Treatment characteristics of included patients with detailed data (N = 576)
| Characteristics . | No. (%) or median (IQR) . | Range . |
|---|---|---|
| Age at first exposure (mo.) | 9.8 (5.3-13.4) | 0-55.8 |
| Age at 75th exposure day (mo.)* | 26.0 (19.5-34.5) | 2.9-115.0 |
| Duration between 1st and 75th exposure day (mo.)* | 15.6 (10.2-23.8) | 2.6-84.8 |
| Intensity of treatment | ||
| Treatment characteristics at first treatment | ||
| Reason of first exposure to FVIII | ||
| Hemorrhage, including head trauma | 488 (84.7) | NA |
| Prophylaxis | 68 (11.8) | NA |
| Surgery | 20 (3.5) | NA |
| Peak treatment moment at first exposure to FVIII | ||
| Peak treatment moment at least 3 days | 149 (25.9) | NA |
| Peak treatment moment at least 5 days | 98 (17) | NA |
| Peak treatment moment at least 10 days | 40 (6.9) | NA |
| Major surgery at first exposure to FVIII | 13 (2.3) | NA |
| Treatment characteristics at subsequent exposure days | ||
| Peak treatment moment during first 75 exposure days | ||
| Peak treatment moment at least 3 days | 386 (67.0) | NA |
| Peak treatment moment at least 5 days | 277 (48.1) | NA |
| Peak treatment moment at least 10 days | 93 (16.1) | NA |
| Ever had a major surgery during first 75 exposure days | 144 (25) | NA |
| Prophylaxis | ||
| Regular prophylaxis† | ||
| No. patients who started regular prophylaxis | 412 (71.5) | NA |
| Age start regular prophylaxis (mo.) | 16.7 (12.4-24.4) | 0.95-96.9 |
| No. exposure days at start prophylaxis | 15 (7-25) | 2-74 |
| Dose of FVIII at start of prophylaxis (IU/kg) | 44.6 (32.3-50.0) | 18.9-235.3 |
| Frequency of FVIII at start of prophylaxis, no. infusions per week | 1 (1-2) | 1-7 |
| Start of regular prophylaxis | ||
| No. patients who started prophylaxis <15 exposure days | 208 (50.5) | NA |
| No. patients who started prophylaxis <5 exposure days | 79 (19.2) | NA |
| Regular prophylaxis at least twice a week | ||
| No. patients who started regular prophylaxis at least twice a week | 316 (54.9) | NA |
| Age start regular prophylaxis at least twice a week (mo.) | 18.7 (12.9-27.2) | 1.0-96.9 |
| No. exposure days at start prophylaxis at least twice a week | 22 (14-40) | 2-74 |
| Intention to start prophylaxis‡ | ||
| No. patients who had an intention to start prophylaxis | 470 (81.6) | NA |
| Age at intention to start prophylaxis (mo.) | 14.6 (10.2-21.4) | 0-96.5 |
| No. exposure days at intention to start prophylaxis | 5 (2-12) | 0-74 |
| Characteristics . | No. (%) or median (IQR) . | Range . |
|---|---|---|
| Age at first exposure (mo.) | 9.8 (5.3-13.4) | 0-55.8 |
| Age at 75th exposure day (mo.)* | 26.0 (19.5-34.5) | 2.9-115.0 |
| Duration between 1st and 75th exposure day (mo.)* | 15.6 (10.2-23.8) | 2.6-84.8 |
| Intensity of treatment | ||
| Treatment characteristics at first treatment | ||
| Reason of first exposure to FVIII | ||
| Hemorrhage, including head trauma | 488 (84.7) | NA |
| Prophylaxis | 68 (11.8) | NA |
| Surgery | 20 (3.5) | NA |
| Peak treatment moment at first exposure to FVIII | ||
| Peak treatment moment at least 3 days | 149 (25.9) | NA |
| Peak treatment moment at least 5 days | 98 (17) | NA |
| Peak treatment moment at least 10 days | 40 (6.9) | NA |
| Major surgery at first exposure to FVIII | 13 (2.3) | NA |
| Treatment characteristics at subsequent exposure days | ||
| Peak treatment moment during first 75 exposure days | ||
| Peak treatment moment at least 3 days | 386 (67.0) | NA |
| Peak treatment moment at least 5 days | 277 (48.1) | NA |
| Peak treatment moment at least 10 days | 93 (16.1) | NA |
| Ever had a major surgery during first 75 exposure days | 144 (25) | NA |
| Prophylaxis | ||
| Regular prophylaxis† | ||
| No. patients who started regular prophylaxis | 412 (71.5) | NA |
| Age start regular prophylaxis (mo.) | 16.7 (12.4-24.4) | 0.95-96.9 |
| No. exposure days at start prophylaxis | 15 (7-25) | 2-74 |
| Dose of FVIII at start of prophylaxis (IU/kg) | 44.6 (32.3-50.0) | 18.9-235.3 |
| Frequency of FVIII at start of prophylaxis, no. infusions per week | 1 (1-2) | 1-7 |
| Start of regular prophylaxis | ||
| No. patients who started prophylaxis <15 exposure days | 208 (50.5) | NA |
| No. patients who started prophylaxis <5 exposure days | 79 (19.2) | NA |
| Regular prophylaxis at least twice a week | ||
| No. patients who started regular prophylaxis at least twice a week | 316 (54.9) | NA |
| Age start regular prophylaxis at least twice a week (mo.) | 18.7 (12.9-27.2) | 1.0-96.9 |
| No. exposure days at start prophylaxis at least twice a week | 22 (14-40) | 2-74 |
| Intention to start prophylaxis‡ | ||
| No. patients who had an intention to start prophylaxis | 470 (81.6) | NA |
| Age at intention to start prophylaxis (mo.) | 14.6 (10.2-21.4) | 0-96.5 |
| No. exposure days at intention to start prophylaxis | 5 (2-12) | 0-74 |
NA, not applicable.
In noninhibitor patients who reached the study end point and had available data on exposure days (n = 340).
The start of regular prophylaxis was defined as the moment on which at least 3 consecutive prophylactic FVIII exposure days are given for a period of at least 2 weeks.
The intention to start prophylaxis was defined as the moment on which at least 1 prophylactic infusion was given or at the time of any surgical procedures to facilitate venous access (implantation of a central venous access device or creation of an arteriovenous fistula).