Bleeding outcomes: overall population
| . | Participants (N = 55) . |
|---|---|
| Median (range) follow-up,∗ wk | 101.9 (52.6-119.7) |
| Model-based ABR (95% CI) | |
| All bleeds | 2.0 (1.49-2.66) |
| Treated bleeds | 0.4 (0.30-0.63) |
| Treated spontaneous bleeds | 0.0† |
| Treated joint bleeds | 0.0 (0.01-0.09) |
| Calculated median ABR (IQR) | |
| All bleeds | 1.0 (0.53-2.93) |
| Treated bleeds | 0.0 (0.00-0.81) |
| Treated spontaneous bleeds | 0.0 (0.00-0.00) |
| Treated joint bleeds | 0.0 (0.00-0.00) |
| Participants with 0 bleeds, n (%) | |
| Zero treated or untreated bleeds | 9 (16.4) |
| Zero treated bleeds | 30 (54.5) |
| Zero treated spontaneous bleeds | 55 (100.0) |
| Zero treated joint bleeds | 52 (94.5) |
| Participants with ≥1 bleed, n (%) | 46 (83.6) |
| Total number of bleeds,‡ n | 207 |
| Cause/type of bleed, n (%) | |
| Spontaneous | 18 (8.7) |
| Joint | 0 (0.0) |
| Muscle | 0 (0.0) |
| Other | 18 (100.0) |
| Traumatic | 182 (87.9) |
| Joint | 4 (2.2) |
| Muscle | 5 (2.7) |
| Other | 173 (95.1) |
| Procedural/surgical§ | 7 (3.4) |
| Joint | 0 (0.0) |
| Muscle | 1 (14.3) |
| Other | 6 (85.7) |
| Participants with ≥1 treated bleed, n (%) | 25 (45.5) |
| Total number of treated bleeds, n | 42 |
| Cause/type of treated bleed, n (%) | |
| Traumatic | 42 (100.0) |
| Joint | 3 (7.1) |
| Muscle | 5 (11.9) |
| Other | 34 (81.0) |
| . | Participants (N = 55) . |
|---|---|
| Median (range) follow-up,∗ wk | 101.9 (52.6-119.7) |
| Model-based ABR (95% CI) | |
| All bleeds | 2.0 (1.49-2.66) |
| Treated bleeds | 0.4 (0.30-0.63) |
| Treated spontaneous bleeds | 0.0† |
| Treated joint bleeds | 0.0 (0.01-0.09) |
| Calculated median ABR (IQR) | |
| All bleeds | 1.0 (0.53-2.93) |
| Treated bleeds | 0.0 (0.00-0.81) |
| Treated spontaneous bleeds | 0.0 (0.00-0.00) |
| Treated joint bleeds | 0.0 (0.00-0.00) |
| Participants with 0 bleeds, n (%) | |
| Zero treated or untreated bleeds | 9 (16.4) |
| Zero treated bleeds | 30 (54.5) |
| Zero treated spontaneous bleeds | 55 (100.0) |
| Zero treated joint bleeds | 52 (94.5) |
| Participants with ≥1 bleed, n (%) | 46 (83.6) |
| Total number of bleeds,‡ n | 207 |
| Cause/type of bleed, n (%) | |
| Spontaneous | 18 (8.7) |
| Joint | 0 (0.0) |
| Muscle | 0 (0.0) |
| Other | 18 (100.0) |
| Traumatic | 182 (87.9) |
| Joint | 4 (2.2) |
| Muscle | 5 (2.7) |
| Other | 173 (95.1) |
| Procedural/surgical§ | 7 (3.4) |
| Joint | 0 (0.0) |
| Muscle | 1 (14.3) |
| Other | 6 (85.7) |
| Participants with ≥1 treated bleed, n (%) | 25 (45.5) |
| Total number of treated bleeds, n | 42 |
| Cause/type of treated bleed, n (%) | |
| Traumatic | 42 (100.0) |
| Joint | 3 (7.1) |
| Muscle | 5 (11.9) |
| Other | 34 (81.0) |
At time of the primary analysis, the median age of the participants was 29 months (min-max, 12-39).
IQR, interquartile range.
The start of the efficacy period for each individual participant was defined as the day of the first emicizumab dose. The end of the efficacy period was defined as the date of the clinical cutoff or the date of withdrawal from the study period (ie, “open-label treatment” and “long-term follow-up” according to the eCRF), whichever was earlier.
ABR could not be estimated via the negative binomial regression model because no treated spontaneous bleeds were observed in the study; as a result, a value of 0.0 is reported in the table instead.
Two participants experienced >10 bleeds, none treated and none in a joint or muscle. One participant experienced 27 bleeds (19 traumatic and 8 spontaneous; 20 of 27 being nosebleeds), with the first bleed recorded at ∼11.6 months of age. The other participant experienced 21 bleeds (all traumatic), with the first bleed reported at ∼13.8 months of age.
Of the 7 procedural/surgical bleeds, 4 bleeds were reported in 1 participant and 1 bleed each was reported in 3 participants.