Invited Review: Utilization of MG-ADL in myasthenia gravis clinical research and care
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The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures myasthenia gravis (MG) symptoms and functional sta-tus. The objective of the current review is to summarize the psychometric proper-ties 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 end-point 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 pri-mary endpoint in clinical trials and in responder threshold analyses to indicate treat-ment 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.
Objectives:
1) Be able to incorporate the Myasthenia Gravis Activities of Daily Living scale (MG-ADL) as an outcome measure in clinical practice;
2) Be able to incorporate the MG-ADL as an outcome measure in research;
3)Understand and be able to apply the concept of minimal symptom expression in the assessment of patients with MG;
ACCREDITATION STATEMENT The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
CREDIT DESIGNATION The AANEM designates this enduring material for a maximum of 1
AMA PRA Category 1 Credits TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Credit expires 6/10/2025.
DISCLOSURE INFORMATIONThis review was funded by argenx. S. Muppidi has served as a paid consultant for Alexion Pharmaceuticals, argenx, and Ra Pharmaceu-ticals. N. Silvestri has served as a paid consultant for Alexion Phar-maceuticals, 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. Any conflicts have been resolved by the Journal in accordance with the Muscle & Nerve editorial process.
FORMATPDF
Srikanth Muppidi, MD; Nicholas J. Silvestri, MD; Robin Tan, PhD; Kimberly Riggs, MPH; Trevor Leighton, MBA, BSc; Glenn A. Phillips PhD