Background
Approximately 5% of all newborns are born small for gestational age (SGA), below 2 standard deviation scores (SDS) for height and/or weight. Beneficial effects of long-term growth hormone (GH) treatment on height have not been studied in short Japanese SGA children.
Aim
The aim of this study was to investigate the long-term efficacy and safety of two doses of GH in short Japanese children born SGA.
Methods
This was a multicenter, double-blind, randomized trial comparing two doses of GH for the treatment of short stature in prepubertal (Tanner Stage 1) Japanese children born SGA with no catch-up growth. Initial GH treatment was 0.033 mg/kg per day n = 39), 0.067 mg/kg per day (n = 38), or no treatment (n = 21) for 52 weeks. During a 208-week extension period, patients in the treated groups continued treatment at the same dose, and those in the no treatment group were randomized to receive either 0.033 (n = 10) or 0.067 mg/kg per day (n = 10) GH. The primary end point was the change in height standard deviation score (HSDS) for chronological age (CA). Secondary end points included change from baseline in height velocity (HV) SDS, bone age (BA), ratio of BA/CA, and metabolic parameters.
Results
A dose-dependent increase in mean HSDS for CA was seen in the two treated groups. After 260 weeks (5 years) of treatment, the mean HSDS for CA increased from –3.00 to –1.78 in the 0.033 mg/kg per day group and from –2.83 to –0.82 in the 0.067 mg/kg per day group. The initial no-treatment group showed a similar dose-dependent increase in HSDS after 4 years of treatment in the extension period. Bone age increased during GH treatment with the mean (standard deviation) change in bone age after 260 weeks being 5.79 (1.05) and 7.15 (1.05) years in the low- and high-dose groups, respectively. Both doses of GH were well tolerated with few treatment-related adverse events.
Conclusions
Long-term treatment with GH improved HSDS in a dose-dependent manner in short, prepubertal Japanese children born SGA and was well tolerated in this patient population.
Clinical Implications
Long-term GH treatment improves the height outcome of SGA children and is well tolerated.
Article info
Identification
Copyright
© 2012 Published by Elsevier Inc.
