Abstract
Background: To understand what life is like for US children with a diagnosis of Growth Hormone Deficiency or Idiopathic Short Stature, the impact of short stature on Health related Quality of Life (HrQoL) was qualitatively examined and needs for care from the young patients and their parents perspective were identified. Methods: Focus group discussions with 26 American-English speaking and nine American-Spanish speaking children and their parents were conducted, transcribed verbatim and subsequently qualitatively analyzed by two independent raters, using an existing coding guideline, based on the multidimensional HrQoL concept and a special software (VERBI-Software MAXQDA 10). Results: A total of 1313 statements for the English-speaking and 447 statements for the Spanish-speaking families were categorized. In the US, the strongest frequency of mention was found for the HrQoL dimension “Social” across respondents, followed by “Treatment” and “Emotion”. Conclusion: Conducting and analyzing data generated from focus groups ensure that young patients’ experiences of disease are represented in the measure of outcomes for use in clinical trials and patient care.
Highlights
To understand what life is like for US children with a diagnosis of Growth Hormone Deficiency or Idiopathic Short Stature, the impact of short stature on Health related Quality of Life (HrQoL) was qualitatively examined and needs for care from the young patients and their parents perspective were identified
In the American-English sample more than half of the children/adolescents were diagnosed with growth hormone deficiency (GHD)
A current opportunity to utilize and further define the appropriateness of items for this HrQoL instrument is in upcoming trials of new long acting formulations of growth hormone (GH) and in the future, in clinical practice. This is the first qualitative study involving children with short stature diagnosed with GHD and idiopathic short stature (ISS) and their parents in the US, complementing a previous study in Europe [35]
Summary
To understand what life is like for US children with a diagnosis of Growth Hormone Deficiency or Idiopathic Short Stature, the impact of short stature on Health related Quality of Life (HrQoL) was qualitatively examined and needs for care from the young patients and their parents perspective were identified. Health professionals can use the results from HrQoL assessments to understand the burden of disease and the potential effectiveness of treatment from the perspective of the patient with the goal of improving their quality of care [4]. A literature review identified five pediatric condition-specific instruments available for use in children and adolescents with short stature None of these measures, provide full coverage of HrQoL domains potentially influenced by short stature [2]. The aim of the European Quality of Life in Short Stature Youth (QoLISSY), was to develop and validate a questionnaire to evaluate the impact of short stature on HrQoL in children and adolescents from both the patient and parent perspectives [5] [6]
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