| Title: |
Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France |
| Authors: |
Tammam, Giulia; Slioui, Abderhmane; Friedman, Diane; Acket, Blandine; Cavalli, Michele; Bresch, Saskia; Lagrange, Emmeline; Sole, Guilhem; Noury, Jean-Baptiste; Barbat Du Closel, Luce; Marois, Clémence; Pakleza, Aleksandra, Nadaj; Tard, Céline; Salort-Campana, Emmanuelle; Merle, Philippe-Edouard; Annane, Djillali; Attarian, Shahram; Duval, Fanny; Frachet, Simon; Svahn, Juliette; Pegat, Antoine; Bouhour, Françoise; Taieb, Guillaume; Praline, Julien; Puma, Angela; Fabry, Vincent; Villa, Luisa; Beltran, Stéphane; Behin, Anthony; Cintas, Pascal; Sacconi, Sabrina |
| Contributors: |
Hôpital Pasteur Nice (CHU); Hôpital Raymond Poincaré AP-HP; Université de Versailles Saint-Quentin-en-Yvelines (UVSQ); Université Paris-Saclay; Département Neurologie CHU Toulouse; Pôle Neurosciences CHU Toulouse; Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse); CHU de Grenoble-Alpes - Centre Hospitalier Universitaire CHU Grenoble (CHUGA); Filière Neuromusculaire (FILNEMUS); CHU de Bordeaux Pellegrin Bordeaux; ERN EURO-NMD – European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD); Centre Hospitalier Régional Universitaire de Brest (CHRU Brest); Lymphocytes B, Autoimmunité et Immunothérapies (LBAI); Université de Brest (UBO EPE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-LabEX IGO Immunothérapie Grand Ouest; Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Institut Brestois Santé Agro Matière (IBSAM); Université de Brest (UBO EPE); Hôpital de la Timone CHU - APHM (TIMONE); CHU Pitié-Salpêtrière AP-HP; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU); Lille Neurosciences & Cognition - U 1172 (LilNCog); Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Centre Hospitalier Régional Universitaire CHU Lille (CHRU Lille); Département de neurologie Lille; Centre Hospitalier Régional Universitaire CHU Lille (CHRU Lille); Neurologie CHU Amiens; CHU Amiens-Picardie; Neuropathies et Innovations Thérapeutiques (NEURIT); OmégaHealth (ΩHealth); Université de Limoges (UNILIM)-Université de Limoges (UNILIM); Service de Neurologie CHU Limoges; CHU Limoges; Hôpital neurologique et neurochirurgical Pierre Wertheimer CHU - HCL; Hospices Civils de Lyon (HCL); Département de Neurologie Hôpital Gui de Chauliac - CHU Montpellier; Hôpital Gui de Chauliac CHU Montpellier; Centre Hospitalier Régional Universitaire Montpellier (CHRU Montpellier)-Centre Hospitalier Régional Universitaire Montpellier (CHRU Montpellier); Institut de Génomique Fonctionnelle (IGF); Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université de Montpellier (UM); Centre Hospitalier Universitaire d'Orléans (CHU Orléans); Centre Hospitalier Régional Universitaire de Tours (CHRU Tours); Institut de Recherche sur le Cancer et le Vieillissement (IRCAN); Université Nice Sophia Antipolis (1965 - 2019) (UNS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Côte d'Azur (UniCA); Nice University Hospital |
| Source: |
ISSN: 2214-3599. |
| Publisher Information: |
CCSD; IOS Press |
| Publication Year: |
2026 |
| Collection: |
Université de Limoges: HAL |
| Subject Terms: |
myasthenia gravis; quality of life; real-world evidence; safety; zilucoplan; effectiveness; corticosteroids; [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology; [SCCO.NEUR]Cognitive science/Neuroscience |
| Description: |
International audience ; ObjectivesTo evaluate the effectiveness and safety of zilucoplan in the real-world treatment setting in France.MethodsThis retrospective cohort study evaluated patients with generalized anti-AChR myasthenia gravis (MG) failing current therapy enrolled in the French early access program (EAP) for zilucoplan. Patients were evaluated at enrollment and at Months 1, 3 and 6 with the MG-ADL score, the Myasthenic Muscle Score (MMS-Garches), and the MG-quality of life questionnaire (MG-QoL15r). MG crises and on-treatment adverse events were documented. MG medication use was compared between the six months before and after enrollment.ResultsForty-eight patients were enrolled and treated with zilucoplan (mean age: 56.2 years; 50.9% men). Thirty patients achieved six months follow-up before the EAP ended. Three patients discontinued zilucoplan due to lack of efficacy. Mean MG-ADL score decreased from 7.4 ± 4.4 to M0 to 2.5 ± 2.7 at M6 (p < 0.0001). 9/43 patients achieved minimal symptom expression at M3 and 12/30 at M6. Mean MMS-Garches score was 67.5 ± 20.0 at M0 and 87.5 ± 13.2 at M6 (p < 0.0001). Mean MG-QoL15r score was 18.2 ± 8.6 at M0 and 8.7 ± 8.3 at M6 (p = 0.0156). 15 patients experienced adverse events, leading to discontinuation in four cases. The mean oral corticosteroid dose decreased from 28.8 ± 11.8 mg to 17.9 ± 11.8 (p = 0.001). Six patients experienced MG crises on treatment, compared to 22 in the six months preceding enrollment. treatment.DiscussionFollowing initiation of zilucoplan treatment, we observed an improvement in MG symptoms and a reduction in MG crises and in exposure to oral corticosteroids. Overall, zilucoplan was well-tolerated with no major adverse events reported.Trial registration informationThe study was registered in the clinicaltrials.gov trial registry under the number NCT06815133 (https://clinicaltrials.gov/study/NCT06815133) on February 4th, 2025. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/41904994; PUBMED: 41904994 |
| DOI: |
10.1177/22143602261437792 |
| Availability: |
https://hal.science/hal-05583855; https://hal.science/hal-05583855v1/document; https://hal.science/hal-05583855v1/file/Tammam%20et%20al%202026.pdf; https://doi.org/10.1177/22143602261437792 |
| Rights: |
https://creativecommons.org/licenses/by-nc/4.0/ ; info:eu-repo/semantics/OpenAccess |
| Accession Number: |
edsbas.70E0A95B |
| Database: |
BASE |