Clinically Meaningful Improvement in Physical Fatigue and Muscle Weakness Fatigability with Rozanolixizumab: Post Hoc Analysis of MG Symptoms PRO Responder Rate in the MycarinG study

Annabel Dimmock

Clinically Meaningful Improvement in Physical Fatigue and Muscle Weakness Fatigability with Rozanolixizumab: Post Hoc Analysis of MG Symptoms PRO Responder Rate in the MycarinG study

Autori

FIAMMETTA VANOLI (DEPARTMENT OF NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES, FONDAZIONE IRCCS, INSTITUTO NAZIONALE NEUROLOGICO CARLO BESTA, MILAN, ITALY; DEPARTMENT OF HUMAN NEUROSCIENCES, SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
JULIAN GROSSKREUTZ (PRECISION NEUROLOGY OF NEUROMUSCULAR DISEASES, DEPARTMENT OF NEUROLOGY, UNIVERSITY OF LÜBECK, LÜBECK, GERMANY – NEUROLOGY)
ALI AAMER HABIB (MDA ALS AND NEUROMUSCULAR CENTER, UNIVERSITY OF CALIFORNIA, IRVINE, IRVINE, CA, USA – NEUROLOGY)
RENATO MANTEGAZZA (DEPARTMENT OF NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES, FONDAZIONE IRCCS, INSTITUTO NAZIONALE NEUROLOGICO CARLO BESTA, MILAN, ITALY – NEUROLOGY)
JOHN VISSING (DEPARTMENT OF NEUROLOGY, RIGSHOSPITALET, UNIVERSITY OF COPENHAGEN, COPENHAGEN, DENMARK – NEUROLOGY)
TUAN VU (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF SOUTH FLORIDA MORSANI COLLEGE OF MEDICINE, TAMPA, FL, USA – NEUROLOGY)
MARION BOEHNLEIN (UCB PHARMA, MONHEIM, GERMANY – NEUROLOGY)
BERNHARD GREVE (UCB PHARMA, MONHEIM, GERMANY – NEUROLOGY)
FIONA GRIMSON (UCB PHARMA, SLOUGH, UK – NEUROLOGY)
ASHA HAREENDRAN (UCB PHARMA, SLOUGH, UK – NEUROLOGY)
VERA BRIL (UNIVERSITY HEALTH NETWORK, TORONTO, ON, CANADA – NEUROLOGY)

Presentatore

FIAMMETTA VANOLI (DEPARTMENT OF NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES, FONDAZIONE IRCCS, INSTITUTO NAZIONALE NEUROLOGICO CARLO BESTA, MILAN, ITALY; DEPARTMENT OF HUMAN NEUROSCIENCES, SAPIENZA UNIVERSITY OF ROME, ROME, ITALY)

Modalità

Poster Session

Abstract

“Background
In MycarinG (NCT03971422), one 6-week cycle of rozanolixizumab significantly improved Myasthenia Gravis Activities of Daily Living (MG-ADL) scores: 70.6% and 31.3% of patients receiving rozanolixizumab and placebo, respectively, achieved MG-ADL response at Day 43. In the novel MG Symptoms Patient Reported Outcome (MGSPRO) scales, rozanolixizumab treatment significantly improved muscle weakness fatigability (MWF), physical fatigue (PF) and bulbar muscle weakness (BMW) scores at Day 43, based on thresholds determined using anchor- and distribution-based methods as defined in a post hoc analysis of Day 29 data. Here, we illustrate the clinical meaningfulness of change in score in MGSPRO scales.

Design/Methods
Patients received rozanolixizumab 7mg/kg or 10mg/kg, or placebo. Change from baseline to Day 43 in MGSPRO scales was assessed and thresholds for clinically meaningful response applied: –16.67 points for MWF and –20.00 points for PF and BMW.

Results
MWF: meaningful improvement at Day 43 was achieved by 46.9% (30/64), 56.5% (35/62) and 28.1% (18/64) of patients receiving rozanolixizumab 7mg/kg, 10mg/kg, and placebo, respectively (–16.67 threshold). PF: meaningful improvement was achieved by 31.3% (20/64), 48.4% (30/62) and 26.6% (17/64) of patients receiving rozanolixizumab 7mg/kg, 10mg/kg, and placebo, respectively (‒20.00 threshold). BMW: meaningful improvement was achieved by 26.6% (17/64), 32.3% (20/62) and 10.9% (7/64) of patients receiving rozanolixizumab 7mg/kg, 10mg/kg, and placebo, respectively (‒20.00 threshold).

Conclusions
A higher percentage of patients treated with rozanolixizumab than placebo had meaningful improvements in PF and MWF symptoms, key aspects of patients’ experiences that are not evaluated in the MG-ADL assessment. Funding: UCB Pharma.”