Neurology. 2025 Apr 8;104(7_Supplement_1):1865. doi: 10.1212/WNL.0000000000208794. Epub 2025 Apr 7.
ABSTRACT
OBJECTIVE: To evaluate the correlation between Myasthenia Gravis (MG) Symptoms Patient-Reported Outcome (MG Symptoms PRO) scale scores and MG Activities of Daily Living (MG-ADL) and Quantitative MG (QMG) subdomain scores using data from the Phase 3 MycarinG study (NCT03971422) of rozanolixizumab in patients with generalized MG.
BACKGROUND: MG Symptoms PRO is a novel tool comprising five independent scales to measure MG symptoms: Muscle Weakness Fatigability, Physical Fatigue, Bulbar Muscle Weakness, Ocular Muscle Weakness and Respiratory Muscle Weakness.
DESIGN/METHODS: In MycarinG, patients were randomly assigned 1:1:1 to once-weekly subcutaneous rozanolixizumab 7 mg/kg, 10 mg/kg or placebo for 6 weeks. Outcome assessments included changes in MG-ADL (primary endpoint at Day 43), QMG and three MG Symptoms PRO scales. This post hoc analysis used MycarinG baseline scores (prior to treatment) to evaluate the correlation between MG Symptoms PRO scales and subdomains of MG-ADL and QMG scores (ocular, bulbar, respiratory and limb weakness/gross motor), using the Pearson coefficient between each pair of scores (strong: ≥0.7; moderate: 0.5-<0.7; weak: 0.3-<0.5).
RESULTS: Overall, 200 patients were enrolled in MycarinG. Correlation coefficients between MG Symptoms PRO scales and MG-ADL subdomain scores were strong between Ocular Muscle Weakness/ocular subdomain (0.78) and Bulbar Muscle Weakness/bulbar subdomain (0.72), and moderate between Respiratory Muscle Weakness/respiratory subdomain (0.58), Physical Fatigue/limb weakness subdomain (0.60) and Muscle Weakness Fatigability/bulbar subdomain (0.52). Correlation coefficients between MG Symptoms PRO scales and QMG subdomain scores were generally weak, with one moderate correlation between Bulbar Muscle Weakness/bulbar subdomain (0.50).
CONCLUSIONS: As expected, the five MG Symptoms PRO scales, which incorporate additional concepts such as physical fatigue, had stronger correlations with comparable concepts in MG-ADL and weaker correlations with QMG likely resulting from comparing patient- and clinician-reported data with different recall times. MG Symptoms PRO can complement existing MG-specific outcome measures to provide a granular assessment of the broad-ranging symptoms of MG. Disclosure: Dr. Pascuzzi has nothing to disclose. Dr. antozzi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. antozzi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for janssen. Dr. antozzi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Dr. antozzi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson Johnson. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alpine Immune Sciences. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Arcellx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron. Author has nothing to discloseDr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for LUPIN. Dr. Sacconi 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. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BIOGEN. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMICUS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. Sacconi 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. Sacconi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for FULCRUM THERAPEUTICS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for AXELYS. Dr. Sacconi has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for OLOGY. The institution of Dr. Sacconi has received research support from Solve FSHD. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Utsugisawa has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Viela Bio. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubisi Tanabe pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for argenx. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion phama. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Japan Blood Products Organization. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Biosciences. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Italfarmaco. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson (Janssen). Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Edgewise Therapeutics. The institution of Prof. Vissing has received research support from Rigshospitalet. The institution of Prof. Vissing has received research support from UCB Pharma. The institution of Prof. Vissing has received research support from Sanofi. The institution of Prof. Vissing has received research support from Roche. The institution of Prof. Vissing has received research support from AnJii. Dr. Regnault has received personal compensation for serving as an employee of Modus Outcomes. Dr. Bloemers has received personal compensation for serving as an employee of UCB. Dr. Bloemers has stock in UCB. Fiona Grimson has received personal compensation for serving as an employee of UCB Pharma. Fiona Grimson has stock in UCB Pharma. Dr. Heras has received personal compensation for serving as an employee of UCB Pharma S.A. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril 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. Bril has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril 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. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.
PMID:40194116 | DOI:10.1212/WNL.0000000000208794
