Early Real-life Experience on Zilucoplan for Generalized Myasthenia Gravis: ZILU25 Multicenter Observational Study
Vincenzo Di Stefano1, Nicasio Rini1, Paolo Emilio Alboini2, Alvino Bisecco3, Valentina Damato4, Laura Fionda5, Francesco Habetswallner6, Raffaele Iorio7, Michelangelo Maestri Tassoni8, Alessia Pugliese9, Francesco Saccà10, CLAUDIA VINCIGUERRA11, Rita Frangiamore12
1University of Palermo, 2Fondazione IRCCS Casa Sollievo della Sofferenza, 3University of Campania "Luigi Vanvitelli", 4University of Florence, 5Sant'Andrea Hospital, 6Cardarelli Hospital, 7Catholic University, 8University of Pisa, 9University of Messina, 10University of Naples Federico II, 11University of Salerno, 12Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Istituto Neurologico Carlo Besta
Objective:
To Assess the efficacy and safety of Zilucoplan in generalized myasthenia gravis in the real-life setting.
Background:
Generalized Myasthenia gravis (gMG) is a rare chronic autoimmune disease affecting
the postsynaptic membrane of the neuromuscular junction. New treatment options,
such as the C5 inhibitors, are growing with promising results. In this study we
investigated the real-world use of Zilucoplan for AChR-positive gMG in Italy.
Design/Methods:
Zilucoplan was self-administered by daily subcutaneous injections. Efficacy was
assessed by Myasthenia Gravis Foundation of America Post-intervention status
(MGFA-PIS), Myasthenia Gravis Activity of Daily Living (MG-ADL), Myasthenia Gravis
Composite (MGC), Myasthenia Gravis quantitative (QMG) scales and 15-items
Myasthenia Gravis Quality of Life (MG-QOL-15) questionnaire at baseline and after 1,
4, 12, 24 and 48 weeks.
Results:
54 patients (37 females, aged 57.3) received zilucoplan with a mean follow-up of 26.4
weeks. We observed a clinical meaningful and significant reduction of MG-ADL, QMG,
MGC and MG-QOL-15 scores from W4 to W12 and sustained at W24 of follow-up. The
MG-ADL responder rate was 75.6% at W12 and 82.9% at W24. Minimal symptoms
expression (MSE) was obtained in in 55.6% of patients at W48 with a mean reduction
of the daily prednisone dose of 8.3 mg at W24. Previous long term IVIg was associated
to a better response to MG-ADL at W12 at multivariate analysis.
Conclusions:
Zilucoplan was well-tolerated and effective in most patients with AChR-positive gMG. A
clinical meaningful effect was reported since the first week and was sustained after 24
weeks. Further studies are needed to confirm these preliminary real-life data.
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