To report the efficacy of Eculizumab in complicated myasthenic patients in a tertiary hospital setting.

Eculizumab is the first disease modifying FDA approved drug for generalized myasthenia gravis(gMG) that improves neuromuscular transmission by preventing complement-mediated destruction of the acetylcholine receptors(AChR).The REGAIN-study showed improvement in MG with Eculizumab but real world experience outside of research studies is sparse. We present our experience in 15 patients with gMG who had sub-optimal response to first-line treatments.

15 patients(M:F=11:4;Age 21-82 years) with seropositive-gMG were treated with Eculizumab.  MG-Activities of Daily Living(MG-ADL), MG-Quality of Life scale(MG-QoL-R) and MG-composite(MGC)-scores were recorded pre-and post-treatment at regular intervals. MG-Foundation of America(MGFA) Class changes were also assessed.

Disease severity prior to treatment with Eculizumab ranged from MGFA-class II-V, with continuous decline, despite multiple immunotherapies. 15/15 patients required prednisone and chronic IVIg, prior to Eculizumab; 14/15 patients received various long term immunosuppressants. Time to maximum response was 1-8 weeks(median=4-weeks) in 13/15 patients. One patient took 8-months to improve and one patient didn’t improve after 8 weeks of Eculizumab but remained on the drug without worsening. Median changes in MG-ADL, MGC and MGQoL-R scores were 5 (range 0-12), 5 (range 2-21) and 8 (range 2-28) respectively. MGC score improved to 0 from 21, 13 and 8 respectively in three patients. Eculizumab was successfully discontinued in one patients after 2 months of treatment with sustained improvement for 4-months off-treatment. MGFA Class severity improved in all patients by at least 1 point(range 1-4). Prior immunotherapies were tapered and/or discontinued in all patients except one in whom continued IVIg was necessary. All patients tolerated the infusions well without adverse effects; 1 patient reported focal alopecia.

Our findings provide evidence of real world efficacy of Eculizumab in AChR-positive-gMG patients who failed first line treatments. We conclude that Eculizumab is effective in most seropositive gMG patient.