Characteristics of the cohorts
. | FVIII-LFB; n = 62 . | Recombinant FVIII*; n = 86 . |
|---|---|---|
| Follow-up | ||
| Period | 1988-1999 | 1991†-2002 |
| Median time to follow-up, mo (range) | 48.4 (1.0-127.5) | 24.6 (0.3-84.4) |
| Mean per patient of interval between 2 inhibitor tests | ||
| Median CEDs (25th-75th percentile) | 5.4 (4.0-7.4) | 5.2 (3.5-7.4) |
| Patients with average interval no higher than 10 CEDs (%) | 51 (82) | 72 (84) |
| Median, mo (25th-75th percentile) | 4.4 (3.3-6.3) | 3.2 (2.4-4.5) |
| Exposure‡ | ||
| Median CEDs (range) | 108 (1-544) | 38 (3-655) |
| Patients with at least 50 CEDs (%) | 40 (65) | 36 (42) |
| Patients with at least 100 CEDs (%) | 32 (52) | 25 (29) |
| Genetic cofactors, no. (%)§ | ||
| High-risk mutations | 36 (58) | 44 (51) |
| Other mutations | 23 (37) | 35 (41) |
| Nonwhite | 7 (11) | 17 (20) |
| Family history of hemophilia and inhibitor | 9 (15) | 6 (7) |
| Family history of hemophilia without inhibitor | 23 (37) | 23 (27) |
| Environmental cofactors, no. (%)§ | ||
| Age at first infusion∥ | ||
| Younger than 6 months | 17 (27) | 18 (21) |
| 6 to 11 months | 21 (34) | 29 (34) |
| End point (%) | ||
| Patients with inhibitors, 0.6 BU or more | 7 (11) | 27 (31) |
| No. of high inhibitors, more than 5 BU | 3 (5) | 13 (15) |
| No. of high inhibitors, more than 5 BU and/or ITI | 4 (6) | 19 (22) |
. | FVIII-LFB; n = 62 . | Recombinant FVIII*; n = 86 . |
|---|---|---|
| Follow-up | ||
| Period | 1988-1999 | 1991†-2002 |
| Median time to follow-up, mo (range) | 48.4 (1.0-127.5) | 24.6 (0.3-84.4) |
| Mean per patient of interval between 2 inhibitor tests | ||
| Median CEDs (25th-75th percentile) | 5.4 (4.0-7.4) | 5.2 (3.5-7.4) |
| Patients with average interval no higher than 10 CEDs (%) | 51 (82) | 72 (84) |
| Median, mo (25th-75th percentile) | 4.4 (3.3-6.3) | 3.2 (2.4-4.5) |
| Exposure‡ | ||
| Median CEDs (range) | 108 (1-544) | 38 (3-655) |
| Patients with at least 50 CEDs (%) | 40 (65) | 36 (42) |
| Patients with at least 100 CEDs (%) | 32 (52) | 25 (29) |
| Genetic cofactors, no. (%)§ | ||
| High-risk mutations | 36 (58) | 44 (51) |
| Other mutations | 23 (37) | 35 (41) |
| Nonwhite | 7 (11) | 17 (20) |
| Family history of hemophilia and inhibitor | 9 (15) | 6 (7) |
| Family history of hemophilia without inhibitor | 23 (37) | 23 (27) |
| Environmental cofactors, no. (%)§ | ||
| Age at first infusion∥ | ||
| Younger than 6 months | 17 (27) | 18 (21) |
| 6 to 11 months | 21 (34) | 29 (34) |
| End point (%) | ||
| Patients with inhibitors, 0.6 BU or more | 7 (11) | 27 (31) |
| No. of high inhibitors, more than 5 BU | 3 (5) | 13 (15) |
| No. of high inhibitors, more than 5 BU and/or ITI | 4 (6) | 19 (22) |
Recombinate (n = 62) or Kogenate (n = 24)
Only 3 patients included in the Recombinate PUPs clinical trial received their first treatment in 1991; the others were treated as from mid-1993, when Recombinate became available in France
The observation period was defined as the number of CEDs until the discovery of the inhibitor or, for patients having no inhibitor, until the last inhibitor test after infusion of clotting factor of origin
There is no significant difference for genetic and environmental cofactors between the 2 cohorts: F8 genotype (P = .601), ethnicity (P = .167), family history of inhibitors (P = .073), or age at first infusion (P = .602)
Mean age at the first infusion is 10.9 months in FVIII-LFB cohort and 12.6 in recombinant FVIII cohort (P = .273)