TABLE 1.
Baseline Patient Demographics and Disease Characteristics
| Variable | PREVENT a | PREVENT OLE b | PREVENT and OLE combined c | |||
|---|---|---|---|---|---|---|
| Placebo (N = 47) | Eculizumab (N = 96) | Placebo/eculizumab d (N = 41) | Eculizumab/eculizumab (N = 78) | Eculizumab (N = 119) | Eculizumab (N = 137) | |
| Age at first dose, mean (SD), yr | 45.0 (13.3) | 43.9 (13.3) | 46.0 (13.8) | 46.6 (13.8) | 46.4 (13.7) | 44.5 (13.5) |
| Sex, n (%) | ||||||
| M | 5 (10.6) | 8 (8.3) | 5 (12.2) | 4 (5.1) | 9 (7.6) | 13 (9.5) |
| F | 42 (89.4) | 88 (91.7) | 36 (87.8) | 74 (94.9) | 110 (92.4) | 124 (90.5) |
| Region, n (%) | ||||||
| Americas | 15 (31.9) | 29 (30.2) | 13 (31.7) | 21 (26.9) | 34 (28.6) | 42 (30.7) |
| Europe | 19 (40.4) | 32 (33.3) | 17 (41.5) | 27 (34.6) | 44 (37.0) | 49 (35.8) |
| Asia‐Pacific | 13 (27.7) | 35 (36.6) | 11 (26.8) | 30 (38.5) | 41 (34.5) | 46 (33.6) |
| Race, n (%) | ||||||
| Asian | 15 (31.9) | 37 (38.5) | 13 (31.7) | 32 (41.0) | 45 (37.8) | 50 (36.5) |
| Black or African American | 8 (17.0) | 9 (9.4) | 7 (17.1) | 3 (3.8) | 10 (8.4) | 16 (11.7) |
| White | 24 (51.1) | 46 (47.9) | 21 (51.2) | 40 (51.3) | 61 (51.3) | 67 (48.9) |
| Other e | 0 (0.0) | 4 (4.2) | 0 (0.0) | 1 (1.3) | 3 (2.5) | 4 (2.9) |
| Historical ARR (within 24 mo before screening for PREVENT), mean (SD) | 2.1 (1.0) | 1.9 (0.9) | 2.2 (1.1) | 1.9 (1.0) | 2.0 (1.0) | 2.0 (1.0) |
| EDSS score | ||||||
| Median (range) | 4.0 (1.0–6.5) | 4.0 (1.0–7.0) | 4.5 (0.0–7.5) | 4.0 (0.0–7.5) | 4.0 (0.0–7.5) | 4.0 (0.0–7.5) |
| Mean (SD) | 4.3 (1.5) | 4.2 (1.6) | 4.3 (1.9) | 4.0 (1.7) | 4.1 (1.8) | 4.2 (1.7) |
| HAI score | ||||||
| Median (range) | 2.0 (0.0–6.0) | 2.0 (0.0–8.0) | 2.0 (0.0–9.0) | 1.0 (0.0–8.0) | 2.0 (0.0–9.0) | 2.0 (0.0–9.0) |
| Mean (SD) | 2.1 (1.4) | 2.4 (2.2) | 2.8 (2.1) | 2.0 (2.2) | 2.3 (2.2) | 2.5 (2.2) |
| mRS score | ||||||
| Median (range) | 2.0 (0.0–4.0) | 2.0 (0.0–4.0) | 2.0 (0.0–6.0) | 2.0 (0.0–4.0) | 2.0 (0.0–6.0) | 2.0 (0.0–6.0) |
| Mean (SD) | 2.1 (1.0) | 2.1 (1.1) | 2.4 (1.4) | 1.9 (1.3) | 2.1 (1.3) | 2.2 (1.2) |
| EQ‐5D‐3L VAS score, median (range) | 60.0 (0.0–9.5) | 70.0 (10.0–100.0) | 65.0 (5.0–100.0) | 79.0 (10.0–100.0) | 70.0 (5.0–100.0) | 68.0 (5.0–100.0) |
| EQ‐5D‐3L index score, median (range) | 0.7 (0.3–1.0) | 0.8 (0.1–1.0) | 0.6 (0.0–1.0) | 0.8 (0.0–1.0) | 0.8 (0.0–1.0) | 0.7 (0.0–1.0) |
Age at first dose and baseline scores at first study drug dose in PREVENT.
Age at first dose and baseline EDSS, HAI, and mRS scores at first study drug dose in the OLE; EQ‐5D‐3L VAS and index scores at last available assessment prior to first study drug dose in the OLE.
Age at first dose and baseline scores at first eculizumab dose in either study.
Patients receiving placebo during PREVENT transitioned to eculizumab in the OLE.
Other includes American Indian, Alaskan native, unknown, and other.
ARR = annualized relapse rate; EDSS = Expanded Disability Status Scale; EQ‐5D‐3L = 3‐level 5‐dimension EuroQol questionnaire; HAI = Hauser Ambulation Index; mRS = modified Rankin Scale; OLE = open‐label extension; SD = standard deviation; VAS = visual analog scale.