Table 1.

Efficacy of intravenous eculizumab in patients with anti-acetylcholine receptor antibody-positive refractory generalized myasthenia gravis in the randomized, double-blind, multicentre, phase 3 REGAIN study [27]

Analyses	Treatment	LSM ranka or LSM change from BLb in total scores at week 26c
		MG-ADL	QMG	MGC	MG-QOL15
Worst-rank ANCOVA
Prespecified	ECU	56.6d	54.7*	57.3	55.5*
	PL	68.3d	70.7	67.7	69.7
		BGD − 11.7	BGD − 16.0	BGD − 10.5	BGD − 14.3
Post hoc sensitivity	ECU	54.8*	53.9**	56.1*	54.6*
	PL	70.2	71.6	69.0	70.6
		BGD − 15.4	BGD − 17.7	BGD − 12.9	BGD − 16.0
Repeated-measures model
Prespecified sensitivity analysis with IST as covariate	ECU	− 4.1**	− 4.6***	− 7.9*	− 13.8***
	PL	− 2.3	− 1.7	− 4.6	− 6.7
		BGD − 1.8	BGD – 2.9	BGD − 3.3	BGD − 7.1
Prespecified sensitivity analysis without IST as covariate	ECU	− 4.2**	− 4.6***	− 8.1*	− 12.6***
	PL	− 2.3	− 1.6	− 4.8	− 5.4
		BGD − 1.9	BGD − 3.0	BGD − 3.3	BGD − 7.2

Results are for the full analysis set comprising 62 patients receiving ECU and 63 patients receiving PL. With the exception of prespecified worst-rank ANCOVA for MG-ADL, p values reported are nominal

BL baseline, BGD between-group difference, ECU eculizumab (900 mg on day 1 and weeks 1, 2, and 3, 1200 mg at week 4, and 1200 mg every second week thereafter), IST immunosuppressive therapy, LSM least-squares mean, MG-ADL myasthenia gravis-activities of daily living, MGC myasthenia gravis composite, MG-QOL15 15-item myasthenia gravis quality of life, PL placebo, QMG quantitative myasthenia gravis

*p < 0.05, ** p < 0.01, *** p ≤ 0.001 vs. PL

^aChange from BL to week 26 in total scores given as LSM rank using a worst-rank ANCOVA

^bAt BL, the mean MG-ADL scores in ECU and PL recipients were 10.5 and 9.9; the mean QMG scores were 17.3 and 16.6; the mean MGC scores were 20.4 and 18.9; and the mean MG-QOL15 scores were 33.6 and 30.7, respectively

^cLower rank or a decrease in total score indicates improvement

^dPrimary endpoint