Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG

Dewilde, S; Philips, G; Paci, S; Beauchamp, J; Chiroli, S; Quinn, C; Day, L; Larkin, M; Palace, J; Berrih-Aknin, S; Claeys, KG; Muppidi, S; Mantegazza, R; Saccà, F; Meisel, A; Bassez, G; Murai, H; Janssen, M

Journal article

Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG

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

Publication status:: Published

Peer review status:: Peer reviewed

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APA Style

Dewilde, S., Philips, G., Paci, S., Beauchamp, J., Chiroli, S., Quinn, C., Day, L., Larkin, M., Palace, J., Berrih-Aknin, S., Claeys, K. G., Muppidi, S., Mantegazza, R., Saccà, F., Meisel, A., Bassez, G., Murai, H., & Janssen, M. (2023). Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG. BMJ Open, 13(1), e066445–e066445.

MLA Style

Dewilde, S, et al. “Patient-Reported Burden of Myasthenia Gravis: Baseline Results of the International Prospective, Observational, Longitudinal Real-World Digital Study MyRealWorld-MG.” BMJ Open, vol. 13, no. 1, 2023, pp. e066445–e066445.

Chicago Style

Dewilde, S, G Philips, S Paci, et al. 2023. “Patient-Reported Burden of Myasthenia Gravis: Baseline Results of the International Prospective, Observational, Longitudinal Real-World Digital Study MyRealWorld-MG.” BMJ Open 13 (1): e066445–e066445.
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