Table 1.
Patients’ characteristics and treatment outcomes: adult patients with haemophilia A
| 2012, N = 358 | 2013, N = 377 | 2014, N = 402 | 2015, N = 431 | 2016, N = 436 | 2017, N = 441 | 2018, N = 453 | 2019, N = 469 | 2020, N = 471 | 2021, N = 470 | |
|---|---|---|---|---|---|---|---|---|---|---|
| Age | ||||||||||
| Mean (SD) | 42 (17) | 42 (17) | 42 (17) | 43 (17) | 44 (17) | 44 (17) | 44 (17) | 44 (17) | 45 (17) | 45 (17) |
| Median (range) | 40 (19–90) | 39 (19–91) | 39 (19–92) | 40 (19–93) | 41 (19–94) | 41 (19–95) | 41 (19–96) | 41 (19–89) | 42 (19–90) | 43 (19–91) |
| Severity, n (%) | ||||||||||
| Mild | 159 (45) | 178 (48) | 196 (49) | 221 (51) | 230 (53) | 234 (53) | 245 (54) | 251 (54) | 258 (55) | 261 (56) |
| Moderate | 39 (11) | 41 (11) | 42 (10) | 42 (9.7) | 36 (8.3) | 36 (8.2) | 39 (8.6) | 39 (8.4) | 41 (8.8) | 37 (7.9) |
| Severe | 149 (42) | 148 (40) | 158 (39) | 160 (37) | 161 (37) | 163 (37) | 161 (36) | 165 (36) | 160 (34) | 163 (35) |
| Inhibitor | 4 (1.1) | 5 (1.3) | 6 (1.5) | 8 (1.9) | 9 (2.1) | 8 (1.8) | 8 (1.8) | 8 (1.7) | 8 (1.7) | 7 (1.5) |
| Treatment regimen, n (%) | ||||||||||
| OD | 314 (88) | 315 (84) | 324 (81) | 342 (79) | 344 (79) | 338 (77) | 348 (77) | 349 (74) | 348 (74) | 340 (72) |
| PX | 44 (12) | 62 (16) | 78 (19) | 89 (21) | 92 (21) | 103 (23) | 105 (23) | 120 (26) | 123 (26) | 130 (28) |
| ABR | ||||||||||
| Mean (SD) | NA | 5.3 (12.2) | 4.7 (9.7) | 3.3 (7.7) | 3.0 (8.3) | 2.5 (6.6) | 1.8 (4.6) | 1.6 (5.0) | 1.2 (3.7) | 0.8 (2.7) |
| Median (range) | 0 (0–124) | 0 (0–55) | 0 (0–59) | 0 (0–60) | 0 (0–54) | 0 (0–39) | 0 (0–51) | 0 (0–44) | 0 (0–24) | |
| JABR | ||||||||||
| Mean (SD) | NA | 3.9 (11.6) | 3.6 (8.5) | 2.3 (6.4) | 2.2 (7.1) | 1.8 (5.4) | 1.1 (3.3) | 1.0 (3.5) | 0.8 (3.1) | 0.5 (2.1) |
| Median (range) | 0 (0–124) | 0 (0–55) | 0 (0–59) | 0 (0–60) | 0 (0–49) | 0 (0–39) | 0 (0–50) | 0 (0–44) | 0 (0–22) | |
| Total of FVIII consumption (IU/kg per year) | ||||||||||
| Mean (SD) | 1264 (1300) | 1256 (1880) | 1486 (1958) | 1273 (1396) | 1579 (2136) | 1475 (1746) | 1781 (2509) | 1845 (1669) | 1875 (1772) | 1928 (1911) |
| Median (IQR) | 857 (274–1907) | 731 (223–1844) | 990 (318–2048) | 802 (190–1961) | 859 (226–2308) | 780 (175–2293) | 1163 (161–2917) | 1555 (345–2952) | 1707 (179–3168) | 1533 (183–3152) |
| Surgery, n (%) | NA | NA | NA | NA | 36 (8.3) | 38 (8.6) | 39 (8.6) | 65 (13.9) | 42 (8.9) | 42 (8.9) |
| Hospitalisation due to bleeding, n (%) | NA | NA | NA | NA | NA | NA | 26 (5.7) | 22 (4.7) | 24 (5.1) | 14 (3.0) |
| Length of hospit. due to bleeding (days) | ||||||||||
| Mean (SD) | NA | NA | NA | NA | NA | NA | 15 (28) | 12 (15) | 18 (50) | 13 (11) |
| Median (IQR) | 7 (4–12) | 5 (2–17) | 7 (6–11) | 10 (3–22) |
NA not available, ABR annual bleeding rate, JABR joint annual bleeding rate, OD on demand, PX prophylaxis, SD standard deviation, IQR interquartile range