ESPE Abstracts (2019) 92 P1-84

Development of a Parent Experience Measure for Parents of Children with Achondroplasia

Kathyrn M. Pfeiffer1, Meryl Brod1, Dorthe Viuff2, Sho Ota2, Jill Gianettoni2, Jonathan Leff2


1The Brod Group, Mill Valley, USA. 2Ascendis Pharma, Palo Alto, USA


Background: Limited research exists on how having a child with achondroplasia impacts parents' daily lives and well-being. The purpose of the study was to gather qualitative evidence to support the development of a parent experience measure that assess the impacts of having a child aged 2 to <12 years with achondroplasia.

Methods: Concept elicitation interviews via individual telephone calls and an in-person focus group were conducted with parents of children with achondroplasia (aged 2 to <12 years) in the United States (US) and Spain using a semi-structured interview guide to elicit parents' experiences. Based on an adapted grounded theory approach, interview transcripts were analyzed to identify important themes and impacts for the development of a validation-ready parent experience measure.

Results: Thirty-six parents (n=31 mothers; n=5 fathers) of children with achondroplasia participated in individual interviews or the focus group (Spain, n=11; US, n=25), including seven parents with achondroplasia. Analyses identified four key domains relevant to the daily lives and well-being of parents of children with achondroplasia, including emotional well-being, caretaking responsibilities, family life, and work. In the parent emotional well-being domain, the most frequent impacts were worry about child's future (75%, n=27), worry about child's physical health (67%, n=24), concerns about child's safety (50%, n=18), feeling stressed/overwhelmed (44%, n=16), worry about child's social well-being (42%, n=15), and worry about child functioning independently (33%, n=12). For caretaking responsibilities, the most frequent impacts were managing child's medical care (e.g., appointments, treatment decisions; 92%, n=33), helping child with self-care (e.g., toileting, bathing, dressing; 67%, n=24), advocating for child (e.g., for school accommodations; 64%, n=23), providing assistance to child (e.g., reaching objects; 56%, n=20), monitoring child (e.g., to ensure safety or monitor health conditions; 47%, n=17), and providing support or guidance for child regarding living with or managing achondroplasia (47%, n=17). For the family life domain, the most frequent impacts included family strain (56%, n=20), family vacations/travel (53%, n=19), and family activities (42%, n=15). In the work domain, the most frequent impact was missed work time (e.g., for doctor appointments; 50%, n=18).

Conclusion: The findings provide evidence for content validity of the parent experience measure for parents of children with achondroplasia aged 2 to <12 years. To fully evaluate the value of new treatments for achondroplasia, it is important to understand and assess impacts on parents' daily lives and well-being, which may be lessened following children's treatment.

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