Baseline demographics and characteristics of patients enrolled in verITI-8 (ITI full analysis set)
| Characteristic . | Enrolled patients (N = 16) . |
|---|---|
| Median (range) age at informed consent, y | 2.1 (0.8-16) |
| Median (range) time from inhibitor to start of rFVIIIFc ITI, wk | 23.1 (3.8-744.9∗) |
| Median (range) historical peak inhibitor titer (pre-ITI), BU/mL | 22.4 (6.2-256) |
| Median (range) inhibitor titer at rFVIIIFc ITI start, BU/mL | 9.7 (0.7-144) |
| No. of FVIII exposure d at first confirmed inhibitor development, n (%) | |
| <25 | 8 (50.0) |
| 25-50 | 5 (31.3) |
| 51-100 | 2 (12.5) |
| >100 | 1 (6.3) |
| Family history of inhibitor, n (%) | |
| Yes | 3 (18.8) |
| No | 13 (81.3) |
| Race, n (%) | |
| White | 12 (75.0) |
| Black or African American | 2 (12.5) |
| Other | 2 (12.5) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 2 (12.5) |
| Not Hispanic or Latino | 14 (87.5) |
| F8 genotype, n (%) | |
| Frameshift | 2 (12.5) |
| Large structural change (>50 base pairs) | 10 (62.5) |
| Intron 22 inversion | 7 (43.8) |
| Intron 1 inversion | 1 (6.3) |
| Deletion | 2 (12.5) |
| Small structural change (in-frame, <50 base pairs) | 1 (6.3) |
| Nonsense | 2 (12.5) |
| Missing genotype data | 1 (6.3) |
| Characteristic . | Enrolled patients (N = 16) . |
|---|---|
| Median (range) age at informed consent, y | 2.1 (0.8-16) |
| Median (range) time from inhibitor to start of rFVIIIFc ITI, wk | 23.1 (3.8-744.9∗) |
| Median (range) historical peak inhibitor titer (pre-ITI), BU/mL | 22.4 (6.2-256) |
| Median (range) inhibitor titer at rFVIIIFc ITI start, BU/mL | 9.7 (0.7-144) |
| No. of FVIII exposure d at first confirmed inhibitor development, n (%) | |
| <25 | 8 (50.0) |
| 25-50 | 5 (31.3) |
| 51-100 | 2 (12.5) |
| >100 | 1 (6.3) |
| Family history of inhibitor, n (%) | |
| Yes | 3 (18.8) |
| No | 13 (81.3) |
| Race, n (%) | |
| White | 12 (75.0) |
| Black or African American | 2 (12.5) |
| Other | 2 (12.5) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 2 (12.5) |
| Not Hispanic or Latino | 14 (87.5) |
| F8 genotype, n (%) | |
| Frameshift | 2 (12.5) |
| Large structural change (>50 base pairs) | 10 (62.5) |
| Intron 22 inversion | 7 (43.8) |
| Intron 1 inversion | 1 (6.3) |
| Deletion | 2 (12.5) |
| Small structural change (in-frame, <50 base pairs) | 1 (6.3) |
| Nonsense | 2 (12.5) |
| Missing genotype data | 1 (6.3) |
14.3 years.