Table 3.
Emicizumab treatment for the complete cohort
| Total cohort (N = 62) | |
|---|---|
| Reason for emicizumab initiation∗ | |
| Bleeding prophylaxis | 50 (80.6%) |
| Facilitate outpatient management | 37 (59.7%) |
| Decrease immunosuppression | 21 (33.9%) |
| Bleeding despite use of other hemostatic agents | 20 (32.3%) |
| Failure to respond to immunosuppression | 10 (16.1%) |
| Presumed or confirmed antibody to porcine FVIII | 5 (8.1%) |
| Time from diagnosis to emicizumab initiation (d), median (range) | 19 (0-1461) |
| Time to bleeding resolution after emicizumab initiation (d), median (range)† | 7 (1-32) |
| Emicizumab impact on immunosuppression plan | |
| Allowed reduced or no concurrent steroid regimen | 21 (33.9%) |
| Allowed no concurrent immunosuppression therapy | 4 (6.5%) |
| Other impact‡ | 4 (6.5%) |
| No impact | 33 (53.2%) |
| Emicizumab loading dosage | 52 (83.9%) |
| 2.5-3 mg/kg weekly§ | 2 (3.2%) |
| 1.5 mg/kg weekly for 4 wk | 5 (8.1%) |
| 6 mg/kg once, then 3 mg/kg weekly | 1 (1.6%) |
| 3 mg/kg once | 1 (1.6%) |
| No loading regimen | 1 (1.6%) |
| Other | |
| Emicizumab maintenance dosage | |
| 1.5 mg/kg weekly | 26 (41.9%) |
| 3 mg/kg every 2 wk | 15 (24.2%) |
| 1.5 mg/kg every 2 wk | 2 (3.2%) |
| 6 mg/kg every 4 wk | 2 (3.2%) |
| 3 mg/kg every 4 wk | 0 (0%) |
| None | 14 (22.6%) |
| Other | 3 (4.8%) |
| Emicizumab dose adjustments | |
| Reduced|| | 2 (3.2%) |
| Increased (for breakthrough bleed) | 1 (1.6%) |
| No change | 59 (95.2%) |
| Emicizumab duration (wk), median (range)¶ | 10 (1-52) |
| Emicizumab discontinued at time of survey | 46 (74.2%) |
| Reason(s) for emicizumab discontinuation,∗n = 46 | |
| Resolution of inhibitor | 27 (58.7%) |
| Resolution of bleeding and/or rising FVIII | 20 (43.4%) |
| Death or loss to follow-up | 4 (8.7%) |
| Adverse event (arthralgias) | 1 (2.2%) |
| Cost/insurance coverage | 1 (2.2%) |
Percentages do not add up to 100% because respondents could select multiple answers for this variable.
Time to bleeding resolution only calculated for the 30 patients who had ongoing bleeding at the time of initiation.
Other IST modifications included allowed steroid monotherapy (n = 2) and altered timing and intensity of IST regimens (besides steroids; n = 2).
Loading dosage was given weekly for 2 to 5 weeks.
Emicizumab was reduced in 2 patients; 1 developed a deep venous thrombosis after which emicizumab was stopped before the dose was reduced, the other had the dose reduced after the patient’s bleeding resolved.
Data available for only 46 patients who had completed their emicizumab course at the time of survey submission.