Real-World Case Series of Ravulizumab Use in Patients with Myasthenia Gravis in Romania DOI Creative Commons

Crisanda Vîlciu,

Oana Antonia Mihalache,

Bogdan Marius Istrate

et al.

Brain Sciences, Journal Year: 2025, Volume and Issue: 15(4), P. 350 - 350

Published: March 28, 2025

Background and Objectives: Ravulizumab, a long-acting C5 complement inhibitor, was approved in the US Europe 2022 as an add-on therapy for standard treatment of AChR-positive generalized MG (gMG). We share our real-world experience with adult patients receiving this Romania. Materials Methods: Six gMG received ravulizumab through Early Access Program (January 2023-October 2024). Patient outcomes were assessed at start q8w using Quantitative (QMG), Activities Daily Living (MG-ADL), Quality Life 15-item revised (MG-QoL15r) scales. Results: Age disease onset ranged from 15 to 35 years. Four six women. Two had severity level IIa, four IIb according Myasthenia Gravis Foundation America (MGFA) classification. Five experienced rapid sustained improvements symptoms MG-ADL score reductions -3 -5 26 weeks post-ravulizumab (except those low baseline score: three one). QMG dropped (-2 12) during period, increased two (+2 +8), remained stable one (zero). Three showed improvement after ≥60 weeks. MG-QoL15r significantly (-22 -10) throughout period. One patient ravulizumab-associated adverse events (vomiting, diarrhea, chills) that resolved within 24 h following symptomatic management, episodes myasthenic exacerbations treatment, discontinued it. Conclusions: All cases presented here early-onset AChR antibody-positive, non-thymomatous MG. Despite differences duration underlying conditions, clinically meaningful symptoms, reduced corticosteroid doses observed all except adding plan.

Language: Английский

Real-World Case Series of Ravulizumab Use in Patients with Myasthenia Gravis in Romania DOI Creative Commons

Crisanda Vîlciu,

Oana Antonia Mihalache,

Bogdan Marius Istrate

et al.

Brain Sciences, Journal Year: 2025, Volume and Issue: 15(4), P. 350 - 350

Published: March 28, 2025

Background and Objectives: Ravulizumab, a long-acting C5 complement inhibitor, was approved in the US Europe 2022 as an add-on therapy for standard treatment of AChR-positive generalized MG (gMG). We share our real-world experience with adult patients receiving this Romania. Materials Methods: Six gMG received ravulizumab through Early Access Program (January 2023-October 2024). Patient outcomes were assessed at start q8w using Quantitative (QMG), Activities Daily Living (MG-ADL), Quality Life 15-item revised (MG-QoL15r) scales. Results: Age disease onset ranged from 15 to 35 years. Four six women. Two had severity level IIa, four IIb according Myasthenia Gravis Foundation America (MGFA) classification. Five experienced rapid sustained improvements symptoms MG-ADL score reductions -3 -5 26 weeks post-ravulizumab (except those low baseline score: three one). QMG dropped (-2 12) during period, increased two (+2 +8), remained stable one (zero). Three showed improvement after ≥60 weeks. MG-QoL15r significantly (-22 -10) throughout period. One patient ravulizumab-associated adverse events (vomiting, diarrhea, chills) that resolved within 24 h following symptomatic management, episodes myasthenic exacerbations treatment, discontinued it. Conclusions: All cases presented here early-onset AChR antibody-positive, non-thymomatous MG. Despite differences duration underlying conditions, clinically meaningful symptoms, reduced corticosteroid doses observed all except adding plan.

Language: Английский

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