Efficacy and safety of long-term continuous growth hormone treatment in children with Prader-Willi syndrome

Roderick F A de Lind van Wijngaarden*, Elbrich P C Siemensma, Dederieke A M Festen, Barto J Otten, Edgar G A H van Mil, Joost Rotteveel, Roelof J H Odink, G C B Karen Bindels-de Heus, Mariëtte van Leeuwen, Danny A J P Haring, Gianni Bocca, E C A Mieke Houdijk, J J Gera Hoorweg-Nijman, René C F M Vreuls, Petr E Jira, A S Paul van Trotsenburg, Boudewijn Bakker, Eelco J Schroor, Jan Willem Pilon, Jan M WitStenvert L S Drop, Anita C S Hokken-Koelega

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


BACKGROUND: Children with Prader-Willi syndrome (PWS) have abnormal body composition and impaired growth. Short-term GH treatment has beneficial effects.

OBJECTIVES: The aim of the study was to investigate effects of long-term continuous GH treatment on body composition, growth, bone maturation, and safety parameters.

SETTING: We conducted a multicenter prospective trial.

DESIGN: Fifty-five children with a mean +/- sd age of 5.9 +/- 3.2 yr were followed during 4 yr of continuous GH treatment (1 mg/m(2) . d). Data were annually obtained in one center: fat percentage (fat%) and lean body mass (LBM) by dual-energy x-ray absorptiometry, height, weight, head circumference, bone age, blood pressure, and fasting IGF-I, IGF binding protein-3, glucose, insulin, glycosylated hemoglobin, total cholesterol, high-density lipoprotein, and low-density lipoprotein. sd scores (SDS) were calculated according to Dutch and PWS reference values (SDS and SDS(PWS)).

RESULTS: Fat%SDS was significantly lower after 4 yr of GH treatment (P < 0.0001). LBMSDS significantly increased during the first year (P = 0.02) but returned to baseline values the second year and remained unchanged thereafter. Mean +/- sd height normalized from -2.27 +/- 1.2 SDS to -0.24 +/- 1.2 SDS (P < 0.0001). Head circumference SDS increased from -0.79 +/- 1.0 at start to 0.07 +/- 1.1 SDS after 4 yr. BMISDS(PWS) significantly decreased. Mean +/- sd IGF-I and the IGF-I/IGF binding protein-3 ratio significantly increased to 2.08 +/- 1.1 and 2.32 +/- 0.9 SDS, respectively. GH treatment had no adverse effects on bone maturation, blood pressure, glucose homeostasis, and serum lipids.

CONCLUSIONS: Our study in children with PWS shows that 4 yr of continuous GH treatment (1 mg/m(2) . d) improves body composition by decreasing fat%SDS and stabilizing LBMSDS and head circumference SDS and normalizes heightSDS without adverse effects. Thus, long-term continuous GH treatment is an effective and safe therapy for children with PWS.

Original languageEnglish
Pages (from-to)4205-15
Number of pages11
JournalJournal of Clinical Endocrinology & Metabolism
Issue number11
Publication statusPublished - Nov 2009
Externally publishedYes


  • Adipose Tissue/anatomy & histology
  • Birth Weight
  • Blood Pressure
  • Body Height
  • Body Weight
  • Bone Density
  • Child
  • Child, Preschool
  • Drug Administration Schedule
  • Fasting
  • Human Growth Hormone/administration & dosage
  • Humans
  • Insulin-Like Growth Factor Binding Protein 3/blood
  • Insulin-Like Growth Factor I/metabolism
  • Prader-Willi Syndrome/drug therapy
  • Prospective Studies
  • Safety


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