{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T03:13:32Z","timestamp":1787022812301,"version":"build-2736575974"},"reference-count":0,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2021,2,1]],"date-time":"2021-02-01T00:00:00Z","timestamp":1612137600000},"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":[[2021,2]]},"abstract":"\n Objective<\/jats:title>\n \n The European Increlex\n \u00ae<\/jats:sup>\n Growth Forum Database Registry monitors the effectiveness and safety of recombinant human insulin-like growth factor-1 (rhIGF1; mecasermin, Increlex\n \u00ae<\/jats:sup>\n ) therapy in patients with severe primary IGF1 deficiency (SPIGFD). We present data from patients with and without a reported genetic diagnosis of Laron syndrome (LS).\n <\/jats:p>\n <\/jats:sec>\n \n Design<\/jats:title>\n Ongoing, open-label, observational registry (NCT00903110).<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n \n Children and adolescents receiving rhIGF1 therapy from 10 European countries were enrolled in 2008\u20132017 (\n n<\/jats:italic>\n \u2009=\u2009242). The treatment-na\u00efve\/prepubertal (NPP) cohort (\n n<\/jats:italic>\n \u2009=\u2009138) was divided into subgroups based on reported genetic diagnosis of LS (\n n<\/jats:italic>\n \u2009=\u200921) or non-LS (\n n<\/jats:italic>\n \u2009=\u2009117). Multivariate analysis of the NPP-non-LS subgroup was conducted to identify factors predictive of growth response (first-year-height standard deviation score (SDS) gain \u2265 0.3). Assessments included change in height and weight over 5 years and adverse events (AEs).\n <\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n \n Height SDS gain from baseline was greater in the NPP-LS than the NPP-non-LS subgroup after 1 years\u2019 treatment (\n P <<\/jats:italic>\n 0.05). In the NPP-non-LS subgroup, 56% were responders; young age at baseline was a positive independent predictive factor (\n P<\/jats:italic>\n < 0.001). NPP-non-LS-responders and the NPP-LS subgroup had a similar mean age (6.07 years vs 7.00 years) at baseline and height SDS gain in year 1 (0.64 vs 0.70), although NPP-non-LS-responders were taller (\n P<\/jats:italic>\n < 0.001) at baseline. BMI SDS changes did not differ across subgroups. Treatment-emergent AEs were experienced by 65.3% of patients; hypoglycaemia was most common.\n <\/jats:p>\n <\/jats:sec>\n \n Conclusions<\/jats:title>\n In most NPP children with SPIGFD, with or without LS, rhIGF1 therapy promotes linear growth. The safety profile was consistent with previous studies.<\/jats:p>\n <\/jats:sec>","DOI":"10.1530\/eje-20-0325","type":"journal-article","created":{"date-parts":[[2020,11,18]],"date-time":"2020-11-18T12:36:17Z","timestamp":1605702977000},"page":"267-276","update-policy":"https:\/\/doi.org\/10.1530\/crossmarkpolicy-7","source":"Crossref","is-referenced-by-count":21,"title":["Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome"],"prefix":"10.1093","volume":"184","author":[{"given":"Peter","family":"Bang","sequence":"first","affiliation":[{"name":"1Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Link\u00f6ping University, Link\u00f6ping, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joachim","family":"Woelfle","sequence":"additional","affiliation":[{"name":"2Children\u2019s Hospital, University of Erlangen, Erlangen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valerie","family":"Perrot","sequence":"additional","affiliation":[{"name":"3Ipsen Pharma, Boulogne-Billancourt, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Sert","sequence":"additional","affiliation":[{"name":"3Ipsen Pharma, Boulogne-Billancourt, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michel","family":"Polak","sequence":"additional","affiliation":[{"name":"4Department of Paediatric Endocrinology, Gynaecology, and Diabetology, AP-HP, Necker-Enfants Malades University Hospital, IMAGINE Institute, University of Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","container-title":["European Journal of Endocrinology"],"original-title":[],"link":[{"URL":"https:\/\/eje.bioscientifica.com\/view\/journals\/eje\/184\/2\/EJE-20-0325.xml","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/eje.bioscientifica.com\/downloadpdf\/journals\/eje\/184\/2\/EJE-20-0325.xml","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,2,14]],"date-time":"2023-02-14T11:14:54Z","timestamp":1676373294000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/ejendo\/article\/184\/2\/267\/6653851"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,2]]},"references-count":0,"journal-issue":{"issue":"2"},"URL":"https:\/\/doi.org\/10.1530\/eje-20-0325","relation":{},"ISSN":["0804-4643","1479-683X"],"issn-type":[{"value":"0804-4643","type":"print"},{"value":"1479-683X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,2]]}}}