Baseline demographics and patient characteristics
| Characteristics . | Prophylaxis (N = 40) . | On-demand (N = 23) . | Overall (N = 63) . |
|---|---|---|---|
| Age, y | |||
| Mean (SD) | 31.6 (15.2) | 35.3 (11.1) | 33.0 (13.9) |
| Adolescents (12-17 y), n | 7 | 0 | 7 |
| Race, n (%) | |||
| White | 33 (82.5) | 19 (82.6) | 52 (82.5) |
| Asian | 6 (15.0) | 4 (17.4) | 10 (15.9) |
| Black/African American | 1 (2.5) | 0 | 1 (1.6) |
| Weight, kg | |||
| Mean (SD) | 69.6 (14.4) | 75.1 (20.7) | 71.6 (17.0) |
| Geographic location, n (%) | |||
| Asia | 6 (15.0) | 4 (17.4) | 10 (15.9) |
| Europe | 21 (52.5) | 15 (65.2) | 36 (57.1) |
| Middle East | 11 (27.5) | 0 | 11 (17.5) |
| North America | 2 (5.0) | 4 (17.4) | 6 (9.5) |
| Previous treatment regimen | |||
| On-demand | 0 | 23 (100.0) | 23 (36.5) |
| Prophylaxis | 40 (100.0) | 0 | 40 (63.5) |
| Previous FIX product | |||
| rFIX | 20 (50.0) | 7 (30.4)* | 27 (42.8) |
| pdFIX | 9 (22.5) | 12 (52.2) | 21 (33.3) |
| rIX-FP† | 11 (27.5) | 4 (17.4) | 15 (23.8) |
| Classification of hemophilia‡, n(%) | |||
| ≤1 IU/dL | 35 (87.5) | 20 (87.0) | |
| ≤2 IU/dL | 40 (100.0) | 23 (100.0) | 63 (100.0) |
| HIV positive | 7 (17.5) | 5 (21.7) | 12 (19.0) |
| HBV positive | 1 (2.5) | 1 (4.3) | 2 (3.2) |
| HCV positive | 10 (25.0) | 13 (56.5) | 23 (36.5) |
| Chronic hemarthrosis/target joint§, n (%) | 21 (52.5) | 14 (60.9) | 35 (55.6) |
| Total ABR|| | |||
| Mean (SD) | 3.4 (3.8) | 24.3 (7.3) | |
| Median (IQR) | 2.0 (0, 4.5) | 23.5 (22, 28) | |
| Spontaneous ABR|| | |||
| Mean (SD) | 1.5 (2.2) | 16.6 (8.6) | |
| Median (IQR) | 0 (0, 2) | 17.0 (10, 24) | |
| Nontrauma ABR||¶ | |||
| Mean (SD) | 1.7 (2.5) | 18.3 (6.7) | |
| Median (IQR) | 1.0 (0, 2) | 21.0 (12, 24) |
| Characteristics . | Prophylaxis (N = 40) . | On-demand (N = 23) . | Overall (N = 63) . |
|---|---|---|---|
| Age, y | |||
| Mean (SD) | 31.6 (15.2) | 35.3 (11.1) | 33.0 (13.9) |
| Adolescents (12-17 y), n | 7 | 0 | 7 |
| Race, n (%) | |||
| White | 33 (82.5) | 19 (82.6) | 52 (82.5) |
| Asian | 6 (15.0) | 4 (17.4) | 10 (15.9) |
| Black/African American | 1 (2.5) | 0 | 1 (1.6) |
| Weight, kg | |||
| Mean (SD) | 69.6 (14.4) | 75.1 (20.7) | 71.6 (17.0) |
| Geographic location, n (%) | |||
| Asia | 6 (15.0) | 4 (17.4) | 10 (15.9) |
| Europe | 21 (52.5) | 15 (65.2) | 36 (57.1) |
| Middle East | 11 (27.5) | 0 | 11 (17.5) |
| North America | 2 (5.0) | 4 (17.4) | 6 (9.5) |
| Previous treatment regimen | |||
| On-demand | 0 | 23 (100.0) | 23 (36.5) |
| Prophylaxis | 40 (100.0) | 0 | 40 (63.5) |
| Previous FIX product | |||
| rFIX | 20 (50.0) | 7 (30.4)* | 27 (42.8) |
| pdFIX | 9 (22.5) | 12 (52.2) | 21 (33.3) |
| rIX-FP† | 11 (27.5) | 4 (17.4) | 15 (23.8) |
| Classification of hemophilia‡, n(%) | |||
| ≤1 IU/dL | 35 (87.5) | 20 (87.0) | |
| ≤2 IU/dL | 40 (100.0) | 23 (100.0) | 63 (100.0) |
| HIV positive | 7 (17.5) | 5 (21.7) | 12 (19.0) |
| HBV positive | 1 (2.5) | 1 (4.3) | 2 (3.2) |
| HCV positive | 10 (25.0) | 13 (56.5) | 23 (36.5) |
| Chronic hemarthrosis/target joint§, n (%) | 21 (52.5) | 14 (60.9) | 35 (55.6) |
| Total ABR|| | |||
| Mean (SD) | 3.4 (3.8) | 24.3 (7.3) | |
| Median (IQR) | 2.0 (0, 4.5) | 23.5 (22, 28) | |
| Spontaneous ABR|| | |||
| Mean (SD) | 1.5 (2.2) | 16.6 (8.6) | |
| Median (IQR) | 0 (0, 2) | 17.0 (10, 24) | |
| Nontrauma ABR||¶ | |||
| Mean (SD) | 1.7 (2.5) | 18.3 (6.7) | |
| Median (IQR) | 1.0 (0, 2) | 21.0 (12, 24) |
HBV, hepatitis B virus; HCV, hepatitis C virus.
Includes 1 subject previously on an investigational rFIX product.
Patients previously enrolled in phase 2 study with rIX-FP.13
FIX activity level (IU/dL) as recorded in subject medical records or measured at screening. All subjects had FIX activity ≤2 IU/dL in order to be eligible for the study.
Percentages are based on the number of patients with chronic hemarthrosis in at least 1 major joint (ankle, knee, or elbow) or a target joint, defined as at least 3 spontaneous bleeding episodes in the same joint in a 6-month period.20
Bleeding episodes in the 12 months prior to study entry.
Nontrauma defined as spontaneous and unknown cause bleeding episodes.