Demetrio Marando
AChR-Positive Generalized Myasthenia Gravis Patients Unresponsive to new targeted molecular therapies: A single-centre Case Series
Autori
- DEMETRIO MARANDO (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
- STEFANIA MORINO (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
- LUCA LEONARDI (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
- LAURA TUFANO (DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
- ANTONIO LAULETTA (DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
- ELENA ROSSINI (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
- FRANCESCA FORCINA (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
- MATTEO GARIBALDI (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY. DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
- GIOVANNI ANTONINI (DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, ROME, ITALY – NEUROLOGY)
- LAURA FIONDA (NEUROMUSCULAR AND RARE DISEASE CENTRE, NEUROLOGY UNIT, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
Presentatore
DEMETRIO MARANDO
Modalità
Poster Session
Abstract
“Background: In recent years, anti-complement drugs and neonatal fc receptor (FcRn) inhibitors have shown promising results in the treatment of generalized MG (gMG), especially in patients refractory to standard immunotherapies.
However, a small part of these patients, could be unresponsive also to new targeted molecular therapies.
Methods: We conducted a retrospective case series analysis involving gMG patients referred to our neuromuscular centre treated with anti-complement drugs (Eculizumab, Ravulizumab and Zilucoplan) and FcRn inhibitors (Efgartigimod, Rozanolixizumab), who showed unsatisfactory response or had to stop treatments because of adverse events. Data concerning demographic and clinical characteristics, clinical scales, adverse events, were collected.
Results: Of 23 gMG patients treated with new targeted drugs, 7 (30%) (3 males, 4 females, mean age 59.1, range 31-79) discontinued the treatment. Three patients had MGFA class IIIA while 4 patients IIIB. All patients underwent both treatments. 6/7 patient showed no clinical response with anti-complement treatment. One patient, despite MG symptoms improvement, started to complain of progressive somnolence and ideomotor slowing one month after anti-complement therapy start, that lead to withdrawal. FcRn inhibitors were discontinued in 5/7 patients for unsatisfactory clinical response. The other 2 patients discontinued because of adverse events: one patient developed endocarditis, while another patient had continuous diarrhoea.
Conclusion: Despite the safety and efficacy of new available drugs for gMG, a small percentage of patients are still unresponsive or show drug-related adverse effects. Further studies on larger samples are warranted, aimed to detect biomarkers of disease activity and predictors of treatment responsiveness.”