Abstract Details

Title The Evolution of Myasthenia Gravis-Activities of Daily Living (MG-ADL) Scale Utilization to Measure Myasthenia Gravis Symptoms and Treatment Response

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 098

Objective To summarize the published evidence of the MG-ADL in myasthenia gravis (MG) research and to describe how the use of the MG-ADL has changed over time.

Background The MG-ADL is an 8-item patient-reported scale that measures functional status related to MG.

Design/Methods A review of published literature was conducted to identify studies utilizing the MG-ADL and a database search of clinicaltrials.gov was conducted to identify Phase 2/3 clinical trials utilizing the MG-ADL.

Results A total of 48 publications and 35 clinical trials were included. The psychometric properties of the MG-ADL indicate that it is a valid and reliable measure of the impact of MG symptoms. The majority (74%) of Phase 2/3 clinical trials have used the MG-ADL as a secondary outcome and have utilized change in MG-ADL score from baseline as an outcome; however, its use as a primary outcome and the use of a responder threshold has increased in the past few years. Of trials beginning in 2017 or later, 41% use the MG-ADL as a primary outcome compared to 11% of trials prior to 2017. A 2-point responder definition has been established as a clinically meaningful response and is the most frequently utilized responder definition within trials. Most recently, minimal symptom expression (MSE), defined as an MG-ADL score of 0 or 1, has emerged as a new, promising outcome, though further research is needed to validate this construct.

Conclusions

The MG-ADL is a validated and meaningful scale that is a well-established outcome in clinical trials and real-world studies to measure MG functional status and treatment response. Studies utilizing the MG-ADL have used change in total score, responder thresholds, and more recently, MSE to measure treatment response. Given the clinical importance of achieving minimal manifestations, MSE is a promising outcome in MG research.

Authors/Disclosures
Srikanth Muppidi, MD, F�鶹��ýӳ�� PRESENTER	Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for J & J pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Nicholas J. Silvestri, MD, F�鶹��ýӳ�� (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
	No disclosure on file
Trevor Leighton	Trevor Leighton has received personal compensation for serving as an employee of argenx BV. Trevor Leighton has received stock or an ownership interest from argenx BV.

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