Patient and treatment characteristics according to used factor VIII product type at first treatment
| . | Recombinant factor VIII product . | Plasma-derived factor VIII product . | Total . | |
|---|---|---|---|---|
| Low VWF content* . | High VWF content* . | |||
| No. of patients | 181 | 33 | 102 | 316 |
| No. of inhibitors (%) | 53 (29) | 5 (15) | 24 (24) | 82 (26) |
| No. of high-titer inhibitors (%) | 43 (24) | 4 (12) | 19 (19) | 66 (21) |
| No. of switched product type (%) | 5 (3) | 14 (42) | 35 (34) | 54 (17) |
| Median date of birth (IQR) | Jul 1996 (Dec 1994-Apr 1998) | Jan 1992 (Dec 1990-Jun 1993) | Mar 1993 (Feb 1991-Dec 1995) | Jun 1995 (Aug 1992-May 1997) |
| Baseline factor VIII activity level less than 0.01 IU/mL, no. (%) | 165 (91) | 32 (97) | 91 (89) | 288 (91) |
| White, no. (%) | 163 (90) | 29 (88) | 89 (87) | 281 (89) |
| Positive family history of hemophilia, no. (%) | 78 (43) | 18 (55) | 45 (46) | 141 (45) |
| Positive family history of inhibitors, no. (%) | 14 (19) | 3 (19) | 5 (11) | 22 (16) |
| High-risk mutation, no. (%) | 107 (66) | 19 (66) | 57 (70) | 183 (68) |
| Median age at first exposure, mo (IQR) | 11 (7-14) | 10 (5-15) | 10 (6-15) | 11 (6-15) |
| Start regular prophylaxis within first 50 exposure days, no. (%) | 105 (58) | 23 (70) | 44 (43) | 172 (54) |
| Median age at start of prophylaxis, mo (IQR) | 21 (14-35) | 20 (15-32) | 18 (12-35) | 20 (14-35) |
| Median cumulative no. of EDs at start of prophylaxis (IQR) | 15 (6-28) | 17 (10-26) | 15 (7-29) | 15 (7-27) |
| Peak treatment moment present, no. (%)† | 129 (71) | 23 (70) | 93 (91) | 245 (78) |
| Surgical procedure, no. (%) | 41 (23) | 8 (24) | 27 (26) | 76 (24) |
| . | Recombinant factor VIII product . | Plasma-derived factor VIII product . | Total . | |
|---|---|---|---|---|
| Low VWF content* . | High VWF content* . | |||
| No. of patients | 181 | 33 | 102 | 316 |
| No. of inhibitors (%) | 53 (29) | 5 (15) | 24 (24) | 82 (26) |
| No. of high-titer inhibitors (%) | 43 (24) | 4 (12) | 19 (19) | 66 (21) |
| No. of switched product type (%) | 5 (3) | 14 (42) | 35 (34) | 54 (17) |
| Median date of birth (IQR) | Jul 1996 (Dec 1994-Apr 1998) | Jan 1992 (Dec 1990-Jun 1993) | Mar 1993 (Feb 1991-Dec 1995) | Jun 1995 (Aug 1992-May 1997) |
| Baseline factor VIII activity level less than 0.01 IU/mL, no. (%) | 165 (91) | 32 (97) | 91 (89) | 288 (91) |
| White, no. (%) | 163 (90) | 29 (88) | 89 (87) | 281 (89) |
| Positive family history of hemophilia, no. (%) | 78 (43) | 18 (55) | 45 (46) | 141 (45) |
| Positive family history of inhibitors, no. (%) | 14 (19) | 3 (19) | 5 (11) | 22 (16) |
| High-risk mutation, no. (%) | 107 (66) | 19 (66) | 57 (70) | 183 (68) |
| Median age at first exposure, mo (IQR) | 11 (7-14) | 10 (5-15) | 10 (6-15) | 11 (6-15) |
| Start regular prophylaxis within first 50 exposure days, no. (%) | 105 (58) | 23 (70) | 44 (43) | 172 (54) |
| Median age at start of prophylaxis, mo (IQR) | 21 (14-35) | 20 (15-32) | 18 (12-35) | 20 (14-35) |
| Median cumulative no. of EDs at start of prophylaxis (IQR) | 15 (6-28) | 17 (10-26) | 15 (7-29) | 15 (7-27) |
| Peak treatment moment present, no. (%)† | 129 (71) | 23 (70) | 93 (91) | 245 (78) |
| Surgical procedure, no. (%) | 41 (23) | 8 (24) | 27 (26) | 76 (24) |
VWF indicates von Willebrand factor antigen; ED, exposure day.
Low VWF content was defined as less than 0.01 IU VWF antigen per IU factor VIII antigen; high VWF content was defined as more than 0.01 IU VWF antigen per IU factor VIII antigen.
Peak treatment moment was defined as an episode of treatment with factor VIII for a bleed or surgery on at least 3 consecutive days.