"Sheffield Children's Hospital NHS Foundation Trust Sheffield UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Bacila","sequence":"additional","affiliation":[{"name":"Department of Oncology and Metabolism University of Sheffield Sheffield UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeremy","family":"Dawson","sequence":"additional","affiliation":[{"name":"Institute of Work Psychology, Management School University of Sheffield Sheffield UK"},{"name":"School of Health and Related Research, University of Sheffield Sheffield UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jillian","family":"Bryce","sequence":"additional","affiliation":[{"name":"Office for Rare Conditions Royal Hospital for Children &amp; Queen Elizabeth University Hospital Glasgow UK"},{"name":"Office for Rare Conditions Royal Hospital for Children &amp; Queen Elizabeth University Hospital Glasgow UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salma R.","family":"Ali","sequence":"additional","affiliation":[{"name":"Office for Rare Conditions Royal Hospital for Children &amp; Queen Elizabeth University Hospital Glasgow UK"},{"name":"Office for Rare Conditions Royal Hospital for Children &amp; Queen Elizabeth University Hospital Glasgow UK"},{"name":"Developmental Endocrinology Research Group University of Glasgow  Glasgow UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica L. T.","family":"van den Akker","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, Sophia Children's Hospital Erasmus Medical Centre  Rotterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T\u00e2nia A. S. S.","family":"Bachega","sequence":"additional","affiliation":[{"name":"Hormones and Molecular Genetics Laboratory LIM 42, Department of Internal Medicine University of Sao Paulo  Sao Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Baronio","sequence":"additional","affiliation":[{"name":"Department of Medical and Surgical Sciences, Pediatric Unit, Endo\u2010ERN Center for Rare Endocrine Diseases S. Orsola\u2010Malpighi University Hospital  Bologna Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niels H.","family":"Birkeb\u00e6k","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Aarhus University Hospital  Aarhus Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6797-7604","authenticated-orcid":false,"given":"Walter","family":"Bonfig","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Technical University Munich  Munich Germany"},{"name":"Department of Pediatrics Klinikum Wels\u2010Grieskirchen  Wels Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hedi C.","family":"van der Grinten","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology Radboud University Medical Centre  Nijmegen the Netherlands"},{"name":"Amalia Children's Hospital Radboud University Medical Centre  Nijmegen the 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