Good practice in Treatment of Autoimmune Myasthenia Gravis in Children

Myasthenia gravis (MG) in pediatric patients presents unique diagnostic and therapeutic challenges, requiring concise approaches distinct from adult MG. Pediatric MG is primarily autoimmune, with acetylcholine receptor (AChR) antibodies detected in ۵۰-۷۰% of juvenile cases, while anti-MuSK and anti-LRP۴ are less common. Clinical hallmarks include fatigable ptosis, ophthalmoparesis, and bulbar weakness, though generalized weakness may develop. Electrodiagnostic studies (repetitive nerve stimulation, single-fiber EMG) and a cautious edrophonium test can support diagnosis, but seronegative cases necessitate genetic testing to exclude CMS. Treatment Strategies: First-line symptomatic therapy involves pyridostigmine (۱-۲ mg/kg/dose every ۴-۶ hours), with close monitoring for cholinergic toxicity. Immunomodulation is often required for generalized or refractory disease: Corticosteroids (e.g., prednisone ۱-۲ mg/kg/day) are effective but require gradual tapering to mitigate growth suppression and metabolic effects. Intravenous immunoglobulin (IVIG) and plasma exchange (PLEX) serve as rescue therapies for acute exacerbations or myasthenic crisis. Steroid-sparing agents (azathioprine, mycophenolate mofetil) are used for long-term control, while rituximab may benefit anti-MuSK-positive cases. Thymectomy is controversial in prepubertal children but may be considered in AChR-positive generalized MG adolescents, mirroring adult protocols. Emerging therapies (eculizumab, efgartigimod) show promise but lack robust pediatric data. Neonatal MG, caused by placental transfer of maternal antibodies, typically resolves within weeks but may require transient pyridostigmine. Multidisciplinary care including respiratory support, physical therapy, and psychosocial support is vital for optimizing outcomes. Conclusion: Pediatric MG management demands early diagnosis, individualized immunotherapy, and careful monitoring of treatment-related complications. Future research should focus on pediatric-specific trials for novel biologics and refined thymectomy guidelines.

کلیدواژه ها:

Myasthenia gravis ، treatment ، Children