title: Long-Term Results of GH Treatment in Silver-Russell Syndrome (SRS): Do They Benefit the Same as Non-SRS Short-SGA?
abstract: Silver-Russell syndrome (SRS) is a genetically heterogeneous syndrome characterized by low birth weight, severe short stature, and variable dysmorphic features. GH treatment is a registered growth-promoting therapy for short children born small for gestational age, including SRS, but there are limited data on the GH response in SRS children and on differences in response among the (epi)genetic SRS subtypes (11p15 aberrations, maternal uniparental disomy of chromosome 7 [mUPD7], and idiopathic SRS).
  
  
  
  To compare growth and adult height between GH-treated small for gestational age children with and without SRS (non-SRS), and to analyze the difference in GH response among SRS genotypes.
  
  
  
  A longitudinal study.
  
  
  
  Sixty-two SRS and 227 non-SRS subjects.
  
  
  
  All subjects received GH treatment (1 mg/m(2)/d).
  
  
  
  Adult height and total height gain.
  
  
  
  The SRS group consisted of 31 children with 11p15 aberrations, 11 children with mUPD7, and 20 children with idiopathic SRS. At the start of GH treatment, mean (SD) height standard deviation score [SDS] was significantly lower in SRS (-3.67 [1.0]) than in non-SRS (-2.92 [0.6]; P < .001). Adult height SDS was lower in SRS (-2.17 [0.8]) than in non-SRS (-1.65 [0.8]; P = .002), but the total height gain SDS was similar. There was a trend toward a greater height gain in mUPD7 than in 11p15 (P = .12).
  
  
  
  Children with SRS have a similar height gain during GH treatment as non-SRS subjects. All (epi)genetic SRS subtypes benefit from GH treatment, with a trend toward mUPD7 and idiopathic SRS having the greatest height gain.
authors.affiliation: Department of Paediatrics (C.C.J.S., J.S.R., A.C.S.H.-K.), Subdivision Endocrinology, Erasmus University Medical Centre, 3015 GJ Rotterdam, The Netherlands; and Dutch Growth Research Foundation (G.R.J.Z., A.C.S.H.-K.), 3016 AH Rotterdam, The Netherlands.
authors.forename: C C J
authors.initials: CC
authors.lastname: Smeets
authors.affiliation: Department of Paediatrics (C.C.J.S., J.S.R., A.C.S.H.-K.), Subdivision Endocrinology, Erasmus University Medical Centre, 3015 GJ Rotterdam, The Netherlands; and Dutch Growth Research Foundation (G.R.J.Z., A.C.S.H.-K.), 3016 AH Rotterdam, The Netherlands.
authors.forename: G R J
authors.initials: GR
authors.lastname: Zandwijken
authors.affiliation: Department of Paediatrics (C.C.J.S., J.S.R., A.C.S.H.-K.), Subdivision Endocrinology, Erasmus University Medical Centre, 3015 GJ Rotterdam, The Netherlands; and Dutch Growth Research Foundation (G.R.J.Z., A.C.S.H.-K.), 3016 AH Rotterdam, The Netherlands.
authors.forename: J S
authors.initials: JS
authors.lastname: Renes
authors.affiliation: Department of Paediatrics (C.C.J.S., J.S.R., A.C.S.H.-K.), Subdivision Endocrinology, Erasmus University Medical Centre, 3015 GJ Rotterdam, The Netherlands; and Dutch Growth Research Foundation (G.R.J.Z., A.C.S.H.-K.), 3016 AH Rotterdam, The Netherlands.
authors.forename: A C S
authors.initials: AC
authors.lastname: Hokken-Koelega
mesh_terms: D000293:Adolescent; D001827:Body Height / Q000187:drug effects*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D007236:Infant, Small for Gestational Age; D008137:Longitudinal Studies; D008297:Male; D011446:Prospective Studies; D056730:Silver-Russell Syndrome / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D019382:Human Growth Hormone