{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T11:40:31Z","timestamp":1761824431560,"version":"3.40.5"},"reference-count":20,"publisher":"Oxford University Press (OUP)","issue":"3","license":[{"start":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T00:00:00Z","timestamp":1661990400000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["eje.bioscientifica.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,9,1]]},"abstract":"<jats:sec>\n<jats:title>Objective<\/jats:title>\n<jats:p>To analyze the effectiveness and safety of growth hormone (GH) replacement treatment in adult patients with Langerhans cell histiocytosis (LCH) and GH deficiency (GHD) enrolled in KIMS (Pfizer International Metabolic Database).<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Patients and methods<\/jats:title>\n<jats:p>Patients with LCH and GHD were studied at baseline and some of them after 1 year of GH treatment. The effectiveness of GH is presented as change after 1 year of treatment (mean, 95% CI). The LCH population was compared to two other groups of patients enrolled in KIMS, granulomatous and lymphocytic hypophysitis.<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Results<\/jats:title>\n<jats:p>At baseline, 81 adults with LCH (27 with childhood onset, 56% females), mean age at GHD onset of 29 (15) years were studied. Diabetes insipidus was diagnosed in 86% of patients. Analysis of 1 year of GH treatment was possible in 37 patients. One-year cross-sectional values for the GH dose were 0.39 (<jats:sc>s.d.<\/jats:sc>\u00b1 0.21) mg and \u22120.5 (\u22121.2 to 0.2) for insulin-like growth factor-1 <jats:sc>s.d<\/jats:sc>. Total cholesterol decreased 0.9 (\u22121.5 to \u22120.3 (mmol\/L); <jats:italic>P<\/jats:italic>\u2009&lt;\u20090.05); AGHDA-QoL-score (<jats:italic>n<\/jats:italic>\u2009=\u200920) was improved by 2.8 points (\u22125.6 to 0.0; <jats:italic>P<\/jats:italic>\u2009&lt;\u20090.05), while mean BMI increased 0.6 \u00b1 3 kg\/m<jats:sup>2<\/jats:sup> (95% CI: \u22120.2 to 1.4). All these effects did not differ from the two other groups after adjusting for age, gender, and baseline values. In 20 of 77 patients included in the safety analysis, 36 serious adverse events were reported during 435 patient-years (82.8\/1000); no new safety signals were reported.<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Conclusion<\/jats:title>\n<jats:p>After 1 year of GH treatment in patients with LCH, metabolic variables and quality of life improved, with no new safety signals.<\/jats:p>\n<\/jats:sec>","DOI":"10.1530\/eje-22-0160","type":"journal-article","created":{"date-parts":[[2022,7,4]],"date-time":"2022-07-04T18:04:51Z","timestamp":1656957891000},"page":"373-381","update-policy":"https:\/\/doi.org\/10.1530\/crossmarkpolicy-7","source":"Crossref","is-referenced-by-count":2,"title":["Pituitary function and the response to GH therapy in patients with Langerhans cell histiocytosis: analysis of the KIMS database"],"prefix":"10.1093","volume":"187","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8462-7753","authenticated-orcid":true,"given":"Philippe","family":"Touraine","sequence":"first","affiliation":[{"name":"Department of Endocrinology and Reproductive Medicine, Centre de Maladies Endocriniennes Rares de la Croissance et du D\u00e9veloppement, H\u00f4pital Universitaire Piti\u00e9 Salp\u00eatri\u00e8re-Charles Foix, Sorbonne Universit\u00e9, Facult\u00e9 de m\u00e9decine, Paris Cedex 13, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6325-2967","authenticated-orcid":true,"given":"Yempabou","family":"Sagna","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, CHU Souro Sanou, Intitut Sup\u00e9rieur des Sciences de la Sant\u00e9 (IN.S.SA), Universit\u00e9 Nazi Boni, Bobo-Dioulasso, Burkina Faso"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anders F","family":"Mattsson","sequence":"additional","affiliation":[{"name":"Pfizer Health AB, Sollentuna, Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pia","family":"Burman","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Sk\u00e5ne University Hospital, University of Lund, Malm\u00f6, Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andr\u00e9 P","family":"Van Beek","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin Ove","family":"Carlsson","sequence":"additional","affiliation":[{"name":"Pfizer Inc., New York, New York, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ferah","family":"Aydin","sequence":"additional","affiliation":[{"name":"Pfizer Health AB, Sollentuna, Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5903-3355","authenticated-orcid":true,"given":"Ulla","family":"Feldt-Rasmussen","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Metabolism, Rigshospitalet, and Institute of Clinical Sciences, Copenhagen University, Copenhagen, Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cecilia","family":"Camacho-H\u00fcbner","sequence":"additional","affiliation":[{"name":"Pfizer Inc., New York, New York, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","reference":[{"key":"bib1","series-title":"Trends in Endocrinology and Metabolism","doi-asserted-by":"publisher","first-page":"231","DOI":"10.1016\/S1043-2760(00)00268-X","article-title":"Growth hormone replacement in adults with growth hormone deficiency: assessment of current knowledge","volume":"11","author":"Carroll","year":"2000","unstructured":"Carroll PVChrist ERS\u00f6nksen PH. 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