Table 1.
Patient demographics and characteristics
| Total (N = 37) | |
|---|---|
| Age, y | |
| Median | 1.0 |
| Min, max | 0, 4 |
| Race, n (%) a | |
| White | 18 (48.6) |
| Asian | 11 (29.7) |
| Black or African American | 5 (13.5) |
| Other | 3 (8.1) |
| Mutation, n b , c | |
| Missense | 21 |
| Nonsense | 6 |
| Splice site | 4 |
| Small deletion | 1 |
| Small duplication | 1 |
| Small insertion | 1 |
| No mutation identified b | 2 |
| Known family history | |
| Hemophilia, n (%) | 15 (40.5) |
| Inhibitor, n | 1 |
Patients who switched from preprophylaxis to prophylaxis are included in all columns.
a
Patients were enrolled from centers in Australia (n = 2), Austria (n = 2), Canada (n = 1), Israel (n = 2), Malaysia (n = 4), Spain (n = 3), Taiwan (n = 3), Thailand (n = 4), the United Kingdom (n = 3), and the United States (n = 13).
b
At the time of data cutoff.
c
Data analyzed in 36 patients so far.