Approved in France since 2022, efgartigimod (Vyvgart®) is currently indicated for adults with generalised autoimmune myasthenia gravis with anti-RACh antibodies. A multicentre retrospective study conducted in China investigated its use in 17 children and adolescents (median age 13.4 years):
- efgartigimod (Vyvgart®) was initiated for an acute exacerbation of myasthenia or insufficient improvement despite treatment (pyridostigmine, immunomodulators),
- it led to rapid and clinically significant improvement in the majority of patients (70.6% in week 1, 73% in week 2, 91.7% in week 4),
- 66.7% of patients had achieved minimal symptom status by week 4.
It should be noted that clinical trials of efgartigimod are currently underway in the paediatric population, including in France.