Participant demographics, baseline clinical characteristics, and medical history
| . | Participants (N = 55) . |
|---|---|
| Age at informed consent, mo | |
| Mean (SD) | 5.0 (3.9) |
| Median (range) | 4.0 (9 days to 11 months 30 days) |
| Age group, n (%) | |
| 0-<3 mo | 25 (45.5) |
| 3-12 mo | 30 (54.5) |
| Sex, n (%), male | 55 (100) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 5 (9.1) |
| Not Hispanic or Latino | 49 (89.1) |
| Unknown | 1 (1.8) |
| Race, n (%) | |
| Asian | 3 (5.5) |
| Black or African American | 1 (1.8) |
| Native Hawaiian or other Pacific Islander | 1 (1.8) |
| White | 48 (87.3) |
| Unknown | 2 (3.6) |
| Weight at baseline, kg | |
| Median (range) | 7.1 (3.2-12.0) |
| Mode of delivery, n (%) | |
| Vaginal delivery (not assisted) | 18 (32.7) |
| Vaginal delivery (assisted) | 6 (10.9) |
| Planned cesarean delivery | 30 (54.4) |
| Emergency cesarean delivery | 1 (1.8) |
| Family history of HA, n (%) | 41 (74.5) |
| Family history of FVIII inhibitors | 7 (12.7) |
| Prior treatment status, n (%) | |
| MTP∗ | 30 (54.5) |
| PUP | 25 (45.5) |
| Hemophilia treatments received prior to first emicizumab dose | |
| Participants with ≥1 treatment, n (%) | 34 (61.8) |
| Total number of treatments, n | 85 |
| Purpose of treatment, n (%) | |
| Treatment for a bleed | 30 (54.5) |
| Preventive dose before activity | 4 (7.3) |
| Preventive dose for procedure/surgery | 3 (5.5) |
| Historical bleeding episodes prior to first emicizumab dose | |
| Participants with ≥1 bleed, n (%) | 36 (65.5) |
| Total number of bleeds, n | 77 |
| Cause/type of bleed, n (%) | |
| Spontaneous | 25 (32.5) |
| Joint | 8 (32.0)† |
| Muscle | 6 (24.0)‡ |
| Other | 11 (44.0)§ |
| Traumatic | 19 (24.7) |
| Joint | 0 (0.0) |
| Muscle | 1 (5.3)‡ |
| Other | 18 (94.7)§ |
| Procedural/surgical | 33 (42.9) |
| Joint | 0 (0.0) |
| Muscle | 8 (24.2)‡ |
| Other | 25 (75.8)§ |
| . | Participants (N = 55) . |
|---|---|
| Age at informed consent, mo | |
| Mean (SD) | 5.0 (3.9) |
| Median (range) | 4.0 (9 days to 11 months 30 days) |
| Age group, n (%) | |
| 0-<3 mo | 25 (45.5) |
| 3-12 mo | 30 (54.5) |
| Sex, n (%), male | 55 (100) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 5 (9.1) |
| Not Hispanic or Latino | 49 (89.1) |
| Unknown | 1 (1.8) |
| Race, n (%) | |
| Asian | 3 (5.5) |
| Black or African American | 1 (1.8) |
| Native Hawaiian or other Pacific Islander | 1 (1.8) |
| White | 48 (87.3) |
| Unknown | 2 (3.6) |
| Weight at baseline, kg | |
| Median (range) | 7.1 (3.2-12.0) |
| Mode of delivery, n (%) | |
| Vaginal delivery (not assisted) | 18 (32.7) |
| Vaginal delivery (assisted) | 6 (10.9) |
| Planned cesarean delivery | 30 (54.4) |
| Emergency cesarean delivery | 1 (1.8) |
| Family history of HA, n (%) | 41 (74.5) |
| Family history of FVIII inhibitors | 7 (12.7) |
| Prior treatment status, n (%) | |
| MTP∗ | 30 (54.5) |
| PUP | 25 (45.5) |
| Hemophilia treatments received prior to first emicizumab dose | |
| Participants with ≥1 treatment, n (%) | 34 (61.8) |
| Total number of treatments, n | 85 |
| Purpose of treatment, n (%) | |
| Treatment for a bleed | 30 (54.5) |
| Preventive dose before activity | 4 (7.3) |
| Preventive dose for procedure/surgery | 3 (5.5) |
| Historical bleeding episodes prior to first emicizumab dose | |
| Participants with ≥1 bleed, n (%) | 36 (65.5) |
| Total number of bleeds, n | 77 |
| Cause/type of bleed, n (%) | |
| Spontaneous | 25 (32.5) |
| Joint | 8 (32.0)† |
| Muscle | 6 (24.0)‡ |
| Other | 11 (44.0)§ |
| Traumatic | 19 (24.7) |
| Joint | 0 (0.0) |
| Muscle | 1 (5.3)‡ |
| Other | 18 (94.7)§ |
| Procedural/surgical | 33 (42.9) |
| Joint | 0 (0.0) |
| Muscle | 8 (24.2)‡ |
| Other | 25 (75.8)§ |
Age is calculated relative to the date when the informed consent form was signed.
Defined as a participant with ≤5 EDs to hemophilia-related treatments containing FVIII, such as plasma-derived FVIII, recombinant FVIII, fresh/frozen plasma, cryoprecipitate, or whole blood products.
Of the 8 total prestudy joint bleeds, 2 each occurred in the elbow and hip and 1 each occurred in the ankle, fingers/thumb, knee, and shoulder.
Of the 15 total prestudy muscle bleeds, the majority (8 bleeds [n = 7 participants]) occurred in the thigh. HA diagnosis was known in 4 of these participants at the time of these bleeds in the thigh. Six of the 8 bleeds in the thigh were procedural bleeds: 3 for vaccination (n =3 participants); 3 for vitamin K administration (n = 2 participants; [in 1 participant, 1 bleed in the left thigh; in another participant, 1 bleed in the left thigh and 1 bleed in the right thigh]). The remaining 2 bleeds in the thigh were 2 spontaneous bleeds in the left thigh (n = 2 participants).
Of the 54 total other prestudy bleeds, 9 occurred in the sole/heel (all due to heel prick for metabolic tests [n = 6 participants], with 2 of 6 having received HA diagnosis at the time of these bleeds), 6 in the back of the hand or the mouth (HA diagnosis known in 5 of 6 cases [n = 4 participants] at the time of these bleeds), and the remainder were distributed across the rest of the body.