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Abstract Details

Subcutaneous Immunoglobulin in Myasthenia Gravis: Results of a North American Open Label Study
Neuromuscular and Clinical Neurophysiology (EMG)
N4 - Neuroscience in the Clinic: Immunotherapies in Neurological Disease (2:15 PM-2:30 PM)
002

Background: IVIg has been demonstrated to improve the Quantitative MG (QMG) score as compared to placebo and in another study to be efficacious in comparison to plasma exchange. In routine care, SCIg might be easier to administer than IVIg.

 

Objectives: To evaluate the efficacy, safety and tolerability of subcutaneous immunoglobulin (SCIg) in the treatment in myasthenia gravis (MG) patients who are on IVIg as part of routine clinicalcare.

 

Methods: This multi-center North American open label prospective investigator-initiated study had 2 components: IVIg Screening Phase (ISP; Weeks -10 to -1) followed by Experimental Treatment Phase (ETP; Weeks 0 to 12). We hypothesized that more than 65% of the patients entering the ETP would have stable QMG score (primary outcome) at Week 12. We recruited 23 patients in the ISP and 22 entered the ETP. 12/22(54.5%) were females and 18(78%) were white, with mean age 51.4 years±17. We had complete ETP QMG data on 19/22; one subject withdrew from ISP owing to worsened condition and two subjects withdrew before Week 4 (needle dislike). The per protocol primary statistical analysis was conducted for n=22 subjects using a one-sided z-test of proportions

at the 5% significance level. Sensitivity analyses were conducted using a cohort of n=22 subjects using ‘worst-case’ imputation scenario as well as post hoc analysis. 

 

Findings: On primary analysis, 19/22 (86.4%; 95% CI:0.72–1.00)were treatment “successes” (p=0.018). Sensitivity analysis using the ‘worst-case’ imputation resulted in 17/22 (77.3%;0.60–0.95) declared as treatment success (p=0.114).Post hoc analysis of the primary outcome confirmed treatment success

in 17/20 (85%;0.69– 1.00) (p=0.0304). There was no difference in the secondary outcome measures though MG composite was better at Week 12. SCIg was safe and well tolerated in this population.

 

Conclusion: Most MG patients who were doing well on IVIg maintained disease stability for another 12 weeks once transitioned to SCIg. 

Authors/Disclosures
Mazen M. Dimachkie, MD, FAAN (University of Kansas Medical Center)
PRESENTER
Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amicus. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fortrea. The institution of Dr. Dimachkie has received research support from NIH. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care.
Vera Bril, MD (Toronto General Hospital) Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril 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. Bril has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care.
Todd D. Levine, MD (Honor Health) Dr. Levine has received personal compensation for serving as an employee of CND life sciences . Dr. Levine has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Nufactor. Dr. Levine has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for PNA. Dr. Levine has stock in CND Life Sciences. Dr. Levine has stock in Corinthian reference lab.
Jaya R. Trivedi, MD, FAAN (UT Southwestern Medical Center) The institution of Dr. Trivedi has received research support from Avidity.
Nicholas J. Silvestri, MD, FAAN (UBMD Neurology) Dr. Silvestri 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. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron. Dr. Silvestri 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. Silvestri 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. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Dr. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. The institution of an immediate family member of Dr. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB. Dr. Silvestri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Takeda. Dr. Silvestri has received publishing royalties from a publication relating to health care.
No disclosure on file
David S. Saperstein, MD (Center for Complex Neurology, EDS & POTS) No disclosure on file
Sharon P. Nations, MD, FAAN (UT Southwestern Med School) No disclosure on file
Hans D. Katzberg, MD, FAAN An immediate family member of Dr. Katzberg has received personal compensation for serving as an employee of Alexion. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Katzberg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Dr. Katzberg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Merz. Dr. Katzberg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson and Johnson. Dr. Katzberg has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Syneos Health. Dr. Katzberg 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. Katzberg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX.
Gil I. Wolfe, MD, FAAN (Univ. At Buffalo, SUNY) Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Grifols. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ArgenX. Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/Ra. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Wolfe has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Grifols. The institution of Dr. Wolfe has received research support from UCB/Ra. The institution of Dr. Wolfe has received research support from Immunovant. The institution of Dr. Wolfe has received research support from Roche. Dr. Wolfe has received personal compensation in the range of $0-$499 for serving as a Advisor with FDA Advisory Panel for Cellular, Tissue and Genetic Therapies.
Laura Herbelin (KU Medical Center) No disclosure on file
No disclosure on file
Andrew Heim Andrew Heim has nothing to disclose.
April L. McVey, MD (April L. McVey, MD) No disclosure on file
No disclosure on file
Jeffrey Statland, MD (University of Kansas Medical Center) Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
Richard J. Barohn, MD, FAAN (University of Missouri) Dr. Barohn has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for NuFactor. The institution of Dr. Barohn has received research support from FDA OPD R01. Dr. Barohn has received intellectual property interests from a discovery or technology relating to health care.
Richard J. Barohn, MD, FAAN (University of Missouri) Dr. Barohn has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for NuFactor. The institution of Dr. Barohn has received research support from FDA OPD R01. Dr. Barohn has received intellectual property interests from a discovery or technology relating to health care.
Mamatha Pasnoor, MD, FAAN (University of Kansas Medical Center) Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx BVBA. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen Pharmaceutical. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for amgen.