Baseline characteristics
. | Data . |
---|---|
No. patients | 366 |
Baseline FVIII activity, no. (%) | |
Less than 0.01 IU/mL | 332 (91) |
0.01 to 0.02 IU/mL | 34 (9) |
Ethnicity, no.(%) | |
White | 327 (90) |
African | 8 (2) |
Asian | 15 (4) |
Other | 15 (4) |
Family history hemophilia, no. (%) | |
Positive | 168 (47) |
Negative | 191 (53) |
Family history inhibitors, no. (%) | |
Positive | 24 (15) |
Negative | 138 (85) |
FVIII gene mutation type, no. (%) | |
Large deletions | 10 (3) |
Nonsense mutations | 30 (10) |
Inversions | 172 (55) |
Small deletions/insertions | 41 (13) |
Missense mutations | 49 (16) |
Other | 10 (3) |
Breastfeeding, no. (%) | |
Yes | 91 (69) |
No | 41 (31) |
Median age at first exposure day, mo (IQR) | 11 (6-15) |
Median age at 50th exposure day, mo* (IQR) | 31 (21-46) |
Duration first and 50th exposure day, mo* (IQR) | 19 (12-32) |
All peak treatment moments, no. (%) | |
Yes | 251 (78) |
No | 71 (22) |
Major peak treatment moments, no. (%) | |
Yes | 176 (55) |
No | 146 (45) |
Surgery during first 50 exposure d, no. (%) | |
Yes | 80 (25) |
No | 242 (75) |
Treatment regimen, no. (%) | |
Prophylaxis | 196 (55) |
On demand | 162 (45) |
Median age at start of prophylaxis, mo (IQR) | 20 (14-35) |
Median no. exposure d at start of prophylaxis (IQR) | 16 (8-28) |
Reason for first exposure to factor VIII, no. (%) | |
Bleed | 286 (84) |
Prophylaxis | 37 (11) |
Surgery | 17 (5) |
. | Data . |
---|---|
No. patients | 366 |
Baseline FVIII activity, no. (%) | |
Less than 0.01 IU/mL | 332 (91) |
0.01 to 0.02 IU/mL | 34 (9) |
Ethnicity, no.(%) | |
White | 327 (90) |
African | 8 (2) |
Asian | 15 (4) |
Other | 15 (4) |
Family history hemophilia, no. (%) | |
Positive | 168 (47) |
Negative | 191 (53) |
Family history inhibitors, no. (%) | |
Positive | 24 (15) |
Negative | 138 (85) |
FVIII gene mutation type, no. (%) | |
Large deletions | 10 (3) |
Nonsense mutations | 30 (10) |
Inversions | 172 (55) |
Small deletions/insertions | 41 (13) |
Missense mutations | 49 (16) |
Other | 10 (3) |
Breastfeeding, no. (%) | |
Yes | 91 (69) |
No | 41 (31) |
Median age at first exposure day, mo (IQR) | 11 (6-15) |
Median age at 50th exposure day, mo* (IQR) | 31 (21-46) |
Duration first and 50th exposure day, mo* (IQR) | 19 (12-32) |
All peak treatment moments, no. (%) | |
Yes | 251 (78) |
No | 71 (22) |
Major peak treatment moments, no. (%) | |
Yes | 176 (55) |
No | 146 (45) |
Surgery during first 50 exposure d, no. (%) | |
Yes | 80 (25) |
No | 242 (75) |
Treatment regimen, no. (%) | |
Prophylaxis | 196 (55) |
On demand | 162 (45) |
Median age at start of prophylaxis, mo (IQR) | 20 (14-35) |
Median no. exposure d at start of prophylaxis (IQR) | 16 (8-28) |
Reason for first exposure to factor VIII, no. (%) | |
Bleed | 286 (84) |
Prophylaxis | 37 (11) |
Surgery | 17 (5) |
*In noninhibitor patients.