Abstract Details

Title Eculizumab in the Treatment of Seronegative Refractory Generalized Myasthenia Gravis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P3 - Poster Session 3 (12:00 PM-1:00 PM)

Poster/Presentation
Number 1-013

Objective To assess the efficacy of Eculizumab in seronegative, refractory generalized MG

Background Myasthenia gravis (MG) is an antibody-mediated disorder that targets the neuromuscular junction (NMJ), resulting in fatigable weakness that affects ocular, bulbar, respiratory and limb muscles. Considerable improvement has been made in the treatment of patients with MG; however, options depend on seropositivity for anti-acetylcholine receptor (anti-AchR) and/or anti-muscle specific kinase (anti-MuSK) antibodies. Eculizumab, a monoclonal antibody that inhibits the complement component C5 and prevents activation of the membrane attack complex (MAC), is approved for treatment of generalized, AChR-positive MG.

Design/Methods Six patients (6 F, 50.8 ± 10.1 yr) with seronegative refractory generalized MG were treated with Eculizumab and followed for 6 months. Myasthenia Gravis – Activities of Daily Living (MG-ADL) scores, frequency of exacerbations and need for other medications were recorded. All patients had been treated with pyridostigmine in the past 6 months and five had also been received prednisone. Other treatments received in the past 6 months were azathioprine, mycophenolate, IVIG and PLEX.

Results MG-ADL scores six months following treatment decreased in all patients treated with Eculizumab. Mean MG-ADL scores at 1 and 6 months following treatment were 11. 33 ± 1.03 and 8.17 ± 0.41, respectively. Additionally, all patients had a reduction in the number of exacerbations following treatment – from an average of 3 exacerbations in the year before treatment to 0.33 in the six months after. Four out of six patients saw a reduction in their prednisone dose in the six months following treatment, with three out of six patients coming off of intravenous immunoglobulin after treatment.

Conclusions

This small analysis provided preliminary evidence for the efficacy of Eculizumab in the treatment of refractory generalized MG. Larger, more robust studies are warranted to evaluate this further.

Authors/Disclosures
Shivangi Singh, MD (Columbia Neurology) PRESENTER	Dr. Singh has nothing to disclose.
Harsh Singh	No disclosure on file
	No disclosure on file
Raghav Govindarajan, MD, FAAN (HSHS St. Elizabeth Medical Group)	Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT pharma. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for MT pharma . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Catalyst. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Argenx. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biohaven. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Takeda. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Abbvie. The institution of Dr. Govindarajan has received research support from Band of Hope . The institution of Dr. Govindarajan has received research support from Alexion. Dr. Govindarajan has received publishing royalties from a publication relating to health care.

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