Page 286 - Haematologica May 2020

J.A. Abrantes et al.
Table 1. Patients' characteristics, treatment and bleeding data by study for patients with plasma factor VIII (FVIII) observations enrolled in the bleeding observation period.
Study
Patients with PK and bleeding data, n (% of total)
Duration of bleeding observation period, months
Mean±SD
Median [range]
Individual FVIII dose during treatment period (IU/kg)
Mean±SD
Median [range]
Patients' characteristics
Age, years Mean±SD Median [range]
Weight, kg Mean±SD Median [range]
Lean body weight,a kg Mean±SD
Median [range]
Body mass index,a kg·m-2 Mean±SD
Median [range]
von Willebrand factor level,b % Mean±SD
Median [range]
Race, n (%) White Black Asian Hispanic Unknown
Treatment history, n (%) On-demand Prophylaxis
N. of target joints at study startc
Median [range]
Summary of bleeding episodes
Total number of bleeds, n (% of total) Patients with at least one bleed, n (% of total) Individual n. of bleeds
Median [range]
Time to first bleed,d days
Mean±SD
Median [range] Bleed type, n (%)
Spontaneous
Non-spontaneous
Spontaneous bleed location, n (%)
Joint
No joint
Bleed severity, n (%)
Mild Moderate Severe
N. of bleeds in the 12 months prior to study starte
Median [range]
LEOPOLD I
62 (36)
11.8 ± 1.53
12.0 [3.08-13.1]
38.4 ± 9.24 37.9 [4.25-80.9]
31.5 ± 12.7 30.0 [12.0-61.0]
76.7 ± 17.3 76.0 [39.0-124]
58.4 ± 8.88 58.5 [35.5-79.2]
25.2 ± 4.63 25.2 [16.2-38.3]
107 ± 34.3 103 [53.0-200]
55 (89) 4 (6.5) 0 (0.0) 2 (3.2) 1 (1.6)
11 (18) 51 (82)
1 [1-4]
240 (38) 45 (39)
2 [0-26]
91.8 ± 96.7 49.1 [1.00-351.9]
155 (65) 85 (35)
131 (55) 109 (45)
123 (51) 91 (38) 26 (11)
5.5 [0-55]
LEOPOLD II
59 (34)
12.2 ± 0.165
12.2 [11.7-12.5]
38.9 ± 9.37 38.7 [5.85-199]
29.0 ± 11.1 27.0 [14.0-59.0]
64.9 ± 13.2 64.0 [46.0-98.0]
52.8 ± 7.18 52.2 [40.9-68.4]
21.4 ± 3.94 20.8 [15.0-30.9]
110 ± 38.9 104 [43.0-242]
30 (51) 1 (1.7) 23 (39) 5 (8.5) 0 (0.0)
59 (100) 0 (0.0)
2 [1-5]
292 (46) 43 (37)
2 [0-33]
63.9 ± 66.0 43.0 [0.606-265]
209 (72) 83 (28)
189 (65) 103 (35)
120 (41) 139 (48) 33 (11)
36 [3-106]
LEOPOLD kids
51 (30)
6.12 ± 0.544
6.09 [3.78-7.22]
36.2 ± 10.5 34.1 [19.2-106]
6.59 ± 2.94 6.00 [1.00-11.0]
25.9 ± 10.8 22.6 [11.0-59.0]
23.3 ± 8.66 20.8 [9.25-48.9]
16.4 ± 2.55 15.7 [13.0-24.6]
NA NA
47 (92) 3 (5.9) 0 (0.0) 1 (2.0) 0 (0.0)
11 (22) 40 (78)
1 [1-2]
101 (16) 28 (24)
1 [0-9]
58.0 ± 46.6 5.00 [1.17-182]
24 (24) 77 (76)
8 (7.9) 93 (92)
52 (52) 46 (46) 3 (3.0)
4 [0-55]
Total
172 (100)
10.3 ± 2.86
12.0 [3.08-13.1]
38.2 ± 9.60 37.9 [4.30-199]
23.2 ± 14.9 22.0 [1.00-61.0]
57.6 ± 25.5 60.0 [11.0-124]
46.0 ± 17.1 50.9 [9.25-79.2]
21.3 ± 5.25 20.3 [13.0-38.3]
109 ± 36.5 104 [43.0-242]
132 (77) 8 (4.7) 23 (13) 8 (4.7) 1 (0.60)
81 (47) 91 (53)
1 [1-5]
633 (100) 116 (100)
2 [0-33]
73.3 ± 76.8 48.2 [0.606-352]
388 (61) 245 (39)
328 (52) 305 (48)
295 (47) 276 (44) 62 (9.8)
11 [0-106]
N/n: number; PK: pharmacokinetics; NA: not available; SD: standard deviation. aLean body weight and body mass index missing for one patient. bvon Willebrand fac- tor levels missing for 55 patients. cNumber of target joints missing for 61 patients. dTime to first bleed based on data from patients experiencing at least one bleed. eNumber of bleeds in the last 12 months missing for 3 patients.
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