Background: The potential benefit of growth hormone (GH) therapy on health-related QOL (HQoL) of children with short stature related to GH deficiency (GHD) or smallness for gestational age (SGA) has not been well documented.
Objective and hypotheses: Our objective was to assess potential disease and treatment related predictors for a poor HQoL in GH treated children. Children with male gender, SGA disorder, greater height deficit at start of GH and poor height gain were expected to be at risk.
Methodology: The QoLISSY questionnaire - a cross-culturally developed height specific instrument - was sent to 157 children with idiopathic GHD and 219 non-syndromic SGA children, between 8 and 18 years old, being treated for at least one year with GH for short stature (height SDS < −2.5) at a Belgian GH treatment center. The questionnaires were filled out by the child.
Results: Median total QoLISSY scores (tQS) of 22 (14 male) GHD and 55 (32 male) SGA children with complete data (20% response rate) were similar (76%), but significantly (P< 0.001) higher than reported in untreated short children (65%). Whereas no gender difference in tQS was present in GHD patients, SGA females had a lower median score (71.4 vs 80.9%). Despite a similar median age and height SDS at start and a similar median height gain at evaluation, height SDS at evaluation, GH exposure time as well as total height gain SDS correlated positively with tQS only in the GHD group. Using linear regression, gender and height gain were the best predictors of the tQS in the whole group.
Conclusion: In a written survey with a low response rate, GH treated SGA girls were found to have the lowest HQoL. Females and children with the lowest height gain GH appear most at risk for presenting the lowest HQoL under GH therapy.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology