Real world experience of Efgartigimod for generalised Myasthenia Gravis – A 17 month prospective study

<h3>Background</h3> We present our experience of treating patients with generalised Myasthenia Gravis (gMG) with Efgartigimod under the UK Early Access to Medicine Scheme (EAMs). <h3>Methods</h3> Data regarding all patients receiving Efgartimod in the NHNN were collected prospectively. Changes in MG Activities of Daily Living (MG-ADL) score, reduction in prednisolone dose and need for rescue therapies were used to assess response. <h3>Results</h3> 18 patients with an average disease duration of 14.6 years (1-40) were treated with Efgartigimod over a 17 month period. Regarding previous treatments, all patients received prednisolone and non-steroidal immunosuppressant therapies (NSITs) (average number of NSITs 2.4 (1-7)). 50% (9/18) had received Rituximab. 67% (12/18) required IVIG/PLEX regularly and 27% (5/18) required intermittent IVIg/PLEX. Mean baseline MG ADL was 11.3. Following the first treatment cycle 89% (16/18) were defined as responders with an average MG-ADL reduction of 7 (3-13). 83% (15/18) of patients reduced their steroid doses. Two patients stopped taking Efgartigimod due to lack of efficacy and need for rescue therapy. 89% (16/18) remained on it with a mean inter-cycle interval of 6.5 weeks. Efgartigimod was well tolerated with only minor side effects reported. <h3>Conclusion</h3> Efartigimod is an effective and well-tolerated treatment for patients with refractory gMG.