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Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia
Sinead NiMhurchadha1, Karen Butler2, Rob Argent3
1S3 Connected Health, Dublin, Ireland. Sinead.NiMhurchadha@S3Connectedhealth.com.
Insights
Parents and children manage daily vosoritide injections for achondroplasia with resilience, motivated by improved health and independence. Greater support is recommended for treatment initiation and home management.
Area of Science:
- Pediatric Endocrinology
- Rare Genetic Disorders
- Pharmacological Treatments
Background:
- Achondroplasia is a genetic disorder affecting bone growth.
- Vosoritide is the first approved pharmacological treatment for achondroplasia.
- Treatment involves at-home injectable administration.
Purpose of the Study:
- To explore parents' and children's experiences with initiating and administering vosoritide at home.
- To understand the challenges and facilitators of at-home vosoritide treatment for achondroplasia.
Main Methods:
- Qualitative telephone interviews were conducted with 15 parents of children undergoing vosoritide treatment in France and Germany.
- Interviews were transcribed and analyzed using thematic analysis.
Main Results:
- Parents learned about vosoritide through research, advocacy groups, or physicians.
- Decisions to treat were based on relieving medical complications and improving independence, considering side effects.
- Hospital initiation and training varied significantly across treatment centers.
- Families faced psychological and practical challenges managing treatment at home, overcome with perseverance and support.
Conclusions:
- Parents and children demonstrate resilience and motivation in managing daily injectable vosoritide treatment.
- Families prioritize long-term health and functional independence over short-term treatment challenges.
- Enhanced support is crucial for effective treatment initiation and home management, improving patient experience.
Introduction:
Vosoritide is the first approved pharmacological treatment for achondroplasia and is indicated for at-home injectable administration by a trained caregiver. This research aimed to explore parents' and children's experience of initiating vosoritide and administering this treatment at home.
Methods:
Qualitative telephone interviews were conducted with parents of children being treated with vosoritide in France and Germany. Interviews were transcribed and analysed using thematic analysis.
Results:
Fifteen parents participated in telephone interviews in September and October 2022. The median age of children in this sample was 8 years old (range 3-13 years) and children had been taking treatment from 6 weeks to 13 months. Four themes document families' experience with vosoritide: (1) awareness of vosoritide treatment, uncovering that parents first heard of vosoritide through their own research, patient advocacy groups, or through their physicians; (2) treatment understanding and decision-making, which found that their decision to take treatment is based on a desire to relieve future medical complications and increase height for improved independence, and they consider the extent to which the treatment has severe side effects; (3) training and initiation, which showed that the hospital initiation and training sessions varied considerably both across and within countries, with different treatment centres taking different approaches; and (4) managing treatment at home brings psychological and practical challenges, which are ultimately overcome with perseverance and available support.
Conclusions:
Parents and children are resilient to challenges posed by a daily injectable treatment and highly motivated to improve their quality of life. Parents are prepared to overcome short-term treatment challenges for future gains in terms of health and functional independence for their children. Greater support could ensure they have the right information to initiate treatment and manage treatment at home, which will improve parents' and children's experience.
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