Understanding burden of illness for child growth hormone deficiency.

PURPOSE: Research demonstrates that children and adolescents with growth hormone deficiency (GHD) are impacted in multiple ways beyond their short stature; however, there are no disease-specific measures to assess these impacts. The purpose of this study was to examine the burden of GHD on children and adolescents, and to conduct concept elicitation to develop a model of the impact of GHD to support a disease-specific outcome measure. METHODS: Four focus groups and 52 telephone interviews were conducted with children with GHD and parents/guardians of children with GHD to understand the experience and impacts from the child's perspective, reported by children or parent-observers about the impact on the child. The interviews and focus groups were conducted in Germany, the United Kingdom, and the United States. Interview transcripts were analyzed thematically based on modified grounded theory principles. RESULTS: There were 73 descriptions of patient's experiences elicited from 70 respondents, as three respondents spoke for two children each. A majority of GHD descriptive narratives refer to boy children (n = 51, 69.9%) and a majority of children had taken GHD treatment (n = 64, 89%). Analysis identified four major areas of GHD impact: Signs and Symptoms (beyond short stature), Physical Aspects of Daily Life, Social Well-Being, and Emotional Well-Being. CONCLUSIONS: The burden of GHD in children and adolescents is considerable and not limited to short stature. The severity of GHD impact on children and adolescents appears to be variable and individualized, but these data indicate that early identification and growth hormone treatment may lead to fewer impacts.

Understanding Treatment Burden for Children Treated for Growth Hormone Deficiency.

OBJECTIVE: Growth 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. METHODS: Four 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. RESULTS: Interviews 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. CONCLUSIONS: Overall 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.

Barriers to initiating insulin in type 2 diabetes patients: development of a new patient education tool to address myths, misconceptions and clinical realities.

PURPOSE: The purpose of this study was to identify patient beliefs as well as clinical realities about insulin that may be barriers to type 2 diabetes patients initiating insulin treatment when recommended by their physician. This information was then used to develop a clinically relevant, cross-culturally valid patient education tool with the goal of providing unbiased, medically informative statements addressing these barriers. METHODS: Thirteen focus groups were conducted in five countries (Germany, Sweden, The Netherlands, UK, and USA) to collect qualitative data on attitudes about insulin therapy from type 2 diabetes patients aged 18 or older whose physician had recommended initiating insulin treatment in the past 6 months (n = 87). Additionally, a panel of four clinical experts was interviewed to ascertain obstacles they experience in initiating insulin with their patients. RESULTS: On the basis of the interview data, the ten questions that asked about the most important barriers were generated. The clinical expert panel then generated clinically accurate and unbiased responses addressing these concerns, and the educational tool "Questions about Starting Insulin: Information on the Myths, Misconceptions and Clinical Realities about Insulin" was drafted. The draft tool was pilot tested in a group of patients and finalized. CONCLUSIONS: Patient misconceptions, as well as some clinical realities, about insulin treatment and diabetes can influence the decision to initiate insulin treatment and ultimately impact disease management. The educational tool developed through this study was designed to help patients who are deciding whether or not to initiate insulin therapy as recommended by their physician, and facilitate patient-health-care provider interactions.