Demographics and baseline characteristics
| Variable . | Cohort 1 (n = 5) . | Cohort 2 (n = 5) . |
|---|---|---|
| Age, y | 69 (35-72) | 35 (33-46) |
| Weight, kg | 84.5 (71.2-89.1) | 84.0 (71.4-96.0) |
| FIX use | ||
| Prophylaxis, IU/week* | 4000 (2000-8000) | 4000 (4000-10 500) |
| Annualized mean, IU/y | 354 800 | 173 200 |
| Mean bleeds in the year before enrollment†, n | ||
| Total | 14.4 | 4.0 |
| Spontaneous | 9.8 | 3.0 |
| Traumatic | 2.8 | 1.0 |
| Unknown | 1.8 | 0.0 |
| Hemophilia Joint Health Scores‡ | 27 (2-49) | 6 (0-17) |
| HIV-positive status, n | 1 | 0 |
| Prior hepatitis C infection, n | 4 | 2 |
| Variable . | Cohort 1 (n = 5) . | Cohort 2 (n = 5) . |
|---|---|---|
| Age, y | 69 (35-72) | 35 (33-46) |
| Weight, kg | 84.5 (71.2-89.1) | 84.0 (71.4-96.0) |
| FIX use | ||
| Prophylaxis, IU/week* | 4000 (2000-8000) | 4000 (4000-10 500) |
| Annualized mean, IU/y | 354 800 | 173 200 |
| Mean bleeds in the year before enrollment†, n | ||
| Total | 14.4 | 4.0 |
| Spontaneous | 9.8 | 3.0 |
| Traumatic | 2.8 | 1.0 |
| Unknown | 1.8 | 0.0 |
| Hemophilia Joint Health Scores‡ | 27 (2-49) | 6 (0-17) |
| HIV-positive status, n | 1 | 0 |
| Prior hepatitis C infection, n | 4 | 2 |
Values are median (minimum-maximum) unless otherwise stated.
One participant in cohort 2 received on-demand treatment and was therefore not included.
Historical bleed data was missing for 1 participant in cohort 2, who was therefore not included.
Joint status was assessed using the Hemophilia Joint Health Score version 2.1.20