TY  - JOUR
Y1  - 2025/01/11/
N2  - 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.
JF  - Journal of Neurology, Neurosurgery and Psychiatry
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher?s terms and conditions.
VL  - 96
ID  - discovery10206623
UR  - https://doi.org/10.1136/jnnp-2024-334086
PB  - BMJ Publishing Group
TI  - Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience
AV  - public
A1  - Dionisio, Joana Moniz
A1  - Ambrose, Philip
A1  - Burke, Georgina
A1  - Farrugia, Maria Elena
A1  - Garcia-Reitboeck, Pablo
A1  - Hewamadduma, Channa
A1  - Hill, Marguerite
A1  - Howard, Robin S
A1  - Jacob, Saiju
A1  - Kullmann, Dimitri
A1  - Leite, Maria Isabel
A1  - Miller, James
A1  - Pinto, Ashwin
A1  - Pritchard, Jane
A1  - Riswick, Thomas
A1  - Sathasivam, Sivakumar
A1  - Thambirajah, Narmathey
A1  - Viegas, Stuart
A1  - Norwood, Fiona
A1  - Spillane, Jennifer
SP  - 322
SN  - 0022-3050
EP  - 328
KW  - Clinical Neurology
KW  -  FC RECEPTOR
KW  -  Life Sciences & Biomedicine
KW  -  MYASTHENIA
KW  -  NEUROMUSCULAR
KW  -  Neurosciences & Neurology
KW  -  Psychiatry
KW  -  Science & Technology
KW  -  Surgery
IS  - 4
ER  -