Primary response in GHD children treatment as a predictor for long-term therapy effectiveness therapy effectiveness.

Introduction: Short stature in growth hormone deficiency (GHD) can be treated with recombinant human growth hormone (rhGH), which is proven to be both safe and effective. However, a considerable number of patients does not achieve satisfying therapy outcomes.

Aim of the study: To evaluate the predictive effect of height increase in the first year of rhGH treatment on long-term therapy outcomes.

Material and methods: 165 short-stature children (mean age 10.72 ±3.33 years; 63% males), diagnosed with GHD, treated with rhGH for at least one year (mean follow-up 4.32 ±1.80 years), divided into 2 groups according to the change in height standard deviation score (SDS) after the first year of rhGH treatment: good responders (GR) and poor responders (PR). Then, in one-year intervals, patient's chronological age, bone age, height, weight, insulin-like growth factor level, and rhGH dose were all assessed.

Results: In the GR group, mean height velocity SDS up to five years of observation was 1.19 ±0.41/year and in the PR group 0.59 ±0.38/year. The differences were statistically significant (p < 0.05).

Conclusions: The primary response to the rhGH treatment in GHD children seems to be a good predictor for long-term therapy outcomes.