Retrospective Analysis of Eculizumab in Patients with Acetylcholine Receptor Antibody-Negative Myasthenia Gravis: A Case Series.
BACKGROUND
The role of the complement cascade in acetylcholine receptor antibody-negative (AChR-) myasthenia gravis (MG) is unclear.


OBJECTIVE
To assess the efficacy and tolerability of eculizumab (terminal complement inhibitor) in patients with AChR-MG.


METHODS
Retrospective chart review of data from six patients treated for 12 months with eculizumab for treatment-refractory, AChR-(by radioimmunoassay) generalized MG (gMG). The eculizumab dose was 900 mg/week for 4 weeks then 1200 mg every 2 weeks. Outcome measures were Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores, number of exacerbations, and qualitative physical assessments based on selected items of the Quantitative Myasthenia Gravis evaluation (ptosis, double vision, eye closure, duration of ability to stretch out limbs).


RESULTS
All patients were female (mean age, 50.8 years). In the 12 months before eculizumab initiation, all measures were relatively stable. After its initiation, clinically meaningful reductions (≥2 points) in total MG-ADL scores were observed before or at 5 months and were maintained to Month 12 in all patients; mean (standard deviation [SD]) scores were 11.3 (0.9) and 5.0 (0.9), respectively. There was also a reduction in the mean (SD) number of exacerbations per patient, from 2.8 (1.2) to 0.3 (0.5) in the 12 months before and after eculizumab initiation, respectively. Physical assessment ratings were improved in all patients. Adverse events were reported in four patients, but all were mild and none were treatment-related.


CONCLUSIONS
This small retrospective analysis provides preliminary evidence for the efficacy of eculizumab in treatment-refractory gMG that was AChR-according to radioimmunoassay. Larger, more robust studies are warranted to evaluate this further.
['Datta|Sorabh|S|', 'Singh|Shivangi|S|', 'Govindarajan|Raghav|R|']
D000203:Activities of Daily Living D000328:Adult D061067:Antibodies, Monoclonal, Humanized D051056:Complement Inactivating Agents D005260:Female D006801:Humans D008875:Middle Aged D009157:Myasthenia Gravis D017063:Outcome Assessment, Health Care D011950:Receptors, Cholinergic D012189:Retrospective Studies
Myasthenia gravis ACh receptors activities of daily living complement inactivating agents corticosteroids neuromuscular junction