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Review
. 2022 Jun;65(6):630-639.
doi: 10.1002/mus.27476. Epub 2022 Jan 6.

Utilization of MG-ADL in myasthenia gravis clinical research and care

Srikanth Muppidi  1 , Nicholas J Silvestri  2 , Robin Tan  3 , Kimberly Riggs  3 , Trevor Leighton  4 , Glenn A Phillips  5
Affiliations
Review

Utilization of MG-ADL in myasthenia gravis clinical research and care

Srikanth Muppidi et al. Muscle Nerve. 2022 Jun.

Abstract

The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures myasthenia gravis (MG) symptoms and functional status. The objective of the current review is to summarize the psychometric properties of the MG-ADL and published evidence of MG-ADL use. A targeted literature review for published studies of the MG-ADL was conducted using a database and gray literature search. A total of 48 publications and 35 clinical trials were included. Studies indicated that the MG-ADL is a reliable and valid measure that has been used as an outcome in clinical trials and observational studies to measure MG symptoms and response to treatment. While most often used as a secondary endpoint in clinical trials, its use as a primary endpoint has increased in recent years. The most common MG-ADL endpoint is change in MG-ADL score from baseline, although there has been an increase in the analysis of a responder threshold using the MG-ADL. A new concept of minimal symptom expression (MSE) has emerged more recently. Duration of treatment effect is another important construct that is being increasingly evaluated using the MG-ADL. The use of the MG-ADL as a primary endpoint in clinical trials and in responder threshold analyses to indicate treatment improvement has increased in recent years. MSE using the MG-ADL shows promise in helping to determine success of treatment and may be the aspirational goal of MG treatment for the future once validated, particularly given the evolving treatment landscape in MG.

Keywords: MG symptoms; MG-ADL; minimal symptom expression; myasthenia gravis; neuromuscular disorder.

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Conflict of interest statement

This review was funded by argenx. S. Muppidi has served as a paid consultant for Alexion Pharmaceuticals, argenx, and Ra Pharmaceuticals. N. Silvestri has served as a paid consultant for Alexion Pharmaceuticals, argenx, and UCB. R. Tan and K. Riggs are employees of Xcenda, which was paid by argenx to conduct the literature review upon which this manuscript is based and to help prepare the manuscript. T. Leighton is a former employee of and owns stock in argenx. G. Phillips is a current employee of and owns stock in argenx. The study concept was initiated by T. Leighton. S. Muppidi and N. Silvestri provided guidance during the review process on concepts that should be addressed. R. Tan and K. Riggs conducted the review. S. Muppidi and N. Silvestri provided input on the results and interpretation of the review. G. Phillips provided input on measurement and psychometric concepts in the review. All authors contributed to the writing, review processes, and final approval of the manuscript.

Figures

FIGURE 1
FIGURE 1
MG‐ADL as a primary or secondary endpoint in clinical trials by year
See this image and copyright information in PMC

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