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Abstract Details

PROMISE-MG: Comparative Effectiveness Study of MG Treatments- Baseline Data
Neuromuscular and Clinical Neurophysiology (EMG)
S56 - Neuromuscular Therapeutics (3:54 PM-4:06 PM)
003

Evidence for comparative effectiveness of immunosuppressive/ immunomodulating (IS/IM) treatments for myasthenia gravis (MG) is not available.

To present baseline data of participants in the PROMISE-MG study.

 

Multicenter, prospective, observational comparative effectiveness study comparing outcomes at 2 years in: 1. MG patients who receive azathioprine (AZT) vs. mycophenolate (MMF), 2. Patients receiving vs. not-receiving AZT/MMF at adequate doses/duration.

Inclusion criteria: autoimmune MG, age >18 years, no IS/IM treatment prior to initial study visit. Patients are treated as in routine clinical practice. Co-primary outcomes are MGQOL-15r, and a composite measure of achieving Minimal Manifestation status with no more than grade 1 CTCAE adverse effects.

De-identified data are entered into a central REDCap database. We used descriptive statistics to analyze initial visit data and Pearson correlation to measure the association between outcome measures.

One hundred sixty seven participants are enrolled from 19 centers: male 62%, 93% Caucasian. Average age at disease onset: 63±14 years (females 61± 17, males 65±11). Sixty percent have generalized MG. MGFA class: I - 40%, II - 37%, III - 20%, IV/V - 3%. Mean disease duration: 1.2± 2.4 years; 76% are AChR-Ab positive, 5% MuSK-Ab positive. Thymus imaging: normal/involuted - 88%, thymoma - 7%. At initial visit, 57% were on pyridostigmine. Treatment was started or changed in 73% (66% pyridostigmine, 23% corticosteroids).  Mean outcome measure scores at initial visit: MGQOL15r - 10.9±8.2, MG Composite (MGC) - 8.6±6.3, MG-MMT - 8.3±6.8, MG-ADL - 5.5± 3.3. Correlations between outcome measures were strong (r= 0.71 to 0.79) for MGC/MG-ADL and MGC/MG-MMT; all other measures correlated moderately with each other (r= 0.54 to 0.68), all p< 0.0001.

Participants in PROMISE-MG are predominantly male with disease onset in the 7th decade.  The highest correlations among outcome measures were between MGC/MG-ADL and MCC/MG-MMT, likely because the MG-ADL and MG-MMT are components of the MGC.

 Dr. Narayanaswami and Dr. Sanders are co-PIs and 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 as a Consultant for Amgen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CVS. 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 $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NMD pharma. Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for serono. 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 Dr. Reddys laboratories. Dr. Narayanaswami has stock in Pfizer. Dr. Narayanaswami has stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. The institution of Dr. Narayanaswami has received research support from Dianthus. Dr. Narayanaswami has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Council 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.
No disclosure on file