Study overview and outcomes
| ID sex . | Capsid (dose in vg/kg) . | Treat age (y) . | Follow-up (y) . | Post ABR (pre ABR) . | Pre . | Early WBCT Y0-Y3 CSA FVIII:C Y0-Y4 . | Mid WBCT Y3-Y7 CSA FVIII:C Y4-Y8 . | Late WBCT Y7-Y11 CSA FVIII:C Y8-Y12 . | Final CSA FVIII:C (OSA FVIII:C) . | Liver DNA vg/dg (RNA copies/ng) . | Spleen DNA vg/dg (RNA copies/ng) . | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| WBCT (min) . | WBCT (min) . | FVIII:C (%) . | WBCT (min) . | FVIII:C (%) . | WBCT (min) . | FVIII:C (%) . | ||||||||
| ELI (F) | 2 6.0e12 | 0.6 | 8.2 | 0.4 (5.2) | 15.9 12.0-22.0 | 5.6 3.6-16.0 | 3.4 2.7-4.0 | 5.7 4.4-7.6 | n/a | 4.8 4.6-5.1 | n/a | n/a (n/a) | 0.044 (6.2) | 0.001 (n/d) |
| VC (M) | 2 1.5e13 | 0.7 | 11.0 | 0.7 (7.2) | 15.4 13.1-17.1 | 5.9 3.0-8.7 | 2.8 2.3-3.3 | n/a | 2.8 1.7-4.0 | 6.4 4.9-7.3 | 3.0 2.3-4.0 | 2.9% (7.3%) | 0.592 (8.3) | n/d (n/d) |
| JUN (M) | 2 2.7e13 | 1.0 | 12.0 | 0.2 (0.0) | 15.9 15.7-16.2 | 5.6 3.4-11.9 | 5.8 4.7-6.9 | 4.2 3.2-4.8 | n/a | 5.6 5.1-6.2 | 6.7 6.1-7.2 | 7.2% (12.7%) | 0.247 (15.9) | 0.005 (n/d) |
| ALX (F) | 6 1.0e13 | 0.7 | 10.5 | 0.0 (2.8) | 14.8 13.0-16.2 | 4.3 2.8-6.3 | 6.6 3.7-9.4 | 4.2 2.1-6.2 | 4.9† | 6.8 6.8-6.9 | 6.7 4.9-8.6 | 8.6% (14.9%) | 0.177 (26.4) | n/d (n/d) |
| MZ (M) | 6 1.0e13 | 1.3 | 10.1 | 0.1 (2.1) | 14.9 10.7-18.7 | 4.7 3.3-5.9 | 4.0 2.4-5.8 | 5.9† | 4.3† | 5.2 5.2-5.2 | 6.2 4.0-7.9 | 7.9% (13.9%) | 0.173 (5.9) | n/d (n/d) |
| FLO (F) | 8 1.0e13 | 1.0 | 10.5 | 0.1 (2.1) | 14.5 13.2-16.3 | 5.3 3.1-7.3 | 2.8 1.8-4.1 | 4.1 1.9-6.3 | 1.6† | 3.2† | 2.5 1.8-3.2 | 1.8% (n/a) | 0.127 (4.4) | 0.033 (n/d) |
| ANG∗ (M) | 2 1.5e13 | 1.0 | 11.5 | 1.5 (5.2) | 15.4 14.0-16.0 | 6.8 4.9-9.9 | n/a | 7.8† | 0.4† | 6.0 4.6-6.9 | 0.5 0.0-0.8 | 0.5% (3.2%) | 0.053 (n/d) | 0.005 (n/d) |
| MG∗ (F) | 6 1.7e13 | 0.5 | 11.5 | 0.9 (10.0) | 16.1 15.9-16.3 | 6.9 4.1-9.7 | n/a | 6.4 5.2-7.7 | 0.8† | 7.7 6.2-13.0 | 0.2 0.0-0.3 | 0.3% (1.7%) | 0.014 (n/d) | n/d (n/d) |
| ID sex . | Capsid (dose in vg/kg) . | Treat age (y) . | Follow-up (y) . | Post ABR (pre ABR) . | Pre . | Early WBCT Y0-Y3 CSA FVIII:C Y0-Y4 . | Mid WBCT Y3-Y7 CSA FVIII:C Y4-Y8 . | Late WBCT Y7-Y11 CSA FVIII:C Y8-Y12 . | Final CSA FVIII:C (OSA FVIII:C) . | Liver DNA vg/dg (RNA copies/ng) . | Spleen DNA vg/dg (RNA copies/ng) . | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| WBCT (min) . | WBCT (min) . | FVIII:C (%) . | WBCT (min) . | FVIII:C (%) . | WBCT (min) . | FVIII:C (%) . | ||||||||
| ELI (F) | 2 6.0e12 | 0.6 | 8.2 | 0.4 (5.2) | 15.9 12.0-22.0 | 5.6 3.6-16.0 | 3.4 2.7-4.0 | 5.7 4.4-7.6 | n/a | 4.8 4.6-5.1 | n/a | n/a (n/a) | 0.044 (6.2) | 0.001 (n/d) |
| VC (M) | 2 1.5e13 | 0.7 | 11.0 | 0.7 (7.2) | 15.4 13.1-17.1 | 5.9 3.0-8.7 | 2.8 2.3-3.3 | n/a | 2.8 1.7-4.0 | 6.4 4.9-7.3 | 3.0 2.3-4.0 | 2.9% (7.3%) | 0.592 (8.3) | n/d (n/d) |
| JUN (M) | 2 2.7e13 | 1.0 | 12.0 | 0.2 (0.0) | 15.9 15.7-16.2 | 5.6 3.4-11.9 | 5.8 4.7-6.9 | 4.2 3.2-4.8 | n/a | 5.6 5.1-6.2 | 6.7 6.1-7.2 | 7.2% (12.7%) | 0.247 (15.9) | 0.005 (n/d) |
| ALX (F) | 6 1.0e13 | 0.7 | 10.5 | 0.0 (2.8) | 14.8 13.0-16.2 | 4.3 2.8-6.3 | 6.6 3.7-9.4 | 4.2 2.1-6.2 | 4.9† | 6.8 6.8-6.9 | 6.7 4.9-8.6 | 8.6% (14.9%) | 0.177 (26.4) | n/d (n/d) |
| MZ (M) | 6 1.0e13 | 1.3 | 10.1 | 0.1 (2.1) | 14.9 10.7-18.7 | 4.7 3.3-5.9 | 4.0 2.4-5.8 | 5.9† | 4.3† | 5.2 5.2-5.2 | 6.2 4.0-7.9 | 7.9% (13.9%) | 0.173 (5.9) | n/d (n/d) |
| FLO (F) | 8 1.0e13 | 1.0 | 10.5 | 0.1 (2.1) | 14.5 13.2-16.3 | 5.3 3.1-7.3 | 2.8 1.8-4.1 | 4.1 1.9-6.3 | 1.6† | 3.2† | 2.5 1.8-3.2 | 1.8% (n/a) | 0.127 (4.4) | 0.033 (n/d) |
| ANG∗ (M) | 2 1.5e13 | 1.0 | 11.5 | 1.5 (5.2) | 15.4 14.0-16.0 | 6.8 4.9-9.9 | n/a | 7.8† | 0.4† | 6.0 4.6-6.9 | 0.5 0.0-0.8 | 0.5% (3.2%) | 0.053 (n/d) | 0.005 (n/d) |
| MG∗ (F) | 6 1.7e13 | 0.5 | 11.5 | 0.9 (10.0) | 16.1 15.9-16.3 | 6.9 4.1-9.7 | n/a | 6.4 5.2-7.7 | 0.8† | 7.7 6.2-13.0 | 0.2 0.0-0.3 | 0.3% (1.7%) | 0.014 (n/d) | n/d (n/d) |