Carlo Antozzi
Efgartigimod in non-AChR generalized myasthenia gravis
Autori
- CARLO ANTOZZI ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA)
- RITA FRANGIAMORE ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
- ELENA RINALDI ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
- FIAMMETTA VANOLI ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
- SILVIA BONANNO ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
- LORENZO MAGGI ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
- FRANCESCA ANDREETTA ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – BIOLOGIA
- ROBERTO ARNABOLDI ( ARGENX ITALIA – BIOLOGIA
- ALESSANDRO PINNA ( ARGENX ITALIA – BIOLOGIA
- REANTO MANTEGAZZA ( FONDAZIONE IRCCS ISTITUTO NEUROLOGICO “”C. BESTA”” – UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI – NEUROLOGIA
Presentatore
CARLO ANTOZZI (UO NEUROIMMUNOLOGIA E MALATTIE NEUROMUSCOLARI, SSD IMMUNOTERAPIA E AFERESI TERAPEUTICA, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO C. BESTA VIA G. CELORIA 11, 20133. MILANO.)
Modalità
Oral Communication
Abstract
The neonatal Fc receptor (FcRn) inhibitor Efgartigimod (EFG) has been approved for treatment of generalized Myasthenia Gravis (gMG) with anti-AChR antibodies. We investigated the efficacy of EFG in non-AChR gMG along a clinical follow-up of 2 years. We treated 13 patients with gMG without anti-AChR antibodies including 5 MuSK+, 6 triple-negative (confirmed by live CBA) and 2 LRP4+. EFG was administered according to the GENERATIVE protocol (consisting of a Fixed period of 2 treatment cycles of 4 infusions at weekly intervals, and a Flexible period during which EFG was given in case of clinical worsening) starting from November 2021. Clinical Outcomes were evaluated by means of the MG-ADL, QMG and MGC rating scales. The mean follow-up was 14.5±6.7 months. Meaningful improvement was observed with the clinical scores adopted (MG-ADL: -5.5; MGC: -8.5; QMG: -4.84). The mean number of cycles/year was 3.9. The interval between cycles was 8.9±3.8 weeks. 46% of patients required hospitalization during the two years before treatment with EFG, but during EFG none of the patients was hospitalized or required immunomodulation with plasmapheresis or immunoglobulins as rescue therapy. MG-ADL improvement greater than 5 points was recorded in 49% of patients. EFG was well tolerated. EFG proved to be effective in non-AChR gMG and was able to modify significantly the course of the disease. Our observation strengthens the role for FcRn inhibition also for MG patients without anti-AChR antibodies, a subgroup of patients that deserves the same quality of care of AChR+ MG.