ESPE Abstracts (2014) 82 LBP-D--3-1005

A 2-Year Multi-Centre, Open Label, Randomized Two Arm Study of Genotropin Treatment in Very Young Children Born Small for Gestational Age: Early Growth and Neurodevelopment

Jean De Scheppera,b, Johan Vanderfaeillieb, Primus-E Mullisc, Raoul Roomand, Lisa Matthewse, Maria Dilleenf, Richard Browningf, Roy Gomezg & Hartmut Wollmannf

aUZ Brussel, Brussels, Belgium; bVrije Universiteit Brussel, Brussels, Belgium; cUniversity Children’s Hospital, Bern, Switzerland; dUniversiteit Antwerpen, Antwerpen, Belgium; eQuanticate, Hitchin, UK; fPfizer, Sandwich, UK; gPfizer, Brussels, Belgium

Background: There are limited data available on the efficacy and safety of GH treatment in very young (<30 months) short children, born small for gestational age (SGA).

Objectives: To determine the effect of 24 months of GH treatment on body height, BMI, and head growth as well as overall psychomotor development (using the Bayley Scale of Infant Development (BSID-II)) and demonstrate its safety in young (aged between 19 and 29 months) short SGA children.

Methods: Sixteen centers from eight European countries participated in the study; 21 short (height<−2.5 S.D.) SGA subjects were treated with GH (Genotropin®) at a dose of 0.035 mg/kg per day for 24 months, with 22 controls untreated. An ANCOVA model was performed on the change from baseline endpoint with appropriate covariates.

Results: GH treatment significantly increased height SDS compared to untreated controls at both month 12 (LS mean (S.E.): 1.03 (0.12) vs 0.14 (0.12)) and month 24 (LS mean (S.E.): 1.63 (0.13) vs 0.43 (0.13)) (P<0.001). Greater changes from baseline in the GH group compared to the control group were observed for head circumference, body weight and BMI. At month 12, there was no significant difference in the BSID-II MDI score and PDI score (P=0.738; P=0.301, respectively). At month 24, the mean change in bone age was >18 months in both groups. The observed AEs reflected the background occurrence of common childhood infections in this age group. Treatment-related SAEs (adenoidal and tonsillar hypertrophy) were reported in 1 (4.8%) subject in the GH group.

Conclusion: GH therapy initiated between the ages of 19 and 29 months in short SGA children, who failed to show early catch-up growth, was well tolerated and promoted rapid growth recovery without improving neurodevelopment. AEs were consistent with the safety profile of GH therapy.