Quality of life before and after 12-months replacement therapy in children with growth hormone deficiency

A systematic review of literature in the Embase, MEDLINE, and Cochrane databases reveals incomplete and contradictory data on the psychosocial consequences of short stature in children, including its impact on quality of life (QoL). The aim of this study was to evaluate the effect of recombinant human growth hormone (rhGH) therapy on the QoL of children diagnosed with growth hormone deficiency (GHD). The QoL was considered as an indicator of psychological and social adaptation of the children. Materials and methods. A main group (46 prepubescent children with GHD) was selected and compared to a matched control group (80 healthy children). The group of children with short stature included all children with GHD of the main group and 48 children with idiopathic short stature.The Peds QL4.0 questionnaires for children and parents were used to determine QoL on the scales of “total score”, “physical health”, “psycho-social health”, “emotional functioning”, “social functioning”, and “school functioning”. Statistical processing of the results using the Spearman's correlation coefficient and by estimating the differences between the mean values of two independent variation series by the value of "p" was performed. Results. A more pronounced decrease in QoL was found in children with GHD if they are under 9 years of age compared to their older peers and in children with height SDS<- 3 compared to those with height SDS between -3 and -2. After 12 months of rhGH treatment, the normalization of global QоL score was found to be associated with optimal growth response, as measured by the Peds QL4.0 scale (rs=0.44, p<0.05). The study also revealed a direct correlation between adherence to replacement therapy and QоL (rs=0.28, p<0.05). Furthermore, there was a positive correlation between high compliance with rhGH therapy and optimal growth response (rs=0.85, p<0.05). Conclusions. The study found that there was a difference in the scores of the Peds QL 4.0 children's questionnaire and the Peds QL 4.0 parents' questionnaire in terms of awareness of physical health problems. However, both questionnaires indicated that after 12 months of rhGH therapy, all scales of the Peds QL 4.0 were normalized. Furthermore, a positive correlation was found between the QоL of children with GHD and their optimal growth response to rhGH therapy. The research was carried out in accordance with the principles of the Helsinki Declaration. The study protocol was approved by the Local Ethics Committee of the participating institution. The informed consent of the patient was obtained for conducting the studies. No conflict of interests was declared by the authors.