153/241 (63.5%)

79/150* (52.7%)

209/283* (73.9%)

943/1202* (78.5%)

Trauma: n/total (%)

79/241 (32.8%)

70/150* (46.7%)

74/283* (26.1%)

258/1202* (21.5%)

Joint bleeds: n/total (%)

191/241 (79.3%)

120/154 (77.9%)

255/293 (87.0%)

924/1197* (77.2%)

Mild/moderate: n/total (%)

215/241 (89.2%)

130/153* (84.9%)

260/293 (88.8%)

1092/1196* (91.3%)

% of bleeds treated with ≤2 infusions

87.0%

96.2%

95.3%

Response to treatment of bleeds assessed as “Excellent” or “Good”: n/total† (%)

190/235 (80.9%)

107/149 (71.8%)

172/279 (61.6%)

834/1196 (69.7%)

Median dose per infusion (range)

31.6 IU/kg
(14-67 IU/kg)

29.4 IU/kg
(19-49 IU/kg)

22.0 IU/kg
(11-35 IU/kg)
Children 12 Years of Age and Younger
A total of 97 bleeding episodes in 28 pediatric subjects were treated with KOVALTRY. Majority (96.9%) of the bleeds were mild to moderate in severity. Fifty-nine (72.8%) bleeds were trauma related. During the 6 month treatment period, the median dose of KOVALTRY for the treatment of breakthrough bleeds was 36.94 IU/kg per infusion (range 20.8–71.6 IU/kg).
Assessment of response to treatment of bleeds was as follows:
Excellent: Abrupt pain relief and/or improvement in signs of bleeding with no additional infusion administered; Good: Definite pain relief and/or improvement in signs of bleeding but possibly requiring more than one infusion for complete resolution; Moderate: Probable or slight improvement in signs of bleeding with at least one additional infusion for complete resolution; Poor: No improvement at all between infusions or condition worsens.
The hemostatic efficacy in on-demand treatment of bleeds was assessed as either “good” or “excellent” in 90.1% of cases (97.8% in the younger age group and 81.0% in the older age group). Majority of bleeds (89.7%) were successfully treated with ≤2 infusions. Response to treatment was similar for children aged 0 to <6 compared to 6 to 12 years of age (see TABLE 10).
Table 10: On-demand Treatment and Control of Bleeding Episodes in Children Treated with KOVALTRY
Characteristics of Bleeding Episodes Study 3
PTPs 0 to <6 yrs
(N=25) PTPs 6 to 12 yrs
(N=26) PTPs 0 to 12 yrs
(N=51)
* Total number of treated bleeds † The % is calculated from number of treated bleeds assessed for response
Total number of bleeds

52

45

97

Spontaneous: n/total (%)

8/44* (18.2%)

12/37* (32.4%)

20/81* (24.7%)

Trauma: n/total (%)

36/44* (81.8%)

23/37* (62.2%)

59/81* (72.8%)

Joint bleeds: n/total (%)

10/52 (19.2%)

22/45 (48.9%)

32/97 (33.0%)

Mild/moderate: n/total (%)

50/52 (96.2%)

44/45 (97.8%)

94/97 (96.9%)

% bleeds treated with ≤2 infusions

92.4%

86.7%

89.7%

Response to treatment of bleeds assessed as “Excellent” or “Good”: n/total† (%)

43/44 (97.8%)

30/37 (81.0%)

73/81 (90.1%)

Median dose per infusion (range)

38.7 IU/kg
(20.8–71.6 IU/kg)

32.4 IU/kg
(21.7–50.0 IU/kg)

36.9 IU/kg
(20.8–71.6 IU/kg)
14.2 Perioperative Management
A total of 14 major and 46 mino