n dose was 36 IU/kg at approximately 28 hour intervals. The number of doses andduration of treatment varied by procedure.
Routine Prophylaxis in Adolescents/Adults
The efficacy of ESPEROCT in routine prophylaxis with Q4D dosing was demonstrated for the adult/adolescent population(see Table 7). In the extension part of the study, treatment success of the Q7D arm was not established. During the MainPhase of the adolescent/adult trial, 186 subjects had a total of 159 exposure years. The median annualized bleeding rate(ABR) for treated bleeds in adults and adolescents treated every 4 days was 1.2 (IQR: 0.0:4.3), and mean ABR was 3.0 (SD: 4.7). When including all bleeds (treated and non-treated), the median ABR was 1.2 (IQR: 0.0; 4.7) and the mean ABR was
3.3 (SD: 4.9).
Table 7: Efficacy in adolescent/adult prophylaxis, median and mean ABRs by age, treatment regimen, and bleed type
Prophylaxis On-demand
Age Range 12–17 years 18–70 years 12–70 years 18–70 years
# of subjects 25 150 175 12
Mean treatment duration
(years) 0.85 0.81 0.82 1.33
Treated bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median ABR (IQR)
Mean ABR (SD)
19 (76)
6 (24)
67
2.2 (0.9;4.7)
3.5 (3.9)
86 (57)
64 (43)
369
1.2 (0.0;3.7)
2.9 (4.8)
105 (60)
70 (40)
436
1.2 (0.0;4.3)
3.0 (4.7)
12 (100)
0
532
30.9 (18.6;38.5)
31.9 (19.1)
All bleeds (treated and
untreated)
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds*
Median ABR (IQR)
Mean ABR (SD)
19 (76)
6 (24)
72
2.2 (0.9;6.0)
3.7 (4.1)
88 (59)
62 (41)
386
1.2 (0.0;4.3)
3.2 (5.1)
107 (61)
68 (39)
458
1.2 (0.0;4.7)
3.3 (4.9)
12 (100)
0
536
31.3 (18.6;38.9)
32.2 (19.1)
Treated spontaneous bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AsBR (IQR)
Mean AsBR (SD)
11 (44)
14 (56)
30
0.0 (0.0;1.5)
1.4 (2.4)
65 (43)
85 (57)
221
0.0 (0.0;1.9)
1.8 (3.7)
76 (43)
99 (57)
251
0.0 (0.0;1.8)
1.7 (3.5)
12 (100)
0
415
19.4 (12.1;31.0)
24.5 (17.3)
Treated traumatic bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AtBR (IQR)
Mean AtBR (SD)
16 (64)
9 (36)
37
1.3 (0.0;2.6)
2.1 (2.9)
57 (38)
93 (62)
146
0.0 (0.0;1.4)
1.1 (2.2)
73 (42)
102 (58)
183
0.0 (0.0;1.7)
1.2 (2.3)
10 (83)
2 (17)
110
4.3 (0.8;9.9)
6.1 (6.2)
Treated joint bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AjBR (IQR)
Mean AjBR (SD)
16 (64)
9 (36)
37
1.2 (0.0;2.8)
1.8 (2.2)
74 (49)
76 (51)
288
0.0(0.0;2.8)
2.3 (4.3)
90 (51)
85 (49)
325
0.9 (0.0;2.8)
2.2 (4.1)
12 (100)
0
309
19.4 (4.5;28.8)
19.7 (15.1)
ABR = annualized bleed rate; IQR = interquartile range, 25th percentile to 75th percentile; SD = standard deviation; AsBR = annualized spontaneous bleed
rate; AtBR = annualized traumatic bleed rate; AjBR = annualized joint bleed rate.
*Reflects all bleeds reported by patients including those where no ESPEROCT was administeredRoutine Prophylaxis in Children <12 Years of AgeOverall, 68 children below 12 years received prophylactic treatment with ESPEROCT at an average dose of approximately65 IU/kg twice weekly. The prophylactic effect of ESPEROCT was demonstrated with a median ABR rate of 2.0 (IQR: 0.0;2.8) and 2.0 (IQR: 0.0; 4.2) for treated bleeds and all bleeds r |
