title: Two-Year Data from a Long-Term Phase IV Study of Recombinant Human Growth Hormone in Short Children Born Small for Gestational Age.
abstract: BACKGROUND
  This ongoing, prospective, open-label, non-comparative, multicenter phase IV study is evaluating the safety and efficacy of recombinant human growth hormone (rhGH; Omnitrope(®), 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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  Sandoz.
authors.affiliation: Department of Endocrinology, von Haunersches Kinderspital, University Hospital Munich, Munich, Germany.
authors.forename: Hans-Peter
authors.initials: HP
authors.lastname: Schwarz
authors.affiliation: Department of Paediatric Endocrinology and Diabetology, Pomeranian Medical University, Szczecin, Poland.
authors.forename: Mieczysław
authors.initials: M
authors.lastname: Walczak
authors.affiliation: Department of Diabetology and Endocrinology, Medical University of Gdansk, Gdańsk, Poland.
authors.forename: Dorota
authors.initials: D
authors.lastname: Birkholz-Walerzak
authors.affiliation: Clinic of Endocrinology and Diabetology, Children's Memorial Health Institute, Warsaw, Poland.|Faculty of Medicine and Health Sciences UJK, Kielce, Poland.
authors.forename: Mieczyslaw
authors.initials: M
authors.lastname: Szalecki
authors.affiliation: "Alfred Russescu" Institute for Mother and Child Care, Bucharest, Romania.
authors.forename: Michaela
authors.initials: M
authors.lastname: Nanu
authors.affiliation: HEXAL AG, Holzkirchen, Germany.
authors.forename: Heike
authors.initials: H
authors.lastname: Woehling
authors.affiliation: HEXAL AG, Holzkirchen, Germany. ellen.schuck@sandoz.com.
authors.forename: Ellen
authors.initials: E
authors.lastname: Schuck
mesh_terms: D001786:Blood Glucose; D001827:Body Height / Q000187:drug effects*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005951:Glucose Tolerance Test; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007236:Infant, Small for Gestational Age; D008297:Male; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use*
chemical_list: D001786:Blood Glucose; D011994:Recombinant Proteins; D019382:Human Growth Hormone
keywords: Endocrinology; Omnitrope; Recombinant human growth hormone; Small for gestational age; Somatropin