PMID- 34113318 OWN - NLM STAT- MEDLINE DCOM- 20211220 LR - 20240402 IS - 1664-2392 (Print) IS - 1664-2392 (Electronic) IS - 1664-2392 (Linking) VI - 12 DP - 2021 TI - Effectiveness and Overall Safety of NutropinAq((R)) for Growth Hormone Deficiency and Other Paediatric Growth Hormone Disorders: Completion of the International Cooperative Growth Study, NutropinAq((R)) European Registry (iNCGS). PG - 676083 LID - 10.3389/fendo.2021.676083 [doi] LID - 676083 AB - OBJECTIVE: The International Cooperative Growth Study, NutropinAq((R)) European Registry (iNCGS) (NCT00455728) monitored long-term safety and effectiveness of recombinant human growth hormone (rhGH; NutropinAq((R)) [somatropin]) in paediatric growth disorders. METHODS: Open-label, non-interventional, post-marketing surveillance study recruiting children with growth disorders. Endpoints included gain in height standard deviation score (SDS), adult height, and occurrence of adverse events (AEs). RESULTS: 2792 patients were enrolled. 2082 patients (74.6%) had growth hormone deficiency (GHD), which was isolated idiopathic in 1825 patients (87.7%). Non-GHD diagnoses included Turner syndrome (TS) (n=199), chronic renal insufficiency (CRI) (n=10), other non-GHD (n=498), and missing data for three participants. Improvements from baseline height SDS occurred at all time points to Month 132, and in all subgroups by disease aetiology. At Month 12, mean (95% CI) change in height SDS by aetiology was: idiopathic GHD 0.63 (0.61;0.66), organic GHD 0.71 (0.62;0.80), TS 0.59 (0.53; 0.65), CRI 0.54 (-0.49;1.56), and other non-GHD 0.64 (0.59;0.69). Mean height ( +/- SD) at the last visit among the 235 patients with adult or near-adult height recorded was 154.0 cm ( +/- 8.0) for girls and 166.7 cm ( +/- 8.0) for boys. The most frequent biological and clinical non-serious drug-related AEs were increased insulin-like growth factor concentrations (314 events) and injection site haematoma (99 events). Serious AEs related to rhGH according to investigators were reported (n=30); the most frequent were scoliosis (4 events), epiphysiolysis (3 events), and strabismus (2 events). CONCLUSIONS: There was an improvement in mean height SDS in all aetiology subgroups after rhGH treatment. No new safety concerns were identified. CI - Copyright (c) 2021 Coutant, Bosch Munoz, Dumitrescu, Schnabel, Sert, Perrot and Dattani. FAU - Coutant, Regis AU - Coutant R AD - Pediatric Endocrinology, Centre Hospitalier Universitaire d'Angers, Angers, France. FAU - Bosch Munoz, Jordi AU - Bosch Munoz J AD - Pediatric Endocrinology, Hospital Arnau de Vilanova, Lleida, Spain. FAU - Dumitrescu, Cristina Patricia AU - Dumitrescu CP AD - Pediatric Endocrinology, Constantin Ion Parhon (C.I.) Parhon National Institute of Endocrinology, Bucharest, Romania. FAU - Schnabel, Dirk AU - Schnabel D AD - Center for Chronic Sick Children, Pediatric Endocrinology, Charite, University Medicine, Berlin, Germany. FAU - Sert, Caroline AU - Sert C AD - Ipsen, Boulogne-Billancourt, France. FAU - Perrot, Valerie AU - Perrot V AD - Ipsen, Boulogne-Billancourt, France. FAU - Dattani, Mehul AU - Dattani M AD - University College London (UCL) Great Ormond Street (GOS) Institute of Child Health and Great Ormond Street Hospital for Children, London, United Kingdom. LA - eng SI - ClinicalTrials.gov/NCT00455728 PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't DEP - 20210525 PL - Switzerland TA - Front Endocrinol (Lausanne) JT - Frontiers in endocrinology JID - 101555782 RN - 0 (Recombinant Proteins) RN - 12629-01-5 (Human Growth Hormone) SB - IM MH - Case-Control Studies MH - Child MH - Child, Preschool MH - Female MH - Follow-Up Studies MH - Growth Disorders/*drug therapy/pathology MH - Human Growth Hormone/deficiency/*therapeutic use MH - Humans MH - Infant MH - International Agencies MH - Male MH - Product Surveillance, Postmarketing/*statistics & numerical data MH - Prognosis MH - Recombinant Proteins/*therapeutic use MH - Registries/*statistics & numerical data MH - Renal Insufficiency, Chronic/*drug therapy/pathology MH - Turner Syndrome/*drug therapy/pathology PMC - PMC8185283 OTO - NOTNLM OT - NutropinAq(R) (somatropin) OT - growth hormone deficiency OT - paediatric GH disorders OT - rhGH, recombinant human GH OT - safety COIS- RC received honoraria for lectures and for the scientific organization of meetings from Sandoz, Ipsen, Novo Nordisk, Lilly, and Pfizer. JB-M received honoraria for lectures from Lilly, Merck Ferring, Sandoz and Ipsen and participated in Ipsen advisory board. DS served in advisory boards for Kyowa Kirin, Ipsen, Merck Serono, Novo Nordisk; received honoraria for lectures and for the scientific organization of meetings from, Hexal/Sandoz Ipsen, Merck Serono, Novo Nordisk and Pfizer; and received research grants from Hexal/Sandoz, Kyowa Kirin and Pfizer. MD received honoraria for lectures and for the scientific organization of meetings from Sandoz, Ipsen, Novo Nordisk and Pfizer. CS and VP are employees of Ipsen. The authors declare that this study received funding from Ipsen. The funder contributed to study design, data collection and analysis, decision to publish, and funded editorial support for preparation of the manuscript. The authors take full responsibility for the entire content of this submitted manuscript and approved submission. The remaining author declares that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. EDAT- 2021/06/12 06:00 MHDA- 2021/12/21 06:00 PMCR- 2021/01/01 CRDT- 2021/06/11 06:43 PHST- 2021/03/04 00:00 [received] PHST- 2021/05/05 00:00 [accepted] PHST- 2021/06/11 06:43 [entrez] PHST- 2021/06/12 06:00 [pubmed] PHST- 2021/12/21 06:00 [medline] PHST- 2021/01/01 00:00 [pmc-release] AID - 10.3389/fendo.2021.676083 [doi] PST - epublish SO - Front Endocrinol (Lausanne). 2021 May 25;12:676083. doi: 10.3389/fendo.2021.676083. eCollection 2021.