Demographics and baseline characteristics (EAS 1)
| Characteristic . | Participants with inhibitors (n = 19) . | Participants without inhibitors (n = 46) . | All (N = 65) . |
|---|---|---|---|
| Age at baseline, y | |||
| Mean (SD) | 27.8 (17.1) | 23.5 (7.3) | 24.8 (11.2) |
| Median (IQR) | 23 (15.0-30.0) | 23 (17.0-29.0) | 23 (16.0-29.0) |
| Age group at baseline, n (%) | |||
| 12-17 y | 7 (36.8) | 12 (26.1) | 19 (29.2) |
| 18-64 y | 11 (57.9) | 34 (73.9) | 45 (69.2) |
| ≥65 y | 1 (5.3) | 0 | 1 (1.5) |
| Sex, male, n (%) | 19 (100) | 46 (100) | 65 (100) |
| Hemophilia type, n (%) | |||
| Hemophilia A | 14 (73.7) | 36 (78.3) | 50 (76.9) |
| Hemophilia B | 5 (26.3) | 10 (21.7) | 15 (23.1) |
| Weight, mean (SD), kg | 68.2 (16.2) | 71.7 (15.3) | 70.7 (15.5) |
| BMI, mean (SD), kg/m2 | 23.7 (4.6) | 23.6 (4.1) | 23.6 (4.2) |
| Race, n (%) | |||
| White | 15 (78.9) | 27 (58.7) | 42 (64.6) |
| Asian | 3 (15.8) | 17 (37.0) | 20 (30.8) |
| Black or African American | 1 (5.3) | 0 | 1 (1.5) |
| Other | 0 | 2 (4.3) | 2 (3.1) |
| Region, n (%) | |||
| North America | 0 | 0 | 0 |
| Europe | 15 (78.9) | 28 (60.9) | 43 (66.2) |
| Asia | 3 (15.8) | 17 (37.0) | 20 (30.8) |
| Other | 1 (5.3) | 1 (2.2) | 2 (3.1) |
| Number of bleeding episodes in the last 6 months before screening, mean (SD)∗ | 4.6 (2.6) | NR | NR |
| Number of bleeding episodes in the last 12 months before screening, mean (SD)∗ | NR | 4.0 (5.2) | NR |
| Inhibitor type, n (%) | |||
| Highest historical inhibitor titer result† | |||
| <5 BU/mL | 1 (5.3) | N/A | 1 (1.5) |
| ≥5 BU/mL | 18 (94.7) | N/A | 18 (27.7) |
| Number of target joints‡, median (IQR) | 0.0 (0.0-1.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) |
| Characteristic . | Participants with inhibitors (n = 19) . | Participants without inhibitors (n = 46) . | All (N = 65) . |
|---|---|---|---|
| Age at baseline, y | |||
| Mean (SD) | 27.8 (17.1) | 23.5 (7.3) | 24.8 (11.2) |
| Median (IQR) | 23 (15.0-30.0) | 23 (17.0-29.0) | 23 (16.0-29.0) |
| Age group at baseline, n (%) | |||
| 12-17 y | 7 (36.8) | 12 (26.1) | 19 (29.2) |
| 18-64 y | 11 (57.9) | 34 (73.9) | 45 (69.2) |
| ≥65 y | 1 (5.3) | 0 | 1 (1.5) |
| Sex, male, n (%) | 19 (100) | 46 (100) | 65 (100) |
| Hemophilia type, n (%) | |||
| Hemophilia A | 14 (73.7) | 36 (78.3) | 50 (76.9) |
| Hemophilia B | 5 (26.3) | 10 (21.7) | 15 (23.1) |
| Weight, mean (SD), kg | 68.2 (16.2) | 71.7 (15.3) | 70.7 (15.5) |
| BMI, mean (SD), kg/m2 | 23.7 (4.6) | 23.6 (4.1) | 23.6 (4.2) |
| Race, n (%) | |||
| White | 15 (78.9) | 27 (58.7) | 42 (64.6) |
| Asian | 3 (15.8) | 17 (37.0) | 20 (30.8) |
| Black or African American | 1 (5.3) | 0 | 1 (1.5) |
| Other | 0 | 2 (4.3) | 2 (3.1) |
| Region, n (%) | |||
| North America | 0 | 0 | 0 |
| Europe | 15 (78.9) | 28 (60.9) | 43 (66.2) |
| Asia | 3 (15.8) | 17 (37.0) | 20 (30.8) |
| Other | 1 (5.3) | 1 (2.2) | 2 (3.1) |
| Number of bleeding episodes in the last 6 months before screening, mean (SD)∗ | 4.6 (2.6) | NR | NR |
| Number of bleeding episodes in the last 12 months before screening, mean (SD)∗ | NR | 4.0 (5.2) | NR |
| Inhibitor type, n (%) | |||
| Highest historical inhibitor titer result† | |||
| <5 BU/mL | 1 (5.3) | N/A | 1 (1.5) |
| ≥5 BU/mL | 18 (94.7) | N/A | 18 (27.7) |
| Number of target joints‡, median (IQR) | 0.0 (0.0-1.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) |
EAS 1 includes all participants who received BPA/CFC prophylaxis and at least 1 dose of 80 mg fitusiran before dose resumption (after the sponsor initiated pause in dosing).
BMI, body mass index; BU, Bethesda unit; N/A, not applicable; NR, not reported.
Number of bleeding episodes in the last 6 months was only reported for participants with inhibitors and in the last 12 months only for participants without inhibitors.
Bethesda assay.
A target joint is defined as a joint where 3 or more spontaneous bleeding episodes in a single joint within a consecutive 6-month period has occurred; where there have been ≤2 bleeding episodes in the joint within a consecutive 12-month period, the joint is no longer considered a target joint.