ESPE Abstracts

Objectives: Recombinant growth hormone (rhGH) treatment helps to achieve a final height close to the parental growth potential in children with GH deficiency (GHD) and small for gestational age (SGA). Less is known about efficacy and safety of long term therapy with biosimilar rhGH. The aim of our study is to assess height gain and safety of therapy with biosimilar rhGH (Omnitrope®, Sandoz) in Czech children with GHD and SGA over the first three years of treatment and to compare our data with longitudinal international study GeNeSIS Italian Cohort (Humatrope®, Eli Lilly).

Methods: In total 165 Czech children included in PATRO (Omnitrope®, Sandoz study) (103 M, 62 F) were enrolled, 100 with idiopathic GHD and 65 SGA. GH treatment started at the age 7.2 years (2.7–15.7) in GHD and 5.9 years (2.9–14.3) in SGA cohort (median, range). All subjects were treated with Omnitrope® in 8 Czech specialised tertiary centres, GHD group for 42.6±19.2 months and SGA 45.9±21.4 months (mean±SD). Auxological data to assess height gain were compared with published data of GeNeSIS study. Adverse events (AEs) were analysed as well.

Results: At the start of therapy, our GHD cohort was shorter compared with the GeNeSIS (-2.9 SD versus -2.4 SD). During first year of therapy, mean growth velocity increased from 4.2 cm/year to 9.4 cm/yr in GHD and from 4.7 to 9.1 cm/year in SGA group (both P < 0.001), which is similar gain as in the GeNeSIS study. Height SDS increased during first year of GH therapy in GHD cohort from –2.9±0.7 to -2.1±0.5 (mean±SD) (P < 0.001), similarly in SGA cohort from -3.0±0.6 to -2.3±0.6 (P < 0.001). The increment corresponds to the reference GeNeSis study. A comparison of the difference between the current and target height after 3 years of treatment showed an improvement in our GHD group from -2.2±0.7 to -0.8±0.6 and from -2.2±0.5 to -0.9±0.6 in SGA (not evaluated in the GeNeSis study). No serious AEs considered to be related to GH were reported in our cohort. Only the minor AEs observed in 29.6% of our patients are probably not related to GH treatment (acute respiratory infections, diarrhea, urinary tract infections, injuries).

Conclusions: Our data confirm significant improvement of growth parameters and safety of therapy with biosimilar rhGH Omnitrope® in Czech children with GHD and SGA which is consistent with results from international database GeNeSIS study.