A Real-life experience with Eculizumab and Efgartigimod in generalized Myasthenia Gravis patients

Elenco Abstract

Laura Fionda

A Real-life experience with Eculizumab and Efgartigimod in generalized Myasthenia Gravis patients

Autori

LAURA FIONDA (NEUROMUSCULAR AND RARE DISEASE CENTER, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
CHIARA PANE (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)
VINCENZO DI STEFANO (BIOMEDICINE, NEUROSCIENCE AND ADVANCED DIAGNOSTIC (BIND) DEPARTMENT, UNIVERSITY OF PALERMO, PALERMO, ITALY – NEUROLOGY)
NUNZIA CUOMO (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)
ALESSIO SARNATARO (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)
CLAUDIA VINCIGUERRA (NEUROLOGY UNIT, MEDICINE, SURGERY AND DENTISTRY DEPARTMENT, UNIVERSITY OF SALERNO, SALERNO, ITALY – NEUROLOGY)
LILIANA BEVILACQUA (NEUROLOGY UNIT, MEDICINE, SURGERY AND DENTISTRY DEPARTMENT, UNIVERSITY OF SALERNO, SALERNO, ITALY – NEUROLOGY)
FILIPPO BRIGHINA (BIOMEDICINE, NEUROSCIENCE AND ADVANCED DIAGNOSTIC (BIND) DEPARTMENT, UNIVERSITY OF PALERMO, PALERMO, ITALY – NEUROLOGY)
NICASIO RINI (BIOMEDICINE, NEUROSCIENCE AND ADVANCED DIAGNOSTIC (BIND) DEPARTMENT, UNIVERSITY OF PALERMO, PALERMO, ITALY – NEUROLOGY)
GIORGIA PUORRO (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)
ANGELA MARSILI (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)
MATTEO GARIBALDI (NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS) DEPARTMENT, FACULTY OF MEDICINE AND PSYCHOLOGY, SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
FRANCESCO SACCA’ (NEUROSCIENCE, REPRODUCTIVE AND ODONTOSTOMATOLOGICAL SCIENCES (NSRO) DEPARTMENT, FEDERICO II UNIVERSITY, NAPLES, ITALY – NEUROLOGY)

Presentatore

LAURA FIONDA (NEUROMUSCULAR AND RARE DISEASE CENTER, SANT’ANDREA HOSPITAL, ROME, ITALY)

Modalità

Oral Communication

Abstract

“Introduction
Eculizumab (ECU) and Efgartigimod (EFGA) are approved for the treatment of generalized Myasthenia Gravis (gMG). Objective of our study is to describe their clinical response in a real-life setting.

Methods
We included patients receiving either ECU or EFGA as part of our clinic practice and retrospectively collected data on the MG activities of daily living (MG-ADL), quantitative MG scale (QMG), MG deterioration/crises, adverse events, and prednisone use.

Results
We enrolled 63 patients, 32 treated with ECU and 31 with EFGA. Median follow-up was 253 days. Both treatments were well tolerated. Patients treated with EFGA were more likely to suspend treatment (HR 3.732; p=0.043). Main reasons were MG deterioration, patient choice, adverse event.
ECU decreased the MG deterioration/crises rate more than EFGA (OR 0.545, p=0.043).
Treatment did not impact on the MG-ADL reduction (F=1.3; p=0.237). Mean prednisone reduction at follow-up was -16.7 mg for ECU and -5.2 mg for EFGA (F=11.7; p=0.001). Eculizumab was superior to Efgartigimod in reducing the QMG score (F=4.9; p=0.003). By week 24, the QMG decreased by -7.8 for ECU and -2.8 for EFGA. Speed of CS reduction was superior in ECU treated patients compared to EFGA (-13.1 mg/month vs -3.2 mg/month; p=0.001).

Conclusion
Eculizumab and Efgartigimod proved to be both effective treatments in a real world setting. Both treatments reduced MG-ADL and QMG in difficult to treat gMG patients. Patients treated with ECU were less likely to suspend treatment or show a deterioration/crisis. Prednisone sparing effect and reduction speed was higher with Eculizumab.”