Clinical outcomes, serum IgG changes, and follow-up observations in the treatment of anti-AChR antibody-positive generalized myasthenia gravis with Efgartigimod
摘要
To investigate the clinical effects, changes in serum IgG levels, and follow-up observations following treatment with Efgartigimod in patients with anti-acetylcholine receptor (anti-AChR) positive generalized myasthenia gravis (gMG).
MethodsThis was a single-center retrospective study. Clinical data and follow-up information were collected for 8 patients with anti-AChR positive gMG treated with Efgartigimod. Clinical severity was assessed before and after treatment using the Quantitative Myasthenia Gravis (QMG) Score and the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale. Serum levels of immunoglobulins IgG, IgA, and IgM were also measured before and after treatment.
ResultsThe cohort consisted of 8 patients (4 male, 4 female), aged 21 to 73 years (ages: 55, 21, 63, 72, 62, 69, 64, 73). The clinical manifestations included external ophthalmoplegia in 8 cases, dysphagia in 7, weakness of the neck muscles in 8, weakness of the limbs in 7, pre-crisis status in 2, and one case of a myasthenic crisis. Disease duration ranged from 3 months to 20 years. At admission, the QMG scores ranged from 5 to 29, and the MG-ADL scores ranged from 3 to 19. Three patients had coexisting autoimmune diseases. After Efgartigimod treatment, all patients showed rapid improvement in clinical symptoms. A clinically meaningful reduction (CMR) in the MG-ADL score was achieved in 100% of patients, with 87.5% achieving a CMR in the QMG score. Notably, 87.5% of patients were responders to both measures. Serum IgG, IgA and IgM levels were measured in six patients throughout the treatment period. While IgG levels showed a rapid and marked reduction post-treatment, IgA and IgM levels remained largely unchanged. The follow-up period ranged from 190 to 414 days (median: 380.5 days). During follow-up, 6 patients achieved minimal manifestation status with only grade 1 adverse events according to CTCAE. One patient experienced symptom recurrence after voluntarily discontinuing treatment, and another showed symptom exacerbation due to a concurrent respiratory infection.
ConclusionEfgartigimod has been shown to rapidly improve clinical symptoms in patients with generalised myasthenia gravis, while also significantly reducing serum IgG levels. When used alongside immunosuppressants, it can help to maintain long-term control of the disease.