ABRs and proportion of participants with 0 and 0 to 3 bleeds over consecutive 24-week intervals among participants of HAVEN 1-4 treated with emicizumab
| . | Weeks 1-24 (n = 391) . | Weeks 25-48 (n = 374) . | Weeks 49-72 (n = 343) . | Weeks 73-96 (n = 283) . | Weeks 97-120 (n = 207) . | Weeks 121-144 (n = 170) . |
|---|---|---|---|---|---|---|
| Treated bleeds | ||||||
| ABR calculated, mean (95% CI) | 1.9 (0.2-7.1) | 0.8 (0-5.2) | 0.8 (0-5.2) | 0.7 (0-5.0) | 0.7 (0-5.0) | 0.7 (0-5.0) |
| Participants with 0 bleeds n, % (95% CI*) | 277, 70.8 (66.1-75.3) | 300, 80.2 (75.8-84.1) | 279, 81.3 (76.8-85.3) | 237, 83.7 (78.9-87.9) | 171, 82.6 (76.8-87.5) | 140, 82.4 (75.8-87.8) |
| Participants with 1-3 bleeds n, % (95% CI*) | 88, 22.5 (18.5–27.0) | 67, 17.9 (14.2-22.2) | 54, 15.7 (12.1-20.0) | 43, 15.2 (11.2-19.9) | 32, 15.5 (10.8-21.1) | 26, 15.3 (10.2-21.6) |
| Participants with 0-3 bleeds n, % (95% CI*) | 365, 93.4 (90.4-95.6) | 367, 98.1 (96.2-99.2) | 333, 97.1 (94.7-98.6) | 280, 98.9 (96.9-99.8) | 203, 98.1 (95.1-99.5) | 166, 97.6 (94.1-99.4) |
| All bleeds | ||||||
| Mean ABR calculated (95% CI) | 3.8 (1.0-9.9) | 2.0 (0.2-7.2) | 1.6 (0.1-6.6) | 1.3 (0.1-6.1) | 1.2 (0.1-5.9) | 1.0 (0-5.5) |
| Participants with 0 bleeds n, % (95% CI*) | 203, 51.9 (46.8–57.0) | 232, 62.0 (56.9-67.0) | 228, 66.5 (61.2-71.5) | 198, 70.0 (64.3-75.3) | 147, 71.0 (64.3-77.1) | 126, 74.1 (66.9-80.5) |
| Participants with 0-3 bleeds n, % (95% CI*) | 332, 84.9 (81.0–88.3) | 349, 93.3 (90.3-95.6) | 323, 94.2 (91.1-96.4) | 275, 97.2 (94.5-98.8) | 201, 97.1 (93.8-98.9) | 166, 97.6 (94.1-99.4) |
| Treated spontaneous bleeds | ||||||
| ABR calculated, mean (95% CI) | 0.8 (0-5.3) | 0.2 (0-4.2) | 0.2 (0-4.2) | 0.2 (0-4.0) | 0.2 (0-4.1) | 0.3 (0-4.2) |
| Participants with 0 bleeds n, % (95% CI*) | 329, 84.1 (80.1-87.6) | 346, 92.5 (89.4-95.0) | 314, 91.5 (88.1-94.3) | 272, 96.1 (93.2-98.0) | 192, 92.8 (88.3-95.9) | 156, 91.8 (86.6-95.4) |
| Participants with 0-3 bleeds n, % (95% CI*) | 383, 98.0 (96.0-99.1) | 372, 99.5 (98.1-99.9) | 343, 100 (98.9-100.0) | 282, 99.6 (98.1-100.0) | 207, 100 (98.2-100.0) | 169, 99.4 (96.8-100.0) |
| Treated joint bleeds | ||||||
| ABR calculated, mean (95% CI) | 1.4 (0.1-6.2) | 0.4 (0-4.6) | 0.5 (0-4.8) | 0.4 (0-4.5) | 0.5 (0-4.6) | 0.4 (0-4.5) |
| Participants with 0 bleeds n, % (95% CI*) | 304, 77.7 (73.3-81.8) | 328, 87.7 (83.9-90.9) | 301, 87.8 (83.8-91.0) | 251, 88.7 (84.4-92.1) | 184, 88.9 (83.8-92.8) | 153, 90.0 (84.5-94.1) |
| Participants with 0-3 bleeds n, % (95% CI*) | 372, 95.1 (92.5-97.1) | 371, 99.2 (97.7-99.8) | 338, 98.5 (96.6-99.5) | 280, 98.9 (96.9-99.8) | 204, 98.6 (95.8-99.7) | 167, 98.2 (94.9-99.6) |
| Treated target joint bleeds | ||||||
| ABR calculated, mean (95% CI) | 0.8 (0-5.2) | 0.3 (0-4.2) | 0.3 (0-4.4) | 0.3 (0-4.2) | 0.2 (0-4.2) | 0.2 (0-4.1) |
| Participants with 0 bleeds n, % (95% CI*) | 336, 85.9 (82.1-89.2) | 350, 93.6 (90.6-95.9) | 320, 93.3 (90.1-95.7) | 264, 93.3 (89.7-95.9) | 193, 93.2 (88.9-96.3) | 160, 94.1 (89.5-97.1) |
| Participants with 0-3 bleeds n, % (95% CI*) | 382, 97.7 (95.7-98.9) | 371, 99.2 (97.7-99.8) | 339, 98.8 (97.0-99.7) | 280, 98.9 (96.9-99.8) | 206, 99.5 (97.3-100.0) | 169, 99.4 (96.8-100.0) |
| . | Weeks 1-24 (n = 391) . | Weeks 25-48 (n = 374) . | Weeks 49-72 (n = 343) . | Weeks 73-96 (n = 283) . | Weeks 97-120 (n = 207) . | Weeks 121-144 (n = 170) . |
|---|---|---|---|---|---|---|
| Treated bleeds | ||||||
| ABR calculated, mean (95% CI) | 1.9 (0.2-7.1) | 0.8 (0-5.2) | 0.8 (0-5.2) | 0.7 (0-5.0) | 0.7 (0-5.0) | 0.7 (0-5.0) |
| Participants with 0 bleeds n, % (95% CI*) | 277, 70.8 (66.1-75.3) | 300, 80.2 (75.8-84.1) | 279, 81.3 (76.8-85.3) | 237, 83.7 (78.9-87.9) | 171, 82.6 (76.8-87.5) | 140, 82.4 (75.8-87.8) |
| Participants with 1-3 bleeds n, % (95% CI*) | 88, 22.5 (18.5–27.0) | 67, 17.9 (14.2-22.2) | 54, 15.7 (12.1-20.0) | 43, 15.2 (11.2-19.9) | 32, 15.5 (10.8-21.1) | 26, 15.3 (10.2-21.6) |
| Participants with 0-3 bleeds n, % (95% CI*) | 365, 93.4 (90.4-95.6) | 367, 98.1 (96.2-99.2) | 333, 97.1 (94.7-98.6) | 280, 98.9 (96.9-99.8) | 203, 98.1 (95.1-99.5) | 166, 97.6 (94.1-99.4) |
| All bleeds | ||||||
| Mean ABR calculated (95% CI) | 3.8 (1.0-9.9) | 2.0 (0.2-7.2) | 1.6 (0.1-6.6) | 1.3 (0.1-6.1) | 1.2 (0.1-5.9) | 1.0 (0-5.5) |
| Participants with 0 bleeds n, % (95% CI*) | 203, 51.9 (46.8–57.0) | 232, 62.0 (56.9-67.0) | 228, 66.5 (61.2-71.5) | 198, 70.0 (64.3-75.3) | 147, 71.0 (64.3-77.1) | 126, 74.1 (66.9-80.5) |
| Participants with 0-3 bleeds n, % (95% CI*) | 332, 84.9 (81.0–88.3) | 349, 93.3 (90.3-95.6) | 323, 94.2 (91.1-96.4) | 275, 97.2 (94.5-98.8) | 201, 97.1 (93.8-98.9) | 166, 97.6 (94.1-99.4) |
| Treated spontaneous bleeds | ||||||
| ABR calculated, mean (95% CI) | 0.8 (0-5.3) | 0.2 (0-4.2) | 0.2 (0-4.2) | 0.2 (0-4.0) | 0.2 (0-4.1) | 0.3 (0-4.2) |
| Participants with 0 bleeds n, % (95% CI*) | 329, 84.1 (80.1-87.6) | 346, 92.5 (89.4-95.0) | 314, 91.5 (88.1-94.3) | 272, 96.1 (93.2-98.0) | 192, 92.8 (88.3-95.9) | 156, 91.8 (86.6-95.4) |
| Participants with 0-3 bleeds n, % (95% CI*) | 383, 98.0 (96.0-99.1) | 372, 99.5 (98.1-99.9) | 343, 100 (98.9-100.0) | 282, 99.6 (98.1-100.0) | 207, 100 (98.2-100.0) | 169, 99.4 (96.8-100.0) |
| Treated joint bleeds | ||||||
| ABR calculated, mean (95% CI) | 1.4 (0.1-6.2) | 0.4 (0-4.6) | 0.5 (0-4.8) | 0.4 (0-4.5) | 0.5 (0-4.6) | 0.4 (0-4.5) |
| Participants with 0 bleeds n, % (95% CI*) | 304, 77.7 (73.3-81.8) | 328, 87.7 (83.9-90.9) | 301, 87.8 (83.8-91.0) | 251, 88.7 (84.4-92.1) | 184, 88.9 (83.8-92.8) | 153, 90.0 (84.5-94.1) |
| Participants with 0-3 bleeds n, % (95% CI*) | 372, 95.1 (92.5-97.1) | 371, 99.2 (97.7-99.8) | 338, 98.5 (96.6-99.5) | 280, 98.9 (96.9-99.8) | 204, 98.6 (95.8-99.7) | 167, 98.2 (94.9-99.6) |
| Treated target joint bleeds | ||||||
| ABR calculated, mean (95% CI) | 0.8 (0-5.2) | 0.3 (0-4.2) | 0.3 (0-4.4) | 0.3 (0-4.2) | 0.2 (0-4.2) | 0.2 (0-4.1) |
| Participants with 0 bleeds n, % (95% CI*) | 336, 85.9 (82.1-89.2) | 350, 93.6 (90.6-95.9) | 320, 93.3 (90.1-95.7) | 264, 93.3 (89.7-95.9) | 193, 93.2 (88.9-96.3) | 160, 94.1 (89.5-97.1) |
| Participants with 0-3 bleeds n, % (95% CI*) | 382, 97.7 (95.7-98.9) | 371, 99.2 (97.7-99.8) | 339, 98.8 (97.0-99.7) | 280, 98.9 (96.9-99.8) | 206, 99.5 (97.3-100.0) | 169, 99.4 (96.8-100.0) |
95% CIs were calculated using the Clopper-Pearson method.
95% CIs of proportion are indicated.