ESPE Abstracts (2016) 86 P-P2-671

Assessing Disease and Treatment Burden for Young Children with Growth Hormone Deficiency (GHD)

Meryl Broda, Lars Wilkinsonb, Suzanne Lessard Alolgaa, Lise Højbjerreb, Jane Becka & Michael Højby Rasmussenb


aThe Brod Group, Mill Valley, California, USA; bNovo Nordisk A/S, Søborg, Denmark


Background: Children with GHD, in addition to short stature, may experience physiological symptoms as well as social and emotional problems. Assessing these impacts is critical for understanding the extent of GHD burden and assessing treatment benefit. Since many children initiating treatment are too young to self-report information, we must rely on adult reporters. However, according to FDA guidelines and established measure development principles, adult reporter information cannot be captured as subjective proxy information as this could bias responses. Thus, it is necessary to develop new methodology to capture objective, observer-reported (ObsRO) information for young children with GHD.

Objective and hypotheses: Develop methodology to capture reliable and valid data to allow for item development for ObsRO measures of disease and treatment burden for young GHD children.

Method: Qualitative interviews (n=34) were conducted (USA, Germany, UK) with parents/guardians of children (ages 4 to <13) with GHD. Respondents were instructed to report only what they ‘had seen, heard or been told by others’. To confirm their ability to respond objectively to questions about their child, respondents were asked to provide concrete examples of observed events or behaviors to support their assumptions about their child.

Results: The methodology developed elicited statements from parents/guardians regarding the impact of GHD on their child, which were validated by concrete examples. For example, reported burden of being treated differently by others was supported by parent/guardian observing children throwing a ball to their child with less force than they did to others. Based on qualitative data analysis of the interviews, a conceptual model of the range of GHD burdens for young children and items for ObsRO burden measures were developed.

Conclusion: Using the ObsRO measures developed for this study will make it is possible to collect reliable and valid disease and treatment burden information for young GHD children.

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