Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience.
Dionísio , Joana Ambrose , Philip Burke, Georgina Farrugia , Maria Garcia-Reitboeck , Pablo Hewamadduma , Channa Hill , Marguerite Howard , Robin Jacob , Saiju Kullmann , Dimitri
Dionísio , Joana
Ambrose , Philip
Burke, Georgina
Farrugia , Maria
Garcia-Reitboeck , Pablo
Hewamadduma , Channa
Hill , Marguerite
Howard , Robin
Jacob , Saiju
Kullmann , Dimitri
Citations
Altmetric:
Affiliation
Other Contributors
Publication date
2025-03-24
Subject
Collections
Research Projects
Organizational Units
Journal Issue
Abstract
Background: We report our experience of patients with generalised myasthenia gravis (gMG) treated with efgartigimod, an neonatal Fc receptor antagonist, under the Early Access to Medicine Scheme (EAMS) in the UK.
Methods: Data from all UK patients treated with efgartigimod under the EAMS July 2022 to July 2023 were collected retrospectively. Efgartigimod was administered as per the ADAPT protocol (consisting of a treatment cycle of four infusions at weekly intervals with further cycles given according to clinical need).
Results: 48 patients with acetylcholine receptor antibody-positive gMG were treated in 12 centres. Most (75%) were female and most had a disease duration of over 10 years. The average MG-Activities of Daily Living (ADL) score at baseline was 11.2. Most (72.9%) patients had undergone thymectomy. 77.0% were taking prednisolone at baseline. All patients had used non-steroidal immunosuppressant treatments, the average number tried was 2.6 (range 1-6). 51% had received rituximab. 54.2% of patients required regular intravenous immunoglobulin/plasma exchange.75% of patients had a mean reduction in the MG-ADL of≥2 points in the first cycle and this remained stable throughout the study. The mean intracycle reduction in the MG-ADL score in the first, second, third and fourth cycles were -4.6 to -3.9, -3.4 and -4.2, respectively. Side effects were generally mild. No rescue treatments were required. At the end of the study, 96% of patients remained on efgartigimod.
Conclusion: Efgartigimod is a safe and effective treatment for patients with refractory, treatment-resistant gMG.
Citation
Moniz Dionísio J, Ambrose P, Burke G, Farrugia ME, Garcia-Reitboeck P, Hewamadduma C, Hill M, Howard RS, Jacob S, Kullmann D, Leite MI, Miller J, Pinto A, Pritchard J, Riswick T, Sathasivam S, Thambirajah N, Viegas S, Norwood F, Spillane J. Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience. J Neurol Neurosurg Psychiatry. 2025 Mar 24;96(4):322-328. doi: 10.1136/jnnp-2024-334086.
Type
Other