Table 2.
Baseline demographics and characteristics of patients who did or did not achieve ‘minimal symptom expression’ at any time during REGAIN and the open-label extension study
| Variable | MG-ADL total score 0–1 | MG-QOL15 total score 0–3 | ||||
|---|---|---|---|---|---|---|
| Did achieve n = 49 | Did not achieve n = 68 | p valuea | Did achieve n = 37 | Did not achieve n = 80 | p valuea | |
| Sex, n (%) | ||||||
| Male | 14 (28.6) | 24 (35.3) | 0.5491 | 11 (29.7) | 27 (33.8) | 0.8322 |
| Female | 35 (71.4) | 44 (64.7) | 26 (70.3) | 53 (66.3) | ||
| Race, n (%) | ||||||
| Asian | 7 (14.3) | 12 (17.6) | 0.5767 | 5 (13.5) | 14 (17.5) | 0.3377 |
| Black or African American | 1 (2.0) | 1 (1.5) | 1 (2.7) | 1 (1.3) | ||
| White | 39 (79.6) | 49 (72.1) | 28 (75.7) | 60 (75.0) | ||
| Other/multiple/unknown | 2 (4.1) | 6 (8.8) | 3 (8.1) | 5 (6.3) | ||
| Age at first eculizumab dose, years, mean (SD) | 47.4 (18.79) | 47.0 (15.25) | 0.8847 | 44.6 (19.23) | 48.4 (15.45) | 0.2611 |
| Duration of MG at first eculizumab doseb, years, mean (SD) | 8.3 (6.57) | 11.2 (9.08) | 0.0474 | 8.7 (5.97) | 10.5 (9.05) | 0.2091 |
| MG-ADL total score at REGAIN baseline, mean (SD) | 9.6 (3.08) | 10.4 (2.55) | 0.1061 | 9.3 (2.79) | 10.5 (2.75) | 0.0380 |
| MG-QOL15 total score at REGAIN baseline, mean (SD) | 31.0 (13.23) | 32.0 (12.00) | 0.6709 | 28.2 (14.14) | 33.1 (11.40) | 0.0487 |
| QMG total score at REGAIN baseline, mean (SD) | 16.8 (5.51) | 17.1 (5.21) | 0.8247 | 17.1 (5.77) | 16.9 (5.13) | 0.9034 |
| Patients with MGFA class at REGAIN screening, n (%) | ||||||
| IIa | 10 (20.4) | 14 (20.6) | 0.7087 | 10 (27.0) | 14 (17.5) | 0.7954 |
| IIb | 11 (22.4) | 8 (11.8) | 7 (18.9) | 12 (15.0) | ||
| IIIa | 13 (26.5) | 21 (30.9) | 10 (27.0) | 24 (30.0) | ||
| IIIb | 10 (20.4) | 18 (26.5) | 8 (21.6) | 20 (25.0) | ||
| IVa | 2 (4.1) | 4 (5.9) | 1 (2.7) | 5 (6.3) | ||
| IVb | 3 (6.1) | 3 (4.4) | 1 (2.7) | 5 (6.3) | ||
| Patients with history of MG crisis before REGAIN, n (%) | 8 (16.3) | 13 (19.1) | 0.8091 | 6 (16.2) | 15 (18.8) | 0.8018 |
| Patients using ISTs before REGAIN, n (%) | ||||||
| 1 IST | 0 (0.0) | 2 (2.9) | 0.1818 | 0 (0.0) | 2 (2.5) | 0.0520 |
| 2 ISTs | 27 (55.1) | 26 (38.2) | 22 (59.5) | 31 (38.8) | ||
| 3 ISTs | 15 (30.6) | 23 (33.8) | 12 (32.4) | 26 (32.5) | ||
| ≥ 4 ISTs | 7 (14.3) | 17 (25.0) | 3 (8.1) | 21 (26.3) | ||
IST immunosuppressive therapy, MG myasthenia gravis, MG-ADL myasthenia gravis activities of daily living scale, MGFA Myasthenia Gravis Foundation of America, MG-QOL15 15-item myasthenia gravis quality of life questionnaire, QMG quantitative myasthenia gravis scale, SD standard deviation
aThe significance of differences between groups was evaluated by calculating p values based on Fisher’s exact test for categorical variables and a two-sample t-test for continuous variables
bTime from MG diagnosis to date of first eculizumab dose