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Abstract Details

Comparison of the Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scores in the MGTX Randomized Trial
Autoimmune Neurology
P2 - Poster Session 2 (5:30 PM-6:30 PM)
15-102
To qualitatively and quantitatively compare the performance of QMG and MG-ADL measures during the MGTX trial.
QMG and MG-ADL are the two most common measures of Myasthenia Gravis (MG) disease severity, but no gold standard has yet been established.  The MGTX trial collected longitudinal MG-ADL and QMG measures on patients randomized to thymectomy plus prednisone (TPP) or prednisone alone (PA).

MG-ADL and QMG disease measures were analyzed by treatment group and follow-up time for the 118 patients with at least one follow-up visit using generalized linear models with repeated measures (GLMs) and autocorrelation [AR(1)] covariance.  Models were compared to determine if both measures similarly captured the group difference and time trend in disease status. Model distributional assumptions were also evaluated.

 

Correlations between the measures were examined for the 123 patients having any observations. Multivariate relationships between QMG and MG-ADL means, standard deviations, and observed correlations were examined graphically and using multivariate ANOVA.

 

Assuming normality for QMG appears adequate, but MG-ADL is better fit by a Poisson distribution. Treatment group and time were significant (p<0.05) in both models. The AR(1) parameter was similarly high for QMG (0.8448) and MG-ADL (0.7225).  Visually the QMG model fits better overall and especially for TPP patients.

 

The baseline correlation between the measures was 0.65 (TPP) and 0.53 (PA).  The increasing multivariate relationship describing the observed correlation between the two measures and their means (HLT p<0.0001) and standard deviations (HLT p<0.0001) at each time point suggests the measures perform more similarly when the disease is less well controlled.

 

QMG and MG-ADL are clearly correlated and appear to perform similarly in measuring MG severity. The model curves and AR(1) coefficients are similar reflecting treatment modifications across study visits. Normality assumptions apply in modeling QMG, thereby facilitating statistical analyses and enabling a wider range of model assessments.
Authors/Disclosures
Tarrant McPherson
PRESENTER
Tarrant McPherson has received research support from Myasthenia Gravis Foundation of America.
Immaculada Aban No disclosure on file
Petra W. Duda, MD, PhD Dr. Duda has received personal compensation for serving as an employee of UCB. Dr. Duda has stock in UCB.
Ramin Farzaneh-Far No disclosure on file
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.
Henry J. Kaminski, MD, FAAN (George Washington University) Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Kaminski has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta Bio. The institution of Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Canopy Immunotherapeutics. Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Curie.bio. Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Samsung. Dr. Kaminski has received personal compensation in the range of $0-$499 for serving as a Consultant for Care Constitution. Dr. Kaminski has stock in mimivax. The institution of Dr. Kaminski has received research support from National Institutes of Health. Dr. Kaminski has received publishing royalties from a publication relating to health care.
Gary R. Cutter, PhD (University of Alabama At Birmingham) Dr. Cutter has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for onsulting or Advisory Boards: Alexion, Antisense Therapeutics/Percheron, Avotres, Biogen, Clene Nanomedicine, Clinical Trial Solutions LLC, Endra Life Sciences, Cognito Therapeutics, Genzyme, Genentech, Immunic, Klein-Buendel Incorporated, Kyverna Therapeutics, Inc. , Linical, Merck/Serono, Noema, Neurogenesis, Perception Neurosciences, Protalix Biotherapeutics, Regeneron, Revelstone Consulting, Roche, SAB Biotherapeutics, Sapience Therapeutics, Scott&Scott LLP, Tenmile.. Dr. Cutter has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Data and Safety Monitoring Boards: Applied Therapeutics, AI therapeutics, AMO Pharma, Argenx, Astra-Zeneca, Avexis Pharmaceuticals, Bristol Meyers Squibb, CSL Behring, Cynata Therapeutics, DiamedicaTherapeutics, Horizon Pharmaceuticals, Immunic, Inhibrix, Karuna Therapeutics, Kezar Life Sciences, Medtronic, Merck, Meiji Seika Pharma, Mitsubishi Tanabe Pharma Holdings, Prothena Biosciences, Novartis, Pipeline Therapeutics (Contineum), Regeneron, Sanofi-Aventis, Teva Pharmaceuticals, United BioSource LLC, University of Texas Southwestern.. Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for JASN.