Moniz Dionísio, J. orcid.org/0000-0003-4434-3593, Ambrose, P., Burke, G. et al. (17 more authors) (2025) Efgartigimod efficacy and safety in refractory myasthenia gravis: UK’s first real-world experience. Journal of Neurology, Neurosurgery & Psychiatry. ISSN 0022-3050
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.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2025 The Authors. Except as otherwise noted, this author-accepted version of a journal article published in Neuromuscular is made available via the University of Sheffield Research Publications and Copyright Policy under the terms of the Creative Commons Attribution 4.0 International License (CC-BY 4.0), which permits unrestricted use, distribution and reproduction in any medium, provided the original work is properly cited. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ |
Keywords: | FC RECEPTOR; MYASTHENIA; NEUROMUSCULAR |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > Department of Neuroscience (Sheffield) |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 21 Jan 2025 08:55 |
Last Modified: | 21 Jan 2025 08:55 |
Status: | Published online |
Publisher: | BMJ |
Refereed: | Yes |
Identification Number: | 10.1136/jnnp-2024-334086 |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:221985 |