Understanding Treatment Burden for Children Treated for Growth Hormone Deficiency

Abstract

ObjectiveGrowth hormone deficiency (GHD) treatment for children requires growth hormone injections, typically administered daily until the child reaches adult height. Child GHD treatment burden is not well understood and no disease-specific measures exist to assess this burden. The purpose of the study was to explore GHD treatment burden for children and their parents by conducting concept elicitation interviews supporting a theoretical model of the impact of GHD treatment.MethodsFour focus groups (in Germany) and 52 telephone interviews (in the UK and USA) were conducted with children/adolescents with GHD aged 8 to <13 years and parents of children with GHD aged ≥4 to <13 years. The purpose of the interviews was to understand the experience of GHD treatment from the child’s perspective, and for parents, the impact of their child’s treatment on themselves. Interview transcripts were analyzed thematically based on modified grounded theory principles.ResultsInterviews with 70 respondents who produced descriptions (n = 73) of patients experiences with GHD treatment (three parents spoke for two children each) were conducted. Analysis identified three major areas of GHD treatment burden for children: physical; emotional well-being; and interference. Parent burdens identified were: emotional well-being and interference. Modifiers such as treatment efficacy and duration, which may impact the degree of treatment burden severity, were identified.ConclusionsOverall treatment burden of child GHD is considerable for children and their parents. The concept elicitation and theoretical model can be used to develop a disease-specific outcome measure, which adequately reflects the burden of GHD treatment for children and their parents.

Identifying Potentially Modifiable Factors Associated with Treatment Non-Adherence in Paediatric Growth Hormone Deficiency: A Systematic Review

“…Patterns of self-reported reasons for non-adherence to rhGH treatment amongst these studies centred around: anxiety and fear about the needle or the pain associated with administering the injection [43-45], the lack of confidence/skill of the person administering the injection [43], ineffectiveness of treatment [43, 45], the lack of freedom to choose the injection device [44], treatment interference issues, i.e., overnight sleepovers or travel activities [43], and poor HCP/patient communication [43, 45]. …”

<p>Exploring Potentially Modifiable Factors That Influence Treatment Non-Adherence Amongst Pediatric Growth Hormone Deficiency: A Qualitative Study</p>

“…The analytical orientation was pre-determined by prior knowledge from a recently conducted systematic review 22 and previous comparable literature. [35][36][37][38][39] The analyst's interpretation within the current thematic analysis was guided by a specific focus on the research objective to specifically explore the potentially modifiable factors that influence treatment non-adherence amongst parents/caregivers of children with GHD, although the analyst was open to new contextual ideas and concepts within the raw data. The intention of the analyst was to explore and integrate the diverse views, perspectives, and experiences of the participants and comprehensively report the semantic reality of participants.…”

“…35,37 Parents/caregivers commonly face a number of increased challenges for the first years of rhGH treatment, and often further into the child's teenage and young adult years. [35][36][37][38][39] Thus, a key way to better understanding the complex issue of pediatric non-adherence to rhGH treatment is to explore the personal perspectives of the parent/caregiver.…”

“…Several qualitative studies have explored factors relating to treatment non-adherence, among various clinical indications for rhGH treatment. [35][36][37][38] There has been limited evidence, however, with regard to the factors that influence non-adherence specifically amongst parents/caregivers of children with GHD. Thus, in light of the findings from the recent systematic review 22 and supporting literature, 4,7,8,15,40,41 the aim of this qualitative study was to specifically explore the potentially modifiable factors that influence non-adherence to rhGH treatment amongst parents/caregivers of children with GHD.…”

What Potentially Modifiable Factors are Associated With Treatment Nonadherence in Pediatric Growth Hormone Deficiency? A Quantitative Study

“…Given the overall implications of non-adherence on GHD patients and their families, healthcare professionals and the health care system, it is evident that treatment nonadherence is an important health issue, that warrants further attention [4,8,[25][26]. In view of the findings from the systematic review [10] and surrounding rhGH literature [1][2][3][4][5][11][12][13][27][28][29][30][31][32][33][34], the key objectives of the current study were to: determine the prevalence rate of nonadherence to rhGH treatment in paediatric GHD, examine the contribution of parental illness beliefs, treatment beliefs and the perceived HCP-parent/caregiver relationship on treatment adherence and in doing so, identify the potentially modifiable factors associated with nonadherence to rhGH treatment.…”