FVIII activity levels over time
| Time after vector (months) . | Rollover population N = 112 . | Directly enrolled population N = 22 . | ||
|---|---|---|---|---|
| CSA . | OSA . | CSA . | OSA . | |
| Month 12 | N = 111 | N = 111 | N = 21 | N = 21 |
| Mean (SD) | 43.6 (45.5) | 64.7 (64.6) | 38.2 (46.3) | 59.7 (67.0) |
| Median (Q1, Q3) | 24 (12.5, 63.7) | 40.0 (20.4, 87.5) | 23.9 (11.2, 52.8) | 40.5 (17.4, 82.6) |
| Min, max | 0, 231.2 | 0, 311.11 | 1.6, 207.4 | 4.4, 294.1 |
| Month 24 | N = 98 | N = 99 | N = 19 | N = 18 |
| Mean (SD) | 25.0 (35.5) | 38.9 (50.7) | 22.0 (28.7) | 36.0 (40.8) |
| Median (Q1, Q3) | 12.7 (5.1, 26.5) | 22.7 (7.9, 45.7) | 8.9 (5.8, 25.9) | 19.5 (7.9, 37.7) |
| Min, max | 0, 187.1 | 0, 271.3 | 0, 110.6 | 2.4, 146.7 |
| Month 36 | N = 96 | N = 97 | N = 15 | N = 15 |
| Mean (SD) | 21.0 (34.0) | 33.8 (47.6) | 20.8 (24.4) | 32.2 (33.1) |
| Median (Q1, Q3) | 10.0 (4.3, 19.8) | 17.1 (7.2, 35.1) | 9.4 (6.6, 31.7) | 20.6 (8.5, 46.7) |
| Min, Max | 0, 217.7 | 0, 291.4 | 0, 74.5 | 1.9, 104.2 |
| Time after vector (months) . | Rollover population N = 112 . | Directly enrolled population N = 22 . | ||
|---|---|---|---|---|
| CSA . | OSA . | CSA . | OSA . | |
| Month 12 | N = 111 | N = 111 | N = 21 | N = 21 |
| Mean (SD) | 43.6 (45.5) | 64.7 (64.6) | 38.2 (46.3) | 59.7 (67.0) |
| Median (Q1, Q3) | 24 (12.5, 63.7) | 40.0 (20.4, 87.5) | 23.9 (11.2, 52.8) | 40.5 (17.4, 82.6) |
| Min, max | 0, 231.2 | 0, 311.11 | 1.6, 207.4 | 4.4, 294.1 |
| Month 24 | N = 98 | N = 99 | N = 19 | N = 18 |
| Mean (SD) | 25.0 (35.5) | 38.9 (50.7) | 22.0 (28.7) | 36.0 (40.8) |
| Median (Q1, Q3) | 12.7 (5.1, 26.5) | 22.7 (7.9, 45.7) | 8.9 (5.8, 25.9) | 19.5 (7.9, 37.7) |
| Min, max | 0, 187.1 | 0, 271.3 | 0, 110.6 | 2.4, 146.7 |
| Month 36 | N = 96 | N = 97 | N = 15 | N = 15 |
| Mean (SD) | 21.0 (34.0) | 33.8 (47.6) | 20.8 (24.4) | 32.2 (33.1) |
| Median (Q1, Q3) | 10.0 (4.3, 19.8) | 17.1 (7.2, 35.1) | 9.4 (6.6, 31.7) | 20.6 (8.5, 46.7) |
| Min, Max | 0, 217.7 | 0, 291.4 | 0, 74.5 | 1.9, 104.2 |
FVIII activity levels in IU/dL; data are from the FDA summary basis for regulatory action of valoctocogene raxaparvovec.48
Min, minimum; max, maximum; SD, standard deviation.