Dionisio, Joana Moniz;
Ambrose, Philip;
Burke, Georgina;
Farrugia, Maria Elena;
Garcia-Reitboeck, Pablo;
Hewamadduma, Channa;
Hill, Marguerite;
... Spillane, Jennifer; + view all
(2025)
Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience.
Journal of Neurology, Neurosurgery and Psychiatry
, 96
(4)
pp. 322-328.
10.1136/jnnp-2024-334086.
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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.
| Type: | Article |
|---|---|
| Title: | Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1136/jnnp-2024-334086 |
| Publisher version: | https://doi.org/10.1136/jnnp-2024-334086 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Clinical Neurology, FC RECEPTOR, Life Sciences & Biomedicine, MYASTHENIA, NEUROMUSCULAR, Neurosciences & Neurology, Psychiatry, Science & Technology, Surgery |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Clinical and Experimental Epilepsy |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10206623 |
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