RITA FRANGIAMORE
Anti-FcRn treatment for generalized Myasthenia Gravis: a real word experience with Efgartigimod in AChR-Ab positive patients.
Autori
- RITA FRANGIAMORE (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – MEDICO NEUROLOGO)
- ELENA RINALDI (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – BIOLOGO)
- FIAMMETTA VANOLI (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – MEDICO NEUROLOGO)
- SILVIA BONANNO (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – MEDICO NEUROLOGO)
- FRANCESCA ANDREETTA (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – BIOLOGO)
- LORENZO MAGGI (1UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY; – MEDICO NEUROLOGO)
- ALESSANDRO PINNA (ARGENX ITALY, S.R.L., MILAN, ITALY – MEDICAL DIRECTOR ARGENX ITALY)
- ROBERTO ARNABOLDI (ARGENX ITALY, S.R.L., MILAN, ITALY – PHD MEDICAL SCIENCE LIAISON ARGENX ITALY)
- CARLO ANTOZZI (UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY – MEDICO NEUROLOGO)
- RENATO MANTEGAZZA (UOC NEUROLOGY 4 – NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES UNIT, FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY – MEDICO NEUROLOGO)
Presentatore
RITA FRANGIAMORE
Modalità
Oral Communication
Abstract
We report our real life experience in 17 patients with AChR-positive generalized MG (gMG) treated with Efgartigimod (EFG) according to the Early Access Program (EAP, version 1.0 Sep. 2021) starting from November 2021 up to December 2023. EFG was added to ongoing therapy with immunosuppressive drugs and/or corticosteroids (mean steroid dose at baseline: 27.5 ± 16.8 mg). Clinical outcomes were measured by the MG-ADL, QMG, MGC and MGQoL-15 rating scales. Meaningful improvement was recorded after each treatment cycle with EFG (of four infusions each) by all rating scales. During the clinical follow-up (mean duration 13.9 months), 88% of patients needed 3 treatment cycles, 71% 4 cycles and 56% 5 or more cycles. A subgroup of 5 patients (29.4%) needed retreatment every 4 weeks. 9 patients (53%) reduced the corticosteroid dose. During treatment with EFG none of the patients were hospitalized or needed treatment with Plex/IVIG compared with the two years before EFG during which 41% needed hospitalization, and admission to the intensive care for one patient. Before EFG, 11 patients (65%) needed one or more cycles of Plex or IVIG. No major side effects or infusion-related reactions occurred. EFG was able to improve considerably the course of the disease since negative events (such as hospitalization or rescue therapies) did not occur along the clinical follow-up. The duration of the clinical response to EFG therapy does not seem to have the same trend in all patients. Despite the proven effectiveness further studies are needed to optimize the treatment protocol.