2025 American Academy of Neurology Abstract Website

Haseeb Bin Najeeb¹, Aishwarya Koppanatham², Arman Amir³, Muhammad Sameer Butt⁴, Eisha Moazzam⁵, Azka Ijaz⁵, Ali Elbagir Ali Mohamed⁶, Hiba Ahmed Adam Omer⁶, Ayesha Azhar³, Alieu Kanu⁷, Shreesha Niraula⁸
¹MBBS, Allama Iqbal Medical College, ²Andhra Medical College, ³Dow University of Health Sciences, ⁴Combined Military Hospital, ⁵King Edward Medical University, ⁶University of Khartoum, ⁷University of Sierra Leone Teaching Hospitals Complex, ⁸Anwer Khan Modern Medical College

Objective:

This study evaluates the efficacy and safety of long-term use of eculizumab for refractory Myasthenia Gravis (MG).

Background:

Myasthenia Gravis (MG) is an autoimmune disorder characterized by antibodies directed against postsynaptic acetylcholine receptors, which block action potential generation and subsequently cause debilitating weakness in ocular and respiratory muscles. Drugs such as acetylcholine esterase inhibitors and immunosuppressives were previously used only for symptomatic relief. Eculizumab, a monoclonal antibody, exerts its therapeutic effect by inhibiting the formation of membrane attack complexes at neuromuscular junctions.

Design/Methods:

A systematic review was performed using electronic databases including PubMed, Embase, and Cochrane. Only controlled, randomised studies comparing eculizumab with placebo in patients with refractory generalised myasthenia gravis (gMG) were included. Eligible studies reported outcomes in patients with AChR+ serology, a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of at least 6, and prior immunosuppressive therapy (IST). Studies with overlapping patient populations and those without a control group were excluded.

Results:

This meta-analysis included four studies with a total of 161 patients. The MG-ADL and Quantitative Myasthenia Gravis (QMG) scores demonstrated significant heterogeneity, with mean differences of 18.117 (95% CI: -12.882 to 49.117; p < 0.001; I² = 100%) and 13.89 (95% CI: -27.154 to 54.937; p < 0.001; I² = 100%), respectively. In contrast, the Myasthenia Gravis Composite (MGC) and Myasthenia Gravis Quality of Life 15 (MGQOL15) scores showed low heterogeneity, with mean differences of 56.950 (95% CI: 56.158 to 57.742; p = 0.386; I² = 0%) and 55.091 (95% CI: 54.291 to 55.892; p = 0.328; I² = 0%), respectively.

Conclusions:

Eculizumab demonstrates significant efficacy in the treatment of refractory Myasthenia Gravis compared to previous treatments. The improvement in MG-ADL and QMG scores supports its effectiveness, despite some heterogeneity. The MGC and MGQOL15 scores, with lower heterogeneity, further support the efficacy of eculizumab.