Quality of Life in Adolescent Boys with Idiopathic Short Stature: Positive Impact of Growth Hormone and Aromatase Inhibitors

Abstract

Background: The combination of growth hormone (GH) and aromatase inhibitors (AI) improves linear growth in severely short adolescent boys; however, the effects of this intervention on quality of life (QoL) are unknown. This study assesses whether GH, AI, or their combination impacts the QoL of adolescent males with idiopathic short stature (ISS) from both the adolescent and the parent perspective. Method: A randomized open-label comparator trial was conducted in 76 pubertal males with ISS who received AI, GH, or AI/GH for 24 months. The condition-specific Quality of Life in Short Stature Youth questionnaire was used to assess QoL. Results: QoL scores were low at baseline in the children’s and parents’ reports. Within-group testing showed that total QoL scores increased significantly at 24 months in the GH and AI/GH group but not the AI group in the children’s report, whereas it increased in all of the groups in the parents’ report. Increases in QoL scores were associated with an increase in height SDS. Conclusions: Treatment with GH and AI/GH was associated with improved QoL scores as measured from both the patients’ and the parents’ perspectives, suggesting that the improved growth resulting from the use of these growth-promoting therapies has beneficial psychosocial effects in adolescent males with ISS followed for 24 months.

High-Level Expression of Alkaline Phosphatase by Adeno-Associated Virus Vector Ameliorates Pathological Bone Structure in a Hypophosphatasia Mouse Model

“…Because the most severe type of HPP affects infants and young children at an age when they are growing rapidly, growth impairment causing short stature is psychologically distressing for both patients and their families, and persistent susceptibility to fractures leads to skeletal deformity and ultimately contributes to impaired physical function. Accordingly, although these problems are not life-threatening, they severely diminish the QOL of patients and their families [32][33][34]. Such impaired growth has already been observed in patients with HPP receiving the current enzyme replacement therapy, with data showing that their height and weight are greater than two standard deviations below the mean [14].…”

The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy

“…” Physical appearance has been identified as the single most important factor contributing to self-worth in adolescents, therefore the improvement in the majority of QoL scores may reflect change in appearance [14] . Severe short stature, such as that often observed in DMD, can be associated with emotional distress and may contribute to diminished QoL [15] . Other common concerns were having a high pitched voice and mobility difficulties, for example when changing clothes.…”

Health-Related Quality of Life Assessment in Children and their Families: Aspects of Importance to the Pediatric Surgeon

“…Often psychosocial characteristics of HRQOL are more challenging to explore for the parents, 38 and parents of children with chronic conditions usually report lower HRQOL than the children themselves. [39][40][41] Within the pediatric surgery context, good agreement of pain intensity and HRQOL between parents and children after pediatric surgery were found. Thus, in this context, the parent report can be seen as a valid proxy for child self-reported pain intensity and HRQOL after having undergone pediatric surgery and after discharge.…”