Abstract Details

Title Burden of Disease in Patients with Refractory Myasthenia Gravis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P5 - Poster Session 5 (5:30 PM-6:30 PM)

Poster/Presentation
Number 12-037

Objective

To explore the demographics, disease characteristics and burden in refractory myasthenia gravis (MG).

Background

Approximately 10-15% of patients have difficult to control disease and are termed as ‘refractory’. The purpose of our study was to further evaluate clinical characteristics, disease burden and financial impact on this population.

Design/Methods This study was a retrospective medical record review of patients with confirmed MG that had been included in the Exploring Outcomes and Characteristics of Myasthenia Gravis (EXPLORE-MG) Registry and followed at the Yale MG Clinic from 2003 to 2018. Information collected include demographics, antibody status, disease phenotype, worst/last MGFA clinical class, thymoma/thymectomy status, history of autoimmunity, age at diagnosis, therapy utilized, hospitalizations, and financial impact of disease. Pre-specified criteria were used to classify the refractory and non-refractory groups.

Results Sixty-two (21%) out of 297 patients were classified as refractory, and further characterized as predominantly women (64.5%) with early-onset generalized MG (p-value <0.001). Refractory patients were more likely to be MuSK antibody positive (p-value <0.001), have a history of prior thymectomy (p-value <0.001) and have coexisting autoimmune disease (p-value 0.02). A worst MGFA class of V was observed in 41% of the refractory group versus 8% in the non-refractory group (p-value <0.001). The number of patients hospitalized ≥1 time(s) and ≥3 times was 74.2% versus 28.5% and 25.8% versus 2% in the refractory and non-refractory groups respectively (p-values <0.001, <0.001). Differences in healthcare utilization, use of rescue therapy and chronic IVIg/PLEX among other variables was observed, consistent with a higher financial burden in the refractory group.

Conclusions Patients with refractory MG have key clinical characteristic differences including a greater burden of disease. Understanding disease burden from both a quality of life and financial perspective as well as further characterizing it remains a high priority as treatment paradigms emerge for patients with difficult to manage MG.

Authors/Disclosures
Aditi Sharma, MBBS (University of Utah) PRESENTER	Dr. Sharma has nothing to disclose.
Bhaskar Roy, MD, FAAN (Yale University)	Dr. Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Roy has stock in Cabaletta bio. . Dr. Roy has stock in Pfizer. Dr. Roy has stock in CAVA. The institution of Dr. Roy has received research support from Abcuro Pharmaceuticals. The institution of Dr. Roy has received research support from Immunovant. The institution of Dr. Roy has received research support from argenx.
Babar Khokhar, MD, FAAN (Brown University Health)	Dr. Khokhar has nothing to disclose.
Richard J. Nowak, MD (Yale University School of Medicine)	Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Nowak has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amgen. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janseen . The institution of Dr. Nowak has received research support from UCB. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Janseen. The institution of Dr. Nowak has received research support from Immunovant . The institution of Dr. Nowak has received research support from argenx. The institution of Dr. Nowak has received research support from Amgen. Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.

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