Exploring the family experience of children aged 2-4 years receiving daily vosoritide injections: A qualitative study.

PURPOSE: Vosoritide is administered as a daily subcutaneous injection in children with achondroplasia. In clinical trials, families of children aged 2-4 years reported difficulty with drug administration due to child fear, pain, and distress. Study aims were to gain a better understanding of the current vosoritide administration experience in this cohort and to investigate whether topical anaesthesia and ice application prior to injections improved the child and family experience.

DESIGN AND METHODS: A qualitative descriptive study design ensured in-depth understanding of family experience. Parents were interviewed to explore experience of vosoritide administration for their child at two time points, before (Phase 1) and after (Phase 2) the introduction of topical anaesthesia and ice application prior to injections. Interviews were analysed using thematic analysis.

RESULTS: Seven families participated. Children's ages ranged from 2 years 2 months to 3 years 11 months. Five themes emerged from data analysis: (1) The reality of the burden of care; (2) Child experience as the greatest obstacle; (3) Parents juggle multiple emotional considerations; (4) Many factors may impact experience; and (5) Short-term and long-term impacts.

CONCLUSIONS: Administration of vosoritide in this cohort presents multiple challenges for families. Factors which influenced experience differed between families. Responses to topical anaesthesia and ice application also varied between children, improving administration experience for some children and worsening experience for others.

PRACTICE IMPLICATIONS: This study highlights the need for individualised care for young children receiving daily injections. Support should be provided to families to identify factors that improve experience.