Abstract
Aside from clinical endpoints like height gain, health-related quality of life has also become an important outcome indicator in the medical field. However, the data on short stature and health-related quality of life is inconsistent. Therefore, we examined changes in health-related quality of life in German children with idiopathic growth hormone deficiency or children born small for gestational age before and after 12 months of human growth hormone treatment. Children with idiopathic short stature without treatment served as a comparison group. At baseline, health-related quality of life data of 154 patients with idiopathic growth hormone deficiency (n = 65), born small for gestational age (n = 58), and idiopathic short stature (n = 31) and one parent each was collected. Of these, 130 completed health-related quality of life assessments after 1-year of human growth hormone treatment. Outcome measures included the Quality of Life in Short Stature Youth questionnaire, as well as clinical and sociodemographic data. Our results showed that the physical, social, and emotional health-related quality of life of children treated with human growth hormone significantly increased, while untreated patients with idiopathic short stature reported a decrease in these domains. Along with this, a statistically significant increase in height in the treated group can be observed, while the slight increase in the untreated group was not significant. In conclusion, the results showed that human growth hormone treatment may have a positive effect not only on height but also in improving patient-reported health-related quality of life of children with idiopathic growth hormone deficiency and children born small for gestational age.
Highlights
Short stature is defined as having a height that is lower than two standard deviation scores below the mean height for age and gender of the reference population and about 3% of the children in a population are of small stature [1]
Reports on psychosocial outcomes due to human growth hormone (hGH) treatment are inconsistent, we hypothesized that HRQOL significantly improves in the intervention group, while no significant HRQOL changes were expected in the comparison group
Our results showed that improving growth of short children by hGH treatment can affect their short-term HRQOL and might prevent possible psychological consequences of short stature
Summary
Short stature is defined as having a height that is lower than two standard deviation scores below the mean height for age and gender of the reference population and about 3% of the children in a population are of small stature [1]. It is a common symptom for a variety of conditions—some of which can be of endocrine nature such as idiopathic growth hormone deficiency (IGHD). Individuals diagnosed with ISS are born normal sized, but have a low growth velocity and often have a family history of short stature [4]. Without catch-up growth in the first 2 years these children remain short [5]
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