Early Experience and Practical Considerations for Clinical Practice

Description

Vosoritide▼ Therapy in Children with Achondroplasia

A group of experts with early experience of vosoritide conclude that the early real-world experience is generally positive, but that sharing key insights will help to increase understanding and highlight optimal ways to manage vosoritide in clinical practice.

Two meetings were held to gather expert insight and experiences. This included geneticists, paediatric endocrinologists, paediatricians, and orthopaedic surgeons from across Europe, America, and the Middle East. Current practices and considerations were discussed, including administration, assessments, and how to manage patient expectations. At the time of the meetings the group was collectively managing over 220 ACH patients on vosoritide.

The findings suggest that a crucial management step is to determine whether adequate multidisciplinary support is in place. Training for families is also considered essential, and this should encompass practical information on administration, recognising and managing injection-site reactions, and the importance of adherence. Equally important is that patients and families should be informed that response to vosoritide can vary in both magnitude and timing, and realistic expectations should be set.

Sharing these insights will increase understanding of vosoritide practicalities, but international consensus guidance is needed to support use of vosoritide in clinical practice.