Baseline characteristics and demographics
| Parameter . | Cohort 1 n = 3 . | Cohort 2 n = 3 . | Total N = 6 . |
|---|---|---|---|
| Sex, male, n (%) | 3 (100) | 3 (100) | 6 (100) |
| Age, mean (SD), y | 59.3 (9.3) | 41.3 (11.5) | 50.3 (13.6) |
| Range | 53-70 | 28-48 | 28-70 |
| White, n (%) | 3 (100) | 3 (100) | 6 (100) |
| FIX activity, mean (SD), IU/dL | 1.7 (5.8) | 0.9 (0.7) | 1.3 (0.7) |
| Range | 1-2 | 0.1-1.5 | 0.1-2 |
| Receiving FIX therapy, n (%) | |||
| On demand | 1 (33.3) | 1 (33.3) | 2 (33.3) |
| Prophylactic | 2 (66.6) | 2 (66.6) | 4 (66.6) |
| Annualized bleeding rate, mean (SD) | 11.0 (12.2) | 4.7 (5.5) | 7.8 (9.1) |
| Range | 3-25 | 1-11 | 1-25 |
| Parameter . | Cohort 1 n = 3 . | Cohort 2 n = 3 . | Total N = 6 . |
|---|---|---|---|
| Sex, male, n (%) | 3 (100) | 3 (100) | 6 (100) |
| Age, mean (SD), y | 59.3 (9.3) | 41.3 (11.5) | 50.3 (13.6) |
| Range | 53-70 | 28-48 | 28-70 |
| White, n (%) | 3 (100) | 3 (100) | 6 (100) |
| FIX activity, mean (SD), IU/dL | 1.7 (5.8) | 0.9 (0.7) | 1.3 (0.7) |
| Range | 1-2 | 0.1-1.5 | 0.1-2 |
| Receiving FIX therapy, n (%) | |||
| On demand | 1 (33.3) | 1 (33.3) | 2 (33.3) |
| Prophylactic | 2 (66.6) | 2 (66.6) | 4 (66.6) |
| Annualized bleeding rate, mean (SD) | 11.0 (12.2) | 4.7 (5.5) | 7.8 (9.1) |
| Range | 3-25 | 1-11 | 1-25 |