Table 2.
Baseline demographics and patient characteristics
| Characteristics | 10 IU/kg | 40 IU/kg | On demand | All |
|---|---|---|---|---|
| Number of patients | 30 | 29 | 15 | 74 |
| Age, years | ||||
| Mean (SD) | 32.4 (13.9) | 30.0 (15.8) | 32.4 (12.0) | 31.4 (14.2) |
| Weight, kg | ||||
| Mean (SD) | 75.6 (13.0) | 70.4 (15.1) | 76.1 (16.6) | 73.7 (14.7) |
| Race, n (%) | ||||
| White | 16 (53.3) | 21 (72.4) | 11 (73.3) | 48 (64.9) |
| Asian | 8 (26.7) | 5 (17.2) | 3 (20.0) | 16 (21.6) |
| Black or African American | 2 (6.7) | 3 (10.3) | — | 5 (6.8) |
| Other | 4 (13.3) | — | 1 (6.7) | 5 (6.8) |
| Previous treatment regimen,* n (%) | ||||
| Prophylaxis | 20 (66.7) | 17 (58.6) | 2 (13.3) | 39 (52.7) |
| On demand | 10 (33.3) | 12 (41.4) | 13 (86.7) | 35 (47.3) |
| Previous prophylaxis patients, n (%) | ||||
| Recombinant FIX | 11 (55.0) | 10 (58.8) | — | 21 (53.8) |
| Plasma FIX product | 9 (45.0) | 7 (41.2) | 2 (100.0) | 18 (46.2) |
| Classification of hemophilia,† n (%) | ||||
| Moderate (1-2 IU/dL) | 7 (23.3) | 5 (17.2) | 2 (13.3) | 14 (18.9) |
| Severe (<1 IU/dL) | 23 (76.7) | 24 (82.8) | 13 (86.7) | 60 (81.1) |
| F9 genotype,‡ n (%) | ||||
| N | 27 (100.0) | 24 (100.0) | 15 (100.0) | 66 (100.0) |
| Null mutations | 7 (25.9) | 1 (4.2) | 5 (33.3) | 13 (19.7) |
| Nonnull mutations | 20 (74.1) | 23 (95.8) | 10 (66.7) | 53 (80.3) |
| Arthropathy at baseline,¶ n (%) | ||||
| Yes | 20 (66.7) | 18 (62.1) | 10 (66.7) | 48 (64.9) |
| No | 10 (33.3) | 11 (37.9) | 5 (33.3) | 26 (35.1) |
| Target joints at baseline,§ n (%) | ||||
| Yes | 13 (43.3) | 15 (51.7) | 12 (80.0) | 40 (54.1) |
| No | 17 (56.7) | 14 (48.3) | 3 (20.0) | 34 (45.9) |
FIX, coagulation factor IX; SD, standard deviation.
Two patients receiving previous prophylaxis chose to enroll into the on-demand treatment group. These 2 patients entered the trial at a time at which the prophylaxis groups were not yet opened for enrollment because of regulatory authority requirements in the patients’ country of origin.
Classification as defined in medical records. Only patients with FIX activity 2 IU/dL or less were eligible.
Null mutation genotypes included large deletions/insertions, inversions, and nonsense mutations. Nonnull mutations included small deletions/insertions, splice site and missense mutations, and substitutions.13 Mutations in the F9 gene were determined by either laboratory analysis carried out in the trial or, alternatively, by post hoc classification of gene defects reported in patients’ medical records where possible.
The presence and extent of arthropathy in joints at baseline was recorded at the discretion of the investigator and was based on descriptions by the patients.
A target joint was defined as 3 or more bleeding episodes in a particular joint within a period of 6 months before trial.