{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T18:54:28Z","timestamp":1793386468268,"version":"4.5.0"},"reference-count":30,"publisher":"MDPI AG","issue":"4","license":[{"start":{"date-parts":[[2025,3,28]],"date-time":"2025-03-28T00:00:00Z","timestamp":1743120000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"AstraZeneca"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Brain Sciences"],"abstract":"<jats:p>Background and Objectives: Ravulizumab, a long-acting C5 complement inhibitor, was approved in the US and Europe in 2022 as an add-on therapy for the standard treatment of AChR-positive generalized MG (gMG). We share our real-world experience with adult patients receiving this therapy in Romania. Materials and Methods: Six AChR-positive gMG patients received ravulizumab through an Early Access Program (January 2023\u2013October 2024). Patient outcomes were assessed at the therapy start and q8w using Quantitative MG (QMG), MG Activities of Daily Living (MG-ADL), and MG Quality of Life 15-item revised (MG-QoL15r) scales. Results: Age at disease onset ranged from 15 to 35 years. Four of the six patients were women. Two patients had gMG severity level of IIa, and four patients of IIb according to the Myasthenia Gravis Foundation of America (MGFA) classification. Five patients experienced rapid and sustained improvements in MG symptoms with MG-ADL score reductions ranged from \u22123 to \u22125 at 26 weeks post-ravulizumab start (except for those with a low baseline score: three and one). QMG score dropped in three patients (\u22122 to 12) during the treatment period, increased in two (+2 and +8), and remained stable in one (zero). Three patients showed sustained improvement in MG symptoms after \u226560 weeks. MG-QoL15r significantly dropped (\u221222 to \u221210) throughout the treatment period. One patient experienced ravulizumab-associated adverse events (vomiting, diarrhea, chills) that resolved within 24 h following symptomatic management, two to three episodes of myasthenic exacerbations during treatment, and discontinued it. Conclusions: All cases presented here had early-onset AChR antibody-positive, non-thymomatous MG. Despite differences in disease duration and underlying conditions, clinically meaningful and sustained improvements in gMG symptoms, and reduced corticosteroid doses were observed in all patients except one after adding ravulizumab to the treatment plan.<\/jats:p>","DOI":"10.3390\/brainsci15040350","type":"journal-article","created":{"date-parts":[[2025,3,28]],"date-time":"2025-03-28T04:36:48Z","timestamp":1743136608000},"page":"350","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Real-World Case Series of Ravulizumab Use in Patients with Myasthenia Gravis in Romania"],"prefix":"10.3390","volume":"15","author":[{"given":"Crisanda","family":"V\u00eelciu","sequence":"first","affiliation":[{"name":"Department of Neurology, \u201cCarol Davila\u201d University of Medicine and Pharmacy, 020021 Bucharest, Romania"},{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oana Antonia","family":"Mihalache","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bogdan Marius","family":"Istrate","sequence":"additional","affiliation":[{"name":"Independent Researcher, 3000 Leuven, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mihaela Aftinia","family":"Marian","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cCarol Davila\u201d University of Medicine and Pharmacy, 020021 Bucharest, Romania"},{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6738-9186","authenticated-orcid":false,"given":"Mirela Ramona","family":"Dr\u0103ghici","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana Mihaela","family":"Petrescu","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cCarol Davila\u201d University of Medicine and Pharmacy, 020021 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5056-0191","authenticated-orcid":false,"given":"Adriana Octaviana","family":"Dul\u0103mea","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cCarol Davila\u201d University of Medicine and Pharmacy, 020021 Bucharest, Romania"},{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniela Cristina","family":"Anghel","sequence":"additional","affiliation":[{"name":"Department of Neurology, \u201cCarol Davila\u201d University of Medicine and Pharmacy, 020021 Bucharest, Romania"},{"name":"Department of Neurology, \u201cFundeni\u201d Clinical Institute, 022328 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,3,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"324","DOI":"10.1016\/j.nmd.2023.02.002","article-title":"Epidemiology and treatment of myasthenia gravis: A retrospective study using a large insurance claims dataset in Germany","volume":"33","author":"Mevius","year":"2023","journal-title":"Neuromuscul. 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