Table 1 Patient demographic and clinical characteristics of each analytic sample
Characteristic | Direct costs Sample n = 794 | Societal costs Sample n = 220 | HRQoL (EQ-5D-Y) Sample n = 185 |
|---|---|---|---|
Age, mean (SD) | 10.5 (4.74) | 10.2 (4.58) | 11.2 (3.79) |
BMI z-score, mean (SD) | 0.6 (1.59) | 0.4 (1.46) | 0.4 (1.39) |
BMI, n (%) | |||
Underweight | 24 (3) | 12 (5) | 8 (4) |
Normal weight | 474 (60) | 132 (60) | 113 (61) |
Overweight | 167 (21) | 41 (19) | 34 (18) |
Obese | 129 (16) | 35 (16) | 30 (16) |
Country, n (%) | |||
Germany | 131 (16) | 55 (25) | 49 (26) |
Spain | 182 (23) | 40 (18) | 27 (15) |
France | 171 (22) | 40 (18) | 30 (16) |
Italy | 197 (25) | 38 (17) | 35 (19) |
United Kingdom | 113 (14) | 47 (21) | 44 (24) |
Haemophilia type, n (%) | |||
A | 627 (79) | 168 (76) | 143 (75) |
B | 167 (21) | 52 (24) | 47 (25) |
Severity, n (%) | |||
Moderate | 331 (42) | 58 (26) | 43 (23) |
Severe | 463 (58) | 162 (74) | 142 (77) |
Comorbidities, n (%) | |||
0 | 648 (82) | 178 (81) | 151 (82) |
1 | 103 (13) | 25 (11) | 21 (11) |
≥ 2 | 43 (5) | 17 (8) | 13 (7) |
Treatment by severity*, n (%) | |||
Overall | n = 794 | n = 220 | n = 185 |
No treatment | 92 (12) | 12 (5) | 8 (4) |
On-demand | 95 (12) | 18 (8) | 17 (9) |
Prophylaxis | 607 (76) | 190 (86) | 160 (86) |
Moderate haemophilia | n = 332 | n = 58 | n = 43 |
No treatment | 91 (27) | 12 (21) | 8 (19) |
On-demand | 48 (15) | 1 (2) | 3 (7) |
Prophylaxis | 192 (58) | 45 (78) | 32 (74) |
Severe haemophilia | n = 462 | n = 162 | n = 142 |
No treatment | 1 (0.2) | 0 | 0 |
On-demand | 47 (10) | 17 (10) | 14 (10) |
Prophylaxis | 415 (90) | 145 (90) | 128 (90) |
Annual bleeding rate, n (%) | |||
Overall | n = 794 | n = 220 | n = 185 |
0 | 119 (15) | 14 (6) | 12 (6) |
1–5 | 609 (77) | 182 (83) | 152 (82) |
> 5 | 66 (8) | 24 (11) | 21 (11) |
Moderate | n = 331 | n = 58 | n = 43 |
0 | 59 (18) | 3 (5) | 3 (7) |
1–5 | 255 (77) | 50 (86) | 35 (81) |
> 5 | 17 (5) | 5 (9) | 5 (12) |
Severe | n = 463 | n = 162 | n = 142 |
0 | 60 (13) | 11 (7) | 9 (6) |
1–5 | 354 (76) | 132 (81) | 117 (82) |
> 5 | 49 (11) | 19 (12) | 16 (11) |
Number of problem joints, n (%) | |||
Overall | n = 794 | n = 220 | n = 185 |
0 | 694 (87) | 177 (80) | 151 (82) |
≥ 1 | 100 (13) | 43 (20) | 34 (18) |
Moderate | n = 331 | n = 58 | n = 43 |
0 | 299 (90) | 48 (83) | 37 (86) |
≥ 1 | 32 (10) | 10 (17) | 6 (14) |
Severe | n = 463 | n = 162 | n = 142 |
0 | 395 (85) | 129 (80) | 114 (80) |
≥ 1 | 68 (15) | 33 (20) | 30 (20) |