Introduction: In Europe, GH is approved for short (height SDS <−2.5) children born small for gestational age (SGA) who fail to show catch-up growth by 4 years of age.
Methods: This report analysed the patterns of GH dose used in everyday clinical practice based on short children born SGA enrolled in NordiNet® IOS, an observational study evaluating the long-term effectiveness and safety of Norditropin®. Average GH dose, exposure time, age and height SDS at baseline and after initiation of GH treatment were calculated. The proportions of patients with low (<30 μg/kg per day), medium (3050 μg/kg per day), and high (≥50) GH dose were analysed as were the proportions with a decrease or an increase of >10%, or no change from baseline dose within the first year of treatment.
Results: Overall, 2652 children born SGA were analysed (mean±S.D.) (exposure, 3.4±2.4 years; age at GH start, 7.8±3.3; and baseline height SDS, −2.9±0.8). The mean±S.D. GH dose for the full treatment period was 38.6±10.0 μg/kg per day. Mean GH dose was similar between genders and unchanged through 4 years of treatment (Table). Baseline height SDS significantly correlated with GH dose (P<0.0001): mean height SDS was −2.8 in the low (n=296; GH, 25.8±5.1 μg/kg per day), −2.9 in the medium (n=2044; GH, 37.3±4.8 μg/kg per day), and −3.1 in the high (n=312; GH, 59±9.4 μg/kg per day) dose groups. Within the first year of treatment, dose increased for 1.7% and decreased for 14.8% of patients. Baseline height SDS was lower in patients with increasing GH dose (−3.1±1.1) than for those with no change (−2.8±0.8) (P=0.003) or decreased (−2.3±0.7) dose (P=0.005).
|GH dose (mean±S.D.)|
|Baseline||Year 1||Year 2||Year 3||Year 4|
Conclusions: In clinical practice, a very narrow range of GH doses is used for short children born SGA and doses tend to remain unchanged over time. Children with the lowest height SDS are more likely to receive GH in the higher dose range.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology