Baseline demographic and disease characteristics
| Demographic . | Group A: emicizumab once weekly, n = 68 . | Group B: emicizumab every 2 wk, n = 10 . | Group C: emicizumab every 4 wk, n = 10 . | Total, N = 88 . |
|---|---|---|---|---|
| Sex, n (%) | ||||
| Male | 68 (100) | 10 (100) | 10 (100) | 88 (100) |
| Age, y | ||||
| Median (range) | 6.0 (1-15) | 8.0 (2-10) | 9.0 (2-11) | 7.0 (1–15) |
| 0 to <2 | 8 (11.8) | 0 | 0 | 8 (9.1) |
| 2 to <6 | 19 (27.9) | 3 (30.0) | 2 (20.0) | 24 (27.3) |
| 6 to <12 | 38 (55.9) | 7 (70.0) | 8 (80.0) | 53 (60.2) |
| ≥12 | 3 (4.4) | 0 | 0 | 3 (3.4) |
| Race, n (%) | ||||
| White | 39 (57.4) | 7 (70.0) | 8 (80.0) | 54 (61.4) |
| Asian | 10 (14.7) | 1 (10.0) | 2 (20.0) | 13 (14.8) |
| African American | 11 (16.2) | 1 (10.0) | 0 | 12 (13.6) |
| Multiple | 2 (2.9) | 0 | 0 | 2 (2.3) |
| Unknown | 6 (8.8) | 1 (10.0) | 0 | 7 (8.0) |
| Ethnicity, n (%) | ||||
| Hispanic or Latino | 5 (7.4) | 1 (10.0) | 1 (10.0) | 7 (8.0) |
| Not Hispanic or Latino | 61 (89.7) | 9 (90.0) | 9 (90.0) | 79 (89.8) |
| Not stated | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Unknown | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Hemophilia severity at baseline, n (%) | ||||
| Mild | 2 (2.9) | 0 | 0 | 2 (2.3) |
| Moderate | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Severe | 65 (95.6) | 10 (100) | 10 (100) | 85 (96.6) |
| Previous regimen, n (%) | ||||
| Episodic | 16 (23.5) | 2 (20.0) | 4 (40.0) | 22 (25.0) |
| Prophylactic | 52 (76.5) | 8 (80.0) | 6 (60.0) | 66 (75.0) |
| Median no. of bleeds in 24 wk prior to study entry (IQR) | 6 (4-9) | 5 (3-10) | 6 (3-8) | 6 (3.5-9) |
| Median time from FVIII inhibitor diagnosis (range), mo | 56.4 (0.5-142.4) | 71.4 (23.3-120.0) | 66.5 (15.0-120.0) | 58.4 (0.5-142.4) |
| Participants with target joints at baseline, n (%) | ||||
| No target joints present | 44 (64.7) | 3 (30.0) | 7 (70.0) | 54 (61.4) |
| Target joints present | 24 (35.3) | 7 (70.0) | 3 (30.0) | 34 (38.6) |
| Highest historical inhibitor titer, BU/mL | ||||
| n | 66 | 10 | 10 | 86 |
| Median (IQR) | 200 (64-614) | 953 (197-1861) | 133 (29-275) | 200 (64-854) |
| Baseline FVIII inhibitor titer | ||||
| ≥5 BU/mL | 50 (73.5) | 8 (80.0) | 5 (50.0) | 63 (71.6) |
| ≥0.6 BU/mL and <5 BU/mL | 13 (19.1) | 1 (10.0) | 3 (30.0) | 17 (19.3) |
| <0.6 BU/mL | 4 (5.9) | 0 (0.0) | 0 (0.0) | 4 (4.5) |
| Unknown | 1 (1.5) | 1 (10.0) | 2 (20.0) | 4 (4.5) |
| Previously treated with ITI, n (%) | ||||
| Yes | 45 (66.2) | 10 (100) | 8 (80.0) | 63 (71.6) |
| No | 23 (33.8) | 0 | 2 (2.0) | 25 (28.4) |
| Demographic . | Group A: emicizumab once weekly, n = 68 . | Group B: emicizumab every 2 wk, n = 10 . | Group C: emicizumab every 4 wk, n = 10 . | Total, N = 88 . |
|---|---|---|---|---|
| Sex, n (%) | ||||
| Male | 68 (100) | 10 (100) | 10 (100) | 88 (100) |
| Age, y | ||||
| Median (range) | 6.0 (1-15) | 8.0 (2-10) | 9.0 (2-11) | 7.0 (1–15) |
| 0 to <2 | 8 (11.8) | 0 | 0 | 8 (9.1) |
| 2 to <6 | 19 (27.9) | 3 (30.0) | 2 (20.0) | 24 (27.3) |
| 6 to <12 | 38 (55.9) | 7 (70.0) | 8 (80.0) | 53 (60.2) |
| ≥12 | 3 (4.4) | 0 | 0 | 3 (3.4) |
| Race, n (%) | ||||
| White | 39 (57.4) | 7 (70.0) | 8 (80.0) | 54 (61.4) |
| Asian | 10 (14.7) | 1 (10.0) | 2 (20.0) | 13 (14.8) |
| African American | 11 (16.2) | 1 (10.0) | 0 | 12 (13.6) |
| Multiple | 2 (2.9) | 0 | 0 | 2 (2.3) |
| Unknown | 6 (8.8) | 1 (10.0) | 0 | 7 (8.0) |
| Ethnicity, n (%) | ||||
| Hispanic or Latino | 5 (7.4) | 1 (10.0) | 1 (10.0) | 7 (8.0) |
| Not Hispanic or Latino | 61 (89.7) | 9 (90.0) | 9 (90.0) | 79 (89.8) |
| Not stated | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Unknown | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Hemophilia severity at baseline, n (%) | ||||
| Mild | 2 (2.9) | 0 | 0 | 2 (2.3) |
| Moderate | 1 (1.5) | 0 | 0 | 1 (1.1) |
| Severe | 65 (95.6) | 10 (100) | 10 (100) | 85 (96.6) |
| Previous regimen, n (%) | ||||
| Episodic | 16 (23.5) | 2 (20.0) | 4 (40.0) | 22 (25.0) |
| Prophylactic | 52 (76.5) | 8 (80.0) | 6 (60.0) | 66 (75.0) |
| Median no. of bleeds in 24 wk prior to study entry (IQR) | 6 (4-9) | 5 (3-10) | 6 (3-8) | 6 (3.5-9) |
| Median time from FVIII inhibitor diagnosis (range), mo | 56.4 (0.5-142.4) | 71.4 (23.3-120.0) | 66.5 (15.0-120.0) | 58.4 (0.5-142.4) |
| Participants with target joints at baseline, n (%) | ||||
| No target joints present | 44 (64.7) | 3 (30.0) | 7 (70.0) | 54 (61.4) |
| Target joints present | 24 (35.3) | 7 (70.0) | 3 (30.0) | 34 (38.6) |
| Highest historical inhibitor titer, BU/mL | ||||
| n | 66 | 10 | 10 | 86 |
| Median (IQR) | 200 (64-614) | 953 (197-1861) | 133 (29-275) | 200 (64-854) |
| Baseline FVIII inhibitor titer | ||||
| ≥5 BU/mL | 50 (73.5) | 8 (80.0) | 5 (50.0) | 63 (71.6) |
| ≥0.6 BU/mL and <5 BU/mL | 13 (19.1) | 1 (10.0) | 3 (30.0) | 17 (19.3) |
| <0.6 BU/mL | 4 (5.9) | 0 (0.0) | 0 (0.0) | 4 (4.5) |
| Unknown | 1 (1.5) | 1 (10.0) | 2 (20.0) | 4 (4.5) |
| Previously treated with ITI, n (%) | ||||
| Yes | 45 (66.2) | 10 (100) | 8 (80.0) | 63 (71.6) |
| No | 23 (33.8) | 0 | 2 (2.0) | 25 (28.4) |
BU, Bethesda units; IQR, interquartile range.