title: Efficacy of growth hormone replacement on anthropometric outcomes, obesity, and lipids in children with optic nerve hypoplasia and growth hormone deficiency.
abstract: BACKGROUND
  Hypopituitarism and obesity are causes of major lifelong morbidity in patients with optic nerve hypoplasia (ONH). Growth hormone deficiency (GHD) affects the majority of children with ONH, though the degree of deficiency and variability of early growth patterns range from early severe retardation to normal initial growth. The utility of early GH replacement for improving anthropometric, body composition, and lipid outcomes in patients with ONH and GHD, especially those with normal initial height velocity, is unknown. This study examines the effects of GH replacement in a cohort of children with ONH and GHD.
  
  
  METHODS
  Controlled clinical trial from 2005-2014. The study included 17 children with ONH and untreated GHD. Those meeting criteria for growth deceleration were assigned to treatment with recombinant human growth hormone (n = 5) while those with normal height velocity were randomized either to treatment (n = 5) or to observation (no intervention, n = 7). Study duration was 3 years. Primary outcome measures included stature, weight, weight-for-stature, and BMI standard deviation score (SDS) at study completion.
  
  
  RESULTS
  Subjects on GH, irrespective of entry growth trajectory, grew more on average in stature than controls by a difference of 0.98 SDS by study end; this effect persisted after adjusting for baseline overweight status. Treatment had an effect on weight SDS only after adjusting for initial overweight status, resulting in an average increase of 0.83 SDS more than controls. Subjects who were overweight at the outset experienced greater gains in both weight and stature SDS. Treatment had no statistically significant impact on weight-for-stature or BMI SDS. A reduction in body fat percentage was observed in those treated, both before (-6.1 %) and after (-4.3 %) adjustment for initial overweight status.
  
  
  CONCLUSIONS
  Early GH replacement has a positive effect on short-term statural outcomes in children with ONH and GHD, even in those exhibiting normal initial linear growth. Results were less conclusive regarding treatment effects on body composition and lipids.
authors.affiliation: The Vision Center, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Carly
authors.initials: C
authors.lastname: Stewart
authors.affiliation: The Vision Center, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Pamela
authors.initials: P
authors.lastname: Garcia-Filion
authors.affiliation: Division of Endocrinology, Diabetes, and Metabolism, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Cassandra
authors.initials: C
authors.lastname: Fink
authors.affiliation: Division of Endocrinology, Diabetes, and Metabolism, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Anna
authors.initials: A
authors.lastname: Ryabets-Lienhard
authors.affiliation: Division of Endocrinology, Diabetes, and Metabolism, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA ; The Saban Research Institute, Children's Hospital Los Angeles, 4661 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Mitchell E
authors.initials: ME
authors.lastname: Geffner
authors.affiliation: The Vision Center, Children's Hospital Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027 USA ; The Saban Research Institute, Children's Hospital Los Angeles, 4661 Sunset Boulevard, Los Angeles, CA 90027 USA.
authors.forename: Mark
authors.initials: M
authors.lastname: Borchert
keywords: Growth hormone deficiency; Growth without growth hormone; Obesity; Optic nerve hypoplasia