Triple-Seronegative Juvenile Myasthenia Gravis Treated With Efgartigimod Alfa: Diagnostic Challenges and Therapeutic Decisions in the Era of Novel Treatments
Charalampos Tzempetzis, Chrysanthi Tsimakidi, Eirini Topoulli, Dionysia Gkougka, Maria GontikaBackground
Juvenile myasthenia gravis (JMG) is a rare autoimmune disorder with limited pediatric evidence and treatment extrapolated from adults. Seronegative disease complicates diagnosis, whereas prolonged corticosteroid use frequently results in substantial toxicity, highlighting the need for steroid-sparing therapies. Although adult studies support the efficacy of efgartigimod alfa, its role in pediatric and seronegative disease remains uncertain.
Methods
We present a case of triple-seronegative JMG successfully treated with efgartigimod alfa, accompanied by a focused literature review of current diagnostic and therapeutic challenges.
Results
A female adolescent with aggressive generalized JMG refractory to corticosteroids and intravenous immunoglobulin. Efgartigimod produced rapid, sustained clinical improvement, enabling corticosteroid tapering, and was well tolerated. This review contextualizes these findings within the evolving evidence for diagnosis and FcRn-targeted therapy in JMG.
Conclusions
This case-based review highlights the diagnostic complexity of seronegative JMG and supports further evaluation of FcRn-targeted therapy in pediatric patients.