PMID- 26886776 OWN - NLM STAT- MEDLINE DCOM- 20161102 LR - 20181202 IS - 1865-8652 (Electronic) IS - 0741-238X (Print) IS - 0741-238X (Linking) VI - 33 IP - 3 DP - 2016 Mar TI - Two-Year Data from a Long-Term Phase IV Study of Recombinant Human Growth Hormone in Short Children Born Small for Gestational Age. PG - 423-34 LID - 10.1007/s12325-016-0301-1 [doi] AB - INTRODUCTION: This ongoing, prospective, open-label, non-comparative, multicenter phase IV study is evaluating the safety and efficacy of recombinant human growth hormone (rhGH; Omnitrope((R)), Sandoz GmbH) in short children born small for gestational age (SGA). Here we report data from patients who have completed 2 years' treatment. METHODS: Eligibility criteria included prepubertal children born SGA with growth disturbances defined as current height standard deviation score (HSDS) <-2.5 and parental adjusted SDS <-1; birth weight and/or length <-2 SDS; and failure of catch-up growth [height velocity (HV) SDS <0 during the last year] by 4 years of age or later. The primary study objective is to assess the long-term effect of Omnitrope treatment on the development of diabetes in short children born SGA. Secondary objectives include evaluation of efficacy, incidence and severity of adverse events (AEs), occurrence of malignancies during treatment, and detection of anti-rhGH antibodies during treatment. RESULTS: In total, 278 children have been enrolled and received study medication; 249 have completed 2 years of treatment. No child has developed diabetes mellitus during the first 2 years; no fasting glucose or 2-h oral glucose tolerance test value exceeded the pre-defined limits of >126 or >200 mg/dL, respectively. No adverse alterations in body mass were noted. Treatment-emergent AEs were experienced by 211 (76.2%) children; most of these were of mild-to-moderate intensity (99.3%) and considered unrelated to study medication (97.6%). Treatment with Omnitrope was effective; mean HSDS was -3.39 at baseline, -2.57 at 1 year and -2.15 at 2 years of treatment. Mean HVSDS (peak-centered) also improved, from -2.13 at baseline to +4.16 at 1 year and +2.23 at 2 years. CONCLUSION: In this second interim analysis, short children born SGA were safely and effectively treated with rhGH (Omnitrope), and 2 years' treatment had no major adverse impact on carbohydrate metabolism or body mass. FUNDING: Sandoz. FAU - Schwarz, Hans-Peter AU - Schwarz HP AD - Department of Endocrinology, von Haunersches Kinderspital, University Hospital Munich, Munich, Germany. FAU - Walczak, Mieczyslaw AU - Walczak M AD - Department of Paediatric Endocrinology and Diabetology, Pomeranian Medical University, Szczecin, Poland. FAU - Birkholz-Walerzak, Dorota AU - Birkholz-Walerzak D AD - Department of Diabetology and Endocrinology, Medical University of Gdansk, Gdansk, Poland. FAU - Szalecki, Mieczyslaw AU - Szalecki M AD - Clinic of Endocrinology and Diabetology, Children's Memorial Health Institute, Warsaw, Poland. AD - Faculty of Medicine and Health Sciences UJK, Kielce, Poland. FAU - Nanu, Michaela AU - Nanu M AD - "Alfred Russescu" Institute for Mother and Child Care, Bucharest, Romania. FAU - Woehling, Heike AU - Woehling H AD - HEXAL AG, Holzkirchen, Germany. FAU - Schuck, Ellen AU - Schuck E AD - HEXAL AG, Holzkirchen, Germany. ellen.schuck@sandoz.com. LA - eng PT - Clinical Trial, Phase IV PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't DEP - 20160217 PL - United States TA - Adv Ther JT - Advances in therapy JID - 8611864 RN - 0 (Blood Glucose) RN - 0 (Recombinant Proteins) RN - 12629-01-5 (Human Growth Hormone) MH - Blood Glucose MH - Body Height/*drug effects MH - Child MH - Child, Preschool MH - Female MH - Glucose Tolerance Test MH - Growth Disorders/*drug therapy MH - Human Growth Hormone/adverse effects/*therapeutic use MH - Humans MH - *Infant, Small for Gestational Age MH - Male MH - Prospective Studies MH - Recombinant Proteins/administration & dosage/*therapeutic use PMC - PMC4833801 OTO - NOTNLM OT - Endocrinology OT - Omnitrope OT - Recombinant human growth hormone OT - Small for gestational age OT - Somatropin EDAT- 2016/02/18 06:00 MHDA- 2016/11/03 06:00 PMCR- 2016/02/17 CRDT- 2016/02/18 06:00 PHST- 2015/12/14 00:00 [received] PHST- 2016/02/18 06:00 [entrez] PHST- 2016/02/18 06:00 [pubmed] PHST- 2016/11/03 06:00 [medline] PHST- 2016/02/17 00:00 [pmc-release] AID - 10.1007/s12325-016-0301-1 [pii] AID - 301 [pii] AID - 10.1007/s12325-016-0301-1 [doi] PST - ppublish SO - Adv Ther. 2016 Mar;33(3):423-34. doi: 10.1007/s12325-016-0301-1. Epub 2016 Feb 17.