Table 2.
Patients’ characteristics and treatment outcomes: adult patients with severe haemophilia A
| 2012, N = 149 | 2013, N = 148 | 2014, N = 158 | 2015, N = 160 | 2016, N = 161 | 2017, N = 163 | 2018, N = 161 | 2019, N = 165 | 2020, N = 160 | 2021, N = 163 | |
|---|---|---|---|---|---|---|---|---|---|---|
| Age | ||||||||||
| Mean (SD) | 40 (16) | 41 (16) | 42 (16) | 42 (16) | 43 (16) | 43 (16) | 43 (16) | 43 (16) | 44 (16) | 44 (17) |
| Median (range) | 38 (19–73) | 39 (19–74) | 40 (19–75) | 40 (19–76) | 41 (19–77) | 40 (19–78) | 40 (19–79) | 40 (19–80) | 42 (19–81) | 41 (19–82) |
| Treatment regimen, n (%) | ||||||||||
| OD | 116 (78) | 93 (63) | 88 (56) | 79 (49) | 76 (47) | 66 (40) | 65 (40) | 52 (32) | 46 (29) | 42 (26) |
| PX | 33 (22) | 55 (37) | 70 (44) | 81 (51) | 85 (53) | 97 (60) | 96 (60) | 113 (68) | 114 (71) | 121 (74) |
| ABR | ||||||||||
| Mean (SD) | NA | 12.4 (16.9) | 10.9 (13.1) | 7.6 (10.9) | 7.3 (12.2) | 6.1 (9.8) | 4.5 (6.9) | 4.2 (7.8) | 2.7 (5.6) | 1.8 (4.2) |
| Median (range) | 6 (0–124) | 5 (0–55) | 3 (0–59) | 2 (0–60) | 2 (0–54) | 2 (0–39) | 1 (0–51) | 1 (0–44) | 0 (0–24) | |
| JABR | ||||||||||
| Mean (SD) | NA | 11.3 (18.0) | 8.8 (12.2) | 5.7 (9.7) | 5.8 (10.8) | 4.5 (8.2) | 2.9 (5.2) | 2.7 (5.5) | 1.9 (4.9) | 1.3 (3.3) |
| Median (range) | 4 (0–124) | 4 (0–55) | 2 (0–59) | 1 (0–60) | 1 (0–49) | 1 (0–39) | 1 (0–50) | 0 (0–44) | 0 (0–22) | |
| Total of FVIII consumption (IU/kg per year) | ||||||||||
| Mean (SD) | 1572 (1378) | 1665 (2154) | 1865 (2186) | 1742 (1432) | 2050 (1909) | 1935 (1613) | 2237 (1612) | 2509 (1535) | 2665 (1597) | 2805 (1588) |
| Median (IQR) | 1219 (531–2143) | 1330 (480–2195) | 1402 (651–2305) | 1467 (674–2453) | 1749 (699–2904) | 1690 (567–2972) | 2095 (582–3317) | 2472 (1512–3405) | 2601 (1704–3733) | 2836 (1747–4105) |
| pdFVIII therapy, n (%) | 62 (82) | 97 (84) | 105 (79) | 93 (73) | 94 (67) | 94 (64) | 74 (51) | 62 (42) | 32 (23) | 23 (16) |
| rFVIII therapy, n (%) | 15 (20) | 26 (22) | 28 (21) | 33 (26) | 51 (36) | 65 (44) | 81 (56) | 96 (65) | 68 (49) | 60 (42) |
| rFVIII EHL therapy, n (%) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 37 (25) | 62 (45) | 93 (65) |
| Surgery, n (%) | NA | NA | NA | NA | 16 (9.9) | 18 (11) | 17 (10.6) | 20 (12.1) | 15 (9.4) | 11 (6.7) |
| Hospitalisation due to bleeding, n (%) | NA | NA | NA | NA | NA | NA | 15 (9.3) | 10 (6.1) | 13 (8.1) | 4 (2.5) |
| Length of hospit. due to bleeding (days) | ||||||||||
| Mean (SD) | NA | NA | NA | NA | NA | NA | 19 (37) | 4 (4) | 7 (5) | 2 (1) |
| Median (IQR) | 7 (4–11) | 2 (1–6) | 7 (2–10) | 2 (1–3) |
NA not available, ABR annual bleeding rate, JABR joint annual bleeding rate, OD on demand, PX prophylaxis, pdFVIII plasma-derived factor VIII, rFVIII recombinant factor VIII, EHL extended half-life, SD standard deviation, IQR interquartile range