ESPE Abstracts (2014) 82 P-D-2-3-440

Two-Year Data from a Long-Term, Phase IV Study of Omnitrope®, a Recombinant Human GH, in Short Children Born Small for Gestational Age

Hans-Peter Schwarza, Dorota Birkholz-Walerzakb, Mieczyslaw Szaleckic, Mieczyslaw Walczakd, Corina Galesanue, David Metrevelif, Jasmin Khan-Bolukig & Ellen Schuckg

aUniversity Children’s Hospital Munich, Munich, Germany; bMedical University, Gdansk, Poland; cChildren’s Health Research Institute, Warsaw, Poland; dPomeranian Medical University, Szczecin, Poland; eUniversity of Medicine and Pharmacy ‘Gr.T.Popa’, Iasi, Romania; fTblisi State Medical University, Tblisi, Georgia; gSandoz Biopharmaceuticals, Holzkirchen, Germany

Background: Children born small for gestational age (SGA) are predisposed to metabolic abnormalities. While the therapeutic benefit of recombinant human growth hormone (rhGH) therapy in improving height is widely recognised, it can affect carbohydrate metabolism, potentially inducing diabetes.

Objective and hypotheses: This ongoing, prospective study aims to evaluate the long-term safety and efficacy of Omnitrope® (somatropin) in children born SGA. Here, interim data from patients who completed 2 years of treatment are provided.

Method: Prepubertal children born SGA were recruited according to standard criteria and will be treated with Omnitrope® and followed at predetermined time intervals until final height is reached.

Results: Of 278 children enrolled, 249 completed their 2-year assessment. Mean oral glucose tolerance (2 h), HbA1c, and fasting glucose levels did not change significantly from baseline to year 2; however, fasting insulin levels increased from baseline to year 1 (Δ+18.03 pmol/l) and from years 1 to 2 (Δ+6.59). Auxological measurements indicated improvements in height parameters from baseline to year 2, including mean height SDS (HSDS; net gain Δ+1.25), height velocity (HV; Δ+3.04 cm/year), and peak-centred HVSDS (baseline: −2.13; year 1:+4.16; year 2:+2.23). Similarly, mean IGF1 SDS and IGFBP3 SDS also increased markedly throughout the study; the mean difference (baseline to Year 2) in molar IGF1/IGFBP3 SDS ratios was: Δ+2.14. Reasons for treatment discontinuation comprised non-response (n=13), withdrawal of consent (n=11), loss to follow-up (n=3), adverse event (n=1), and other (n=1). There was one treatment-related serious adverse event (severe headache); the patient recovered and continued treatment. The incidence of anti-rhGH antibodies remained low and transient during the study (1.2% at year 2) and did not lead to discontinuation.

Conclusion: At this 2-year follow-up, no patient developed diabetes during rhGH treatment and there were no concerning or clinically relevant safety findings. In addition, Omnitrope treatment was effective, as documented by all height parameters.