A 2-year multicentre, open-label, randomized, controlled study of growth hormone (Genotropin®) treatment in very young children born small for gestational age: Early Growth and Neurodevelopment (EGN) Study.
OBJECTIVE
In Europe, growth hormone (GH) treatment for children born small for gestational age (SGA) can only be initiated after 4 years of age. However, younger age at treatment initiation is a predictor of favourable response. To assess the effect of GH treatment on early growth and cognitive functioning in very young (<30 months), short-stature children born SGA.


METHODS
A 2-year, randomized controlled, multicentre study (NCT00627523; EGN study), in which patients received either GH treatment or no treatment for 24 months.


METHODS
Children aged 19-29 months diagnosed as SGA at birth, and for whom sufficient early growth data were available, were eligible. Patients were randomized (1:1) to GH treatment (Genotropin®, Pfizer Inc.) at a dose of 0·035 mg/kg/day by subcutaneous injection, or no treatment.


METHODS
The primary objective was to assess the change from baseline in height standard deviation score (SDS) after 24 months of GH treatment.


RESULTS
Change from baseline in height SDS was significantly greater in the GH treatment vs control group at both month 12 (1·03 vs 0·14) and month 24 (1·63 vs 0·43; both P < 0·001). Growth velocity SDS was significantly higher in the GH treatment vs control group at 12 months (P < 0·001), but not at 24 months. There was no significant difference in mental or psychomotor development indices between the two groups.


CONCLUSIONS
GH treatment for 24 months in very young short-stature children born SGA resulted in a significant increase in height SDS compared with no treatment.
Department of Paediatric Endocrinology, Universitair Ziekenhuis Brussel, Brussels, Belgium.
Jean
J
De Schepper
Faculty of Psychology and Educational Sciences, Vrije Universiteit Brussel, Brussels, Belgium.
Johan
J
Vanderfaeillie
Division of Paediatric Endocrinology, Inselspital, University Children's Hospital, Bern, Switzerland.
Primus-E
PE
Mullis
Department of Paediatrics, Universitair Ziekenhuis Antwerp, Antwerpen, Belgium.
Raoul
R
Rooman
Biostatistics, Quanticate, Herts, UK.
Anna
A
Robertson
Pfizer Belgium, Brussels, Belgium.
Maria
M
Dilleen
Pfizer Belgium, Brussels, Belgium.
Roy
R
Gomez
Pfizer Belgium, Brussels, Belgium.
Hartmut A
HA
Wollmann
D000234:Adenoids / Q000473:pathology; D001827:Body Height / Q000187:drug effects*; D002657:Child Development / Q000187:drug effects*; D002675:Child, Preschool; D005260:Female; D019382:Human Growth Hormone / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D006984:Hypertrophy / Q000139:chemically induced; D007223:Infant; D007231:Infant, Newborn; D007236:Infant, Small for Gestational Age; D007279:Injections, Subcutaneous; D008297:Male; D011597:Psychomotor Performance / Q000187:drug effects*; D013997:Time Factors; D016896:Treatment Outcome
D019382:Human Growth Hormone