Baseline demographics and disease characteristics across HAVEN 1-4 clinical trials
| . | HAVEN 1 . | HAVEN 2 . | HAVEN 3 . | HAVEN 4 . | Total . |
|---|---|---|---|---|---|
| Patients enrolled, n* | 113 | 88 | 152 | 48 | 401 |
| Participants in ITT group (efficacy population), n | 113 | 88 | 151† | 48 | 400 |
| Participants treated with emicizumab (safety population), n | 112‡ | 88 | 151† | 48 | 399 |
| Duration of exposure, median (IQR), wk | 109.3 (92.1-167.1) | 92.1 (68.3-124.4) | 163.4 (108.1-170.4) | 150.6 (84.4-153.0) | 120.4 (89.0-164.4) |
| Total patient-years of emicizumab exposure | 270.5 | 166.1 | 417.5 | 116.2 | 970.3 |
| Age | |||||
| Median (range), y | 29.0 (12-75) | 7.0 (1-15) | 38.0 (13-77) | 38.0 (14-68) | 28.0 (1-77) |
| <18 y, n (%) | 32 (28.3) | 88 (100) | 8 (5.3) | 4 (8.3) | 132 (32.90) |
| ≥65 y, n (%) | 5 (4.4) | 0 (0) | 5 (3.3) | 3 (6.3) | 13 (3.2) |
| Race, n (%) | |||||
| White | 75 (66.4) | 54 (61.4) | 102 (67.1) | 36 (75.0) | 267 (66.6) |
| Asian | 21 (18.6) | 13 (14.8) | 32 (21.0) | 10 (20.8) | 76 (19.0) |
| African American | 11 (9.7) | 12 (13.6) | 8 (5.3) | 1 (2.1) | 32 (8.0) |
| Other or unknown | 6 (5.3) | 9 (10.2) | 10 (6.6) | 1 (2.1) | 26 (6.4) |
| Previous treatment regimen, n (%) | |||||
| Episodic | 64 (56.6) | 22 (25.0) | 88 (58.3) | 18 (37.5) | 192 (48.0) |
| Prophylactic | 49 (43.4) | 66 (75.0) | 63 (41.7) | 30 (62.5) | 208 (52.0) |
| FVIII inhibitors at baseline, n (%) | |||||
| Yes | 113 (100) | 88 (100) | 0 (0) | 8 (16.7) | 209 (52.1) |
| No | 0 (0) | 0 (0) | 152 (100) | 40 (83.3) | 192 (47.9) |
| Previously underwent immune tolerance induction therapy, n (%) | 58 (51.3) | 63 (71.6) | 0 (0)§ | 6 (12.5) | 127 (31.7) |
| Bleeds in 24 wk prior to study entry, median (IQR), n | 10.0 (6.0-17.0) | 6.0 (3.5-9.0) | 9.0 (3.0-17.0) | 5.0 (2.0-10.5) | 8.0 (5.0-15.0) |
| Presence of target joints¶ at baseline, n (%) | 77 (68.8) | 34 (38.6) | 102 (67.1) | 31 (64.6) | 244 (61.0) |
| . | HAVEN 1 . | HAVEN 2 . | HAVEN 3 . | HAVEN 4 . | Total . |
|---|---|---|---|---|---|
| Patients enrolled, n* | 113 | 88 | 152 | 48 | 401 |
| Participants in ITT group (efficacy population), n | 113 | 88 | 151† | 48 | 400 |
| Participants treated with emicizumab (safety population), n | 112‡ | 88 | 151† | 48 | 399 |
| Duration of exposure, median (IQR), wk | 109.3 (92.1-167.1) | 92.1 (68.3-124.4) | 163.4 (108.1-170.4) | 150.6 (84.4-153.0) | 120.4 (89.0-164.4) |
| Total patient-years of emicizumab exposure | 270.5 | 166.1 | 417.5 | 116.2 | 970.3 |
| Age | |||||
| Median (range), y | 29.0 (12-75) | 7.0 (1-15) | 38.0 (13-77) | 38.0 (14-68) | 28.0 (1-77) |
| <18 y, n (%) | 32 (28.3) | 88 (100) | 8 (5.3) | 4 (8.3) | 132 (32.90) |
| ≥65 y, n (%) | 5 (4.4) | 0 (0) | 5 (3.3) | 3 (6.3) | 13 (3.2) |
| Race, n (%) | |||||
| White | 75 (66.4) | 54 (61.4) | 102 (67.1) | 36 (75.0) | 267 (66.6) |
| Asian | 21 (18.6) | 13 (14.8) | 32 (21.0) | 10 (20.8) | 76 (19.0) |
| African American | 11 (9.7) | 12 (13.6) | 8 (5.3) | 1 (2.1) | 32 (8.0) |
| Other or unknown | 6 (5.3) | 9 (10.2) | 10 (6.6) | 1 (2.1) | 26 (6.4) |
| Previous treatment regimen, n (%) | |||||
| Episodic | 64 (56.6) | 22 (25.0) | 88 (58.3) | 18 (37.5) | 192 (48.0) |
| Prophylactic | 49 (43.4) | 66 (75.0) | 63 (41.7) | 30 (62.5) | 208 (52.0) |
| FVIII inhibitors at baseline, n (%) | |||||
| Yes | 113 (100) | 88 (100) | 0 (0) | 8 (16.7) | 209 (52.1) |
| No | 0 (0) | 0 (0) | 152 (100) | 40 (83.3) | 192 (47.9) |
| Previously underwent immune tolerance induction therapy, n (%) | 58 (51.3) | 63 (71.6) | 0 (0)§ | 6 (12.5) | 127 (31.7) |
| Bleeds in 24 wk prior to study entry, median (IQR), n | 10.0 (6.0-17.0) | 6.0 (3.5-9.0) | 9.0 (3.0-17.0) | 5.0 (2.0-10.5) | 8.0 (5.0-15.0) |
| Presence of target joints¶ at baseline, n (%) | 77 (68.8) | 34 (38.6) | 102 (67.1) | 31 (64.6) | 244 (61.0) |
Data are from Oldenburg et al,8 Young et al,9 Mahlangu et al,10 and Pipe et al.11
ITT, intent-to-treat.
The demographics table is based on those who enrolled (N = 401). Participants included in the efficacy analysis only (N = 400).
One participant in HAVEN 3 assigned to no prophylaxis was lost to follow-up prior to the switch to emicizumab and, therefore, was not treated. This patient was excluded from the efficacy and safety analyses.
One participant in HAVEN 1 assigned to an active arm discontinued prior to first emicizumab treatment and was excluded from the safety analyses.
Historic immune tolerance induction therapy information was not recorded explicitly in the case report form for this noninhibitor study. However, 1 HAVEN 3 participant is known to have undergone immune tolerance induction therapy in 1987, because this information was listed as previous/concomitant medications for the participant.
In line with International Society on Thrombosis and Haemostasis definitions,16 target joints were defined as major joints (eg, hip, elbow, wrist, shoulder, knee, and ankle) in which ≥3 spontaneous bleeding events occurred over a 24-week treatment period.