Figure 1.
Figure 2.
Clinical effectiveness of emicizumab (N=30)
| BEFORE EMICIZUMAB (MEAN - SD / N (%)) | AFTER EMICIZUMAB (MEAN - SD / N (%)) | P-VALUE* | |
|---|---|---|---|
| FVIII inhibitor presence | 15 (50%) | 1 (3.3%) | < 0.001 |
| Joint bleeding episodes | 2.3 ± 1.9 | 0.0 ± 0.0 | < 0.001 |
| ABR | 2.9 ± 2.4 | 0.0 ± 0.0 | < 0.001 |
| Requirement of rescue medication | 22 (73.3%) | 0.0 ± 0.0 | < 0.001 |
| Number of ED visits | 3.3 ± 2.8 | 0.0 ± 0.1 | < 0.001 |
| HRQoL total score | 78.0 ± 5.5 | 30.5 ± 3.7 | < 0.001 |
Demographic and baseline clinical characteristics of the study population (N=30)
| MEAN – SD / N (%) | |
|---|---|
| Age (years) | 16.8 ± 11.4 |
| Minimum | 2 |
| Maximum | 44 |
| Gender | |
| Male | 30 (100%) |
| Previous prophylaxis | |
| Efmoroctocog alfa, FVIII | 24 (80%) |
| Recombinant antihaemophilic FVIII | 1 (3.3%) |
| Efmoroctocog alfa, FVIII and recombinant antihaemophilic FVIII | 5 (16.7%) |
| FVIII inhibitor bypassing activity | 1 (3.3%) |
| Duration of emicizumab therapy (months) | 11 ± 2.3 |
| Reported joint bleeding before emicizumab | |
| No bleeding | 6 (20%) |
| Knee | 12 (40%) |
| Elbow | 7 (23.3%) |
| Ankle | 10 (33.3%) |
| Hip | 1 (3.3%) |
| Calf | 1 (3.3%) |
| Use of rescue medication before emicizumab | |
| None | 8 (26.6%) |
| Recombinant coagulation FVIIa | 6 (20%) |
| Recombinant coagulation FVIIa and FVIII inhibitor bypassing activity | 3 (10%) |
| Efmoroctocog alfa, FVIII | 8 (26.6%) |
| Prothrombin complex concentrate | 5 (16.7%) |
| Indication of rescue medication before emicizumab (N=22) | |
| Joint bleeding | 20 (90.9%) |
| Joint surgery | 1 (4.5%) |
| Port-a-cath site bleeding | 1 (4.5%) |
| Gastrointestinal bleeding | 1 (4.5%) |
| Epistaxis | 1 (4.5%) |