Overall baseline demographics and characteristics
| . | Overall (N = 33) . |
|---|---|
| Age, mo* | |
| Median | 7.2 |
| Range | 0.96–24.0 |
| Age category, y, n (%) | |
| <1 | 26 (78.8) |
| 1 | 5 (15.2) |
| 2 | 2 (6.1) |
| Race, n (%) | |
| White | 22 (66.7) |
| Black | 1 (3.0) |
| Asian | 1 (3.0) |
| Not reported because of confidentiality regulations | 5 (15.2) |
| Other | 4 (12.1) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 2 (6.1) |
| Not Hispanic or Latino | 26 (78.8) |
| Not reported because of confidentiality regulations | 5 (15.2) |
| Geographic location, n (%)† | |
| Europe | 20 (60.6) |
| North America | 11 (33.3) |
| Other | 2 (6.1) |
| Family history of inhibitors, n (%) | |
| Yes | 6 (18.2) |
| No | 0 (0.0) |
| Unknown | 27 (81.8) |
| F9 genotype, n (%) | |
| Missense | 14 (42.4) |
| Nonsense | 11 (33.3) |
| Promoter or regulatory region mutation | 2 (6.1) |
| Large structure change (>50 base pairs) | 1 (3.0) |
| Frameshift | 1 (3.0) |
| Splice site change | 1 (3.0) |
| Unknown | 3 (9.1) |
| Vaccination within the last year, n (%) | |
| Yes | 27 (81.8) |
| No | 6 (18.2) |
| . | Overall (N = 33) . |
|---|---|
| Age, mo* | |
| Median | 7.2 |
| Range | 0.96–24.0 |
| Age category, y, n (%) | |
| <1 | 26 (78.8) |
| 1 | 5 (15.2) |
| 2 | 2 (6.1) |
| Race, n (%) | |
| White | 22 (66.7) |
| Black | 1 (3.0) |
| Asian | 1 (3.0) |
| Not reported because of confidentiality regulations | 5 (15.2) |
| Other | 4 (12.1) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 2 (6.1) |
| Not Hispanic or Latino | 26 (78.8) |
| Not reported because of confidentiality regulations | 5 (15.2) |
| Geographic location, n (%)† | |
| Europe | 20 (60.6) |
| North America | 11 (33.3) |
| Other | 2 (6.1) |
| Family history of inhibitors, n (%) | |
| Yes | 6 (18.2) |
| No | 0 (0.0) |
| Unknown | 27 (81.8) |
| F9 genotype, n (%) | |
| Missense | 14 (42.4) |
| Nonsense | 11 (33.3) |
| Promoter or regulatory region mutation | 2 (6.1) |
| Large structure change (>50 base pairs) | 1 (3.0) |
| Frameshift | 1 (3.0) |
| Splice site change | 1 (3.0) |
| Unknown | 3 (9.1) |
| Vaccination within the last year, n (%) | |
| Yes | 27 (81.8) |
| No | 6 (18.2) |
Percentages are based on number of patients with data available in safety analysis set.
Age at time of informed consent.
Europe includes Denmark, France, Ireland, Italy, The Netherlands, Poland, Sweden, and the United Kingdom. North America includes Canada and the United States. Other countries include Australia and New Zealand.