Abstract Details

Title PROMISE-MG: A Multicenter Prospective Observational Comparative Effectiveness Study of Myasthenia Gravis Treatments

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S29 - Preclinical and Observational Studies of Neuromuscular Diseases (1:00 PM-1:12 PM)

Poster/Presentation
Number 001

Background

PROMISE-MG is the first comparative effectiveness study of MG immunosuppressive treatments.

Objective

We aimed to compare patient-reported and clinician-reported outcomes in 1. MG patients receiving azathioprine (AZT) vs. mycophenolate mofetil (MMF); 2. Patients receiving adequate doses and duration of AZT or MMF vs. not receiving adequate doses/duration.

Design/Methods This was a prospective multicenter cohort study. Patients were treated per their physician’s judgment and followed up to 36 months. The primary outcome measure was the Myasthenia Gravis- Quality of Life-15, revised (MGQOL-15r). The Co-primary outcome was a composite measure (achieving MGFA Post-Intervention Status Minimal Manifestations or better with Common Terminology Criteria for Adverse Events of treatment ≤ Grade 1). A weighted generalized linear regression model with propensity score overlap weighting was used to estimate treatment effects after accounting for measured confounding.

Results

One hundred and sixty-seven patients were enrolled. Eighty-two received either MMF or AZT. A similar proportion of participants receiving either treatment had clinically meaningful improvement in MGQOL-15r (≥5 point reduction: MMF -75%, AZT -65%, weighted difference 3% favoring MMF, 95% CI -22% to 15.8%, p=0.8, mean difference in maximum improvement 2.6 points favoring MMF, CI 0.42 to 4.85, p=0.02). Two percent more participants receiving MMF achieved the co-primary outcome measure (95% CI -15% to 19%, p=0.8). The proportion of participants achieving meaningful improvement in MG-QOL15r were similar in those who did or did not receive adequate treatment dose/duration (71% v. 70.6%, weighted difference in proportions 5% favoring MMF, 95% CI -24% to 14%, p=0.6).

Conclusions

Patients receiving either MMF or AZT achieved clinically meaningful improvements, and there was no difference between them. Those who received adequate dose/duration of these drugs achieved similar improvement to those who did not. Further analyses will evaluate secondary outcomes, adverse effects of AZT/MMF and the effective doses of these agents in real-world practice.

Narayanaswami and Sanders contributed equally

Authors/Disclosures
Pushpa Narayanaswami, MD, MBBS, FAAN (Beth Israel Deaconess Medical Center) PRESENTER	Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami 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. Narayanaswami 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 Muscle and Nerve, Wiley. Dr. Narayanaswami has stock in Moderna. Dr. Narayanaswami has stock in Dr. Reddys laboratories. Dr. Narayanaswami has stock in Pfizer. Dr. Narayanaswami has stock in Viatris. Dr. Narayanaswami has stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Advisory Board with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities.
Donald B. Sanders, MD, FAAN (Duke University Medical Center)	Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Accordant Health Services. Dr. Sanders has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta BIO. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kashiv Biosciences. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Momenta Pharmaceuticals. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen RnD. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi-aventis. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for VielaBio. Dr. Sanders has received publishing royalties from a publication relating to health care.
Jeff Guptill, MD, FAAN (argenx US)	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx.
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