Giorgia Camera
Rapid regression of bulbar symptoms in myasthenia gravis refractory clinical relapse after eculizumab infusion as rescue therapy
Autori
- GIORGIA CAMERA (ASST PAPA GIOVANNI XXIII BERGAMO – MD, NEUROLOGO)
- PAOLO PAONE (ASST PAPA GIOVANNI XXIII BERGAMO – STATISTICO )
- EMANUELA AGAZZI (ASST PAPA GIOVANNI XXIII BERGAMO – MD, NEUROLOGO)
- PAOLA BAZZI ( ASST BRIANZA, OSPEDALE VIMERCATE – MD, NEUROLOGO)
- DARIO ALIMONTI (ASST PAPA GIOVANNI XXIII BERGAMO – MD, NEUROLOGO)
- MANLIO SGARZI (ASST PAPA GIOVANNI XXIII BERGAMO – MD, NEUROLOGO)
Presentatore
GIORGIA CAMERA (ASST PAPA GIOVANNI XXIII BERGAMO)
Modalità
Poster Session
Abstract
“Introduction: A 32-year-old woman was diagnosed with myasthenia gravis positive for anti-acetylcholine receptor antibodies and thymoma removed in 2018. Upon admission to our institution, the therapy was: prednisone 50 mg per day, azathioprine 100 mg per day and pyridostigmine 30 mg four times a day. She had incomplete control of her symptoms, for this reason she was enrolled in a clinical trial with a new subcutaneous complement inhibitor.
Observation: Three months after randomization and still in the double-blind period in which symptoms appeared to be under control, she had a severe bulbar relapse with rapid worsening. After unblinding for safety (Placebo) aggressive treatment was adopted during hospitalization, with plasmapheresis, intravenous immunoglobulin and high-dose corticosteroids, but her condition continued to worsen, culminating in MGFA score IVb. ADL score was 15 with facial diplegia, marked dysarthria with speech difficulty, jaw ptosis, head drop, severe limb weakness, and a gastric tube was placed. Eculizumab was administered as rescue therapy. After the first administration the ADLs dropped from 15 to 4 allowing the removal of the gastric tube. Over the next week the patient’s symptoms continued to improve, reaching a state of excellent health.
Conclusion: The role of the complement system in MG has been demonstrated, and it has been determined that complement inhibition in patients with MG can prevent disease induction and reverse its progression [1]. This case confirms the effectiveness of Eculizumab in patients refractory [2] to classic immunotherapies, also showing a high speed of action with rapid and lasting improvement of symptoms.”