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

Background

We present our experience of treating patients with generalised Myasthenia Gravis (gMG) with Efgartigimod under the UK Early Access to Medicine Scheme (EAMs).

Methods

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.

Results

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.

Conclusion

Efartigimod is an effective and well-tolerated treatment for patients with refractory gMG.