Demographics and clinical characteristics at baseline
| Original dose regimen . | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| . | Inhibitor (n = 15) . | Noninhibitor (n = 19) . | Overall (N = 34) . | ||||||
| Hemophilia A (n = 13) . | Hemophilia B (n = 2) . | Total (n = 15) . | Hemophilia A (n = 14) . | Hemophilia B (n = 5) . | Total (n = 19) . | Hemophilia A (n = 27) . | Hemophilia B (n = 7) . | Total (n = 34) . | |
| Mean age (SD), y | 34.0 (6.9) | 25.0 (2.8) | 32.8 (7.2) | 40.6 (12.8) | 36.6 (10.9) | 39.6 (12.2) | 37.4 (10.8) | 33.3 (10.6) | 36.6 (10.7) |
| Weight, mean (SD), kg | 74.9 (15.1) | 85.7 (6.5) | 76.3 (14.6) | 77.1 (10.0) | 74.1 (14.2) | 76.4 (10.9) | 76.1 (12.5) | 77.4 (13.2) | 76.3 (12.4) |
| Race, n (%) | |||||||||
| Caucasian/White | 13 (100) | 2 (100) | 15 (100) | 13 (92.9) | 5 (100) | 18 (94.7) | 26 (96.3) | 7 (100) | 33 (97.1) |
| Asian/Oriental | 0 | 0 | 0 | 1 (7.1) | 0 | 1 (5.3) | 1 (3.7) | 0 | 1 (2.9) |
| Region, n (%) | |||||||||
| North America | 0 | 1 (50.0) | 1 (6.7) | 1 (7.1) | 0 | 1 (5.3) | 1 (3.7) | 1 (14.3) | 2 (5.9) |
| Europe | 13 (100) | 1 (50.0) | 14 (93.3) | 13 (92.9) | 5 (100) | 18 (94.7) | 26 (96.3) | 6 (85.7) | 32 (94.1) |
| Moderate FVIII or FIX (1%-5%), n (%) | 0 | 0 | 0 | 1 (7.1) | 2 (40.0) | 3 (15.8) | 1 (3.7) | 2 (28.6) | 3 (8.8) |
| Severe FVIII or FIX (<1%), n (%) | 13 (100) | 2 (100) | 15 (100) | 13 (92.9) | 3 (60.0) | 16 (84.2) | 26 (96.3) | 5 (71.4) | 31 (91.2) |
| Historic ABR at parent study entry, median (Q1; Q3) | 40.0 (20.0; 48.0) | 40.0 (26.0; 54.0) | 40.0 (20.0; 48.0) | 7.0 (0.0; 24.0) | 4.0 (4.0; 6.0) | 4.0 (0.0; 22.0) | 20.0 (4.0; 40.0) | 6.0 (4.0; 26.0) | 20.0 (4.0; 38.0) |
| History of hepatitis C, n (%) | 11 (84.6) | 0 | 11 (73.3) | 12 (85.7) | 3 (60.0) | 15 (78.9) | 23 (85.2) | 3 (42.9) | 26 (76.5) |
| Exposure, median (Q1; Q3), d∗ | 1054.0 (359.0; 1138.0) | 1271.0 (1101.0; 1441.0) | 1101.0 (359.0; 1258.0) | 1265.5 (479.0; 1534.0) | 1382.0 (1128.0; 1556.0) | 1382.0 (479.0; 1555.0) | 1133.0 (359.0; 1415.0) | 1382.0 (1101.0; 1556.0) | 1135.0 (479.0; 1441.0) |
| AT-based dose regimen | |||||||||
| Inhibitor (n = 8) | Noninhibitor (n = 10) | Overall (N = 18) | |||||||
| Hemophilia A (n = 6) | Hemophilia B (n = 2) | Total (n = 8) | Hemophilia A (n = 6) | Hemophilia B (n = 4) | Total (n = 10) | Hemophilia A (n = 12) | Hemophilia B (n = 6) | Total (n = 18) | |
| Mean age (SD), y | 35.2 (4.6) | 25.0 (2.8) | 32.6 (6.2) | 46.7 (17.9) | 35.8 (12.4) | 42.3 (16.2) | 40.9 (13.8) | 32.2 (11.2) | 38.0 (13.4) |
| Weight, mean (SD), kg | 85.8 (14.2) | 96.5 (4.6) | 88.5 (13.1) | 78.0 (13.2) | 79.4 (16.2) | 78.5 (13.6) | 81.9 (13.7) | 85.1 (15.5) | 82.9 (13.9) |
| Moderate (1%-5%), n (%) | 0 | 0 | 0 | 1 (16.7) | 2 (50.0) | 3 (30.0) | 1 (8.3) | 2 (33.3) | 3 (16.7) |
| Race, Caucasian/White, n (%) | 6 (100) | 2 (100) | 8 (100) | 6 (100) | 4 (100) | 10 (100) | 12 (100) | 6 (100) | 18 (100) |
| Region, n (%) | |||||||||
| North America | 0 | 1 (50.0) | 1 (12.5) | 1 (16.7) | 0 | 1 (10.0) | 1 (8.3) | 1 (16.7) | 2 (11.1) |
| Europe | 6 (100) | 1 (50.0) | 7 (87.5) | 5 (83.3) | 4 (100) | 9 (90.0) | 11 (91.7) | 5 (83.3) | 16 (88.9) |
| Severe FVIII or FIX (<1%), n (%) | 6 (100) | 2 (100) | 8 (100) | 5 (83.3) | 2 (50.0) | 7 (70.0) | 11 (91.7) | 4 (66.70) | 15 (83.3) |
| Historic ABR at parent study entry, median (Q1; Q3) | 34.0 (20.0; 48.0) | 40.0 (26.0; 54.0) | 37.0 (20.0; 51.0) | 1.0 (0.0; 24.0) | 4.0 (2.0; 5.0) | 3.0 (0.0; 6.0) | 20.0 (1.0; 43.0) | 5.0 (4.0; 26.0) | 18.0 (2.0; 38.0) |
| History of hepatitis C, n (%) | 6 (100) | 0 | 6 (75.0) | 5 (83.3) | 2 (50.0) | 7 (70.0) | 11 (91.7) | 2 (33.3) | 13 (72.2) |
| Exposure, median (Q1; Q3), d† | 546.5 (298.0; 615.0) | 471.0 (400.0; 542.0) | 525.0 (349.0; 600.0) | 574.5 (559.0; 594.0) | 570.5 (397.5; 596.0) | 570.5 (559.0; 594.0) | 574.5 (432.5; 603.5) | 555.0 (400.0; 573.0) | 568.0 (400.0; 594.0) |
| Original dose regimen . | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| . | Inhibitor (n = 15) . | Noninhibitor (n = 19) . | Overall (N = 34) . | ||||||
| Hemophilia A (n = 13) . | Hemophilia B (n = 2) . | Total (n = 15) . | Hemophilia A (n = 14) . | Hemophilia B (n = 5) . | Total (n = 19) . | Hemophilia A (n = 27) . | Hemophilia B (n = 7) . | Total (n = 34) . | |
| Mean age (SD), y | 34.0 (6.9) | 25.0 (2.8) | 32.8 (7.2) | 40.6 (12.8) | 36.6 (10.9) | 39.6 (12.2) | 37.4 (10.8) | 33.3 (10.6) | 36.6 (10.7) |
| Weight, mean (SD), kg | 74.9 (15.1) | 85.7 (6.5) | 76.3 (14.6) | 77.1 (10.0) | 74.1 (14.2) | 76.4 (10.9) | 76.1 (12.5) | 77.4 (13.2) | 76.3 (12.4) |
| Race, n (%) | |||||||||
| Caucasian/White | 13 (100) | 2 (100) | 15 (100) | 13 (92.9) | 5 (100) | 18 (94.7) | 26 (96.3) | 7 (100) | 33 (97.1) |
| Asian/Oriental | 0 | 0 | 0 | 1 (7.1) | 0 | 1 (5.3) | 1 (3.7) | 0 | 1 (2.9) |
| Region, n (%) | |||||||||
| North America | 0 | 1 (50.0) | 1 (6.7) | 1 (7.1) | 0 | 1 (5.3) | 1 (3.7) | 1 (14.3) | 2 (5.9) |
| Europe | 13 (100) | 1 (50.0) | 14 (93.3) | 13 (92.9) | 5 (100) | 18 (94.7) | 26 (96.3) | 6 (85.7) | 32 (94.1) |
| Moderate FVIII or FIX (1%-5%), n (%) | 0 | 0 | 0 | 1 (7.1) | 2 (40.0) | 3 (15.8) | 1 (3.7) | 2 (28.6) | 3 (8.8) |
| Severe FVIII or FIX (<1%), n (%) | 13 (100) | 2 (100) | 15 (100) | 13 (92.9) | 3 (60.0) | 16 (84.2) | 26 (96.3) | 5 (71.4) | 31 (91.2) |
| Historic ABR at parent study entry, median (Q1; Q3) | 40.0 (20.0; 48.0) | 40.0 (26.0; 54.0) | 40.0 (20.0; 48.0) | 7.0 (0.0; 24.0) | 4.0 (4.0; 6.0) | 4.0 (0.0; 22.0) | 20.0 (4.0; 40.0) | 6.0 (4.0; 26.0) | 20.0 (4.0; 38.0) |
| History of hepatitis C, n (%) | 11 (84.6) | 0 | 11 (73.3) | 12 (85.7) | 3 (60.0) | 15 (78.9) | 23 (85.2) | 3 (42.9) | 26 (76.5) |
| Exposure, median (Q1; Q3), d∗ | 1054.0 (359.0; 1138.0) | 1271.0 (1101.0; 1441.0) | 1101.0 (359.0; 1258.0) | 1265.5 (479.0; 1534.0) | 1382.0 (1128.0; 1556.0) | 1382.0 (479.0; 1555.0) | 1133.0 (359.0; 1415.0) | 1382.0 (1101.0; 1556.0) | 1135.0 (479.0; 1441.0) |
| AT-based dose regimen | |||||||||
| Inhibitor (n = 8) | Noninhibitor (n = 10) | Overall (N = 18) | |||||||
| Hemophilia A (n = 6) | Hemophilia B (n = 2) | Total (n = 8) | Hemophilia A (n = 6) | Hemophilia B (n = 4) | Total (n = 10) | Hemophilia A (n = 12) | Hemophilia B (n = 6) | Total (n = 18) | |
| Mean age (SD), y | 35.2 (4.6) | 25.0 (2.8) | 32.6 (6.2) | 46.7 (17.9) | 35.8 (12.4) | 42.3 (16.2) | 40.9 (13.8) | 32.2 (11.2) | 38.0 (13.4) |
| Weight, mean (SD), kg | 85.8 (14.2) | 96.5 (4.6) | 88.5 (13.1) | 78.0 (13.2) | 79.4 (16.2) | 78.5 (13.6) | 81.9 (13.7) | 85.1 (15.5) | 82.9 (13.9) |
| Moderate (1%-5%), n (%) | 0 | 0 | 0 | 1 (16.7) | 2 (50.0) | 3 (30.0) | 1 (8.3) | 2 (33.3) | 3 (16.7) |
| Race, Caucasian/White, n (%) | 6 (100) | 2 (100) | 8 (100) | 6 (100) | 4 (100) | 10 (100) | 12 (100) | 6 (100) | 18 (100) |
| Region, n (%) | |||||||||
| North America | 0 | 1 (50.0) | 1 (12.5) | 1 (16.7) | 0 | 1 (10.0) | 1 (8.3) | 1 (16.7) | 2 (11.1) |
| Europe | 6 (100) | 1 (50.0) | 7 (87.5) | 5 (83.3) | 4 (100) | 9 (90.0) | 11 (91.7) | 5 (83.3) | 16 (88.9) |
| Severe FVIII or FIX (<1%), n (%) | 6 (100) | 2 (100) | 8 (100) | 5 (83.3) | 2 (50.0) | 7 (70.0) | 11 (91.7) | 4 (66.70) | 15 (83.3) |
| Historic ABR at parent study entry, median (Q1; Q3) | 34.0 (20.0; 48.0) | 40.0 (26.0; 54.0) | 37.0 (20.0; 51.0) | 1.0 (0.0; 24.0) | 4.0 (2.0; 5.0) | 3.0 (0.0; 6.0) | 20.0 (1.0; 43.0) | 5.0 (4.0; 26.0) | 18.0 (2.0; 38.0) |
| History of hepatitis C, n (%) | 6 (100) | 0 | 6 (75.0) | 5 (83.3) | 2 (50.0) | 7 (70.0) | 11 (91.7) | 2 (33.3) | 13 (72.2) |
| Exposure, median (Q1; Q3), d† | 546.5 (298.0; 615.0) | 471.0 (400.0; 542.0) | 525.0 (349.0; 600.0) | 574.5 (559.0; 594.0) | 570.5 (397.5; 596.0) | 570.5 (559.0; 594.0) | 574.5 (432.5; 603.5) | 555.0 (400.0; 573.0) | 568.0 (400.0; 594.0) |
If Q2M regimen was administrated for the last recommended dose, duration of treatment exposure in days = (date of last dose of study drug under the AT-based dose regimen – date of first dose of study drug under the AT-based dose regimen) + 56. If QM regimen was administrated for the last recommended dose, duration of treatment exposure (days) = (date of last dose of study drug under the AT-based dose regimen – date of first dose of study drug under the AT-based dose regimen) + 28.
Q1, quartile 1; Q3, quartile 3; QM, once monthly; Q2M, every second month.
Duration of treatment exposure (days) = (date of last dose of study drug under the original dose regimen – date of first dose of study drug in LTE14762 study) + 28, excluding the 2 dose suspensions in LTE14762.
Participants exposed to ≥1 doses under the AT-based dose regimen were summarized.