Table II.
Outcome according to residual factor activity level.
| 1 IU/dL n=5 |
2 IU/dL n=23 |
3 IU/dL n=26 |
4 IU/dL n=13 |
5 IU/dL n=8 |
|
|---|---|---|---|---|---|
| Haemophilia B | 1 (20%) | 3 (13%) | 3 (12%) | 0 | 1 (13%) |
|
| |||||
| Bleeding and treatment | |||||
| Age at 1st joint bleed (years) | 2.8 (2.3–2.8) | 4.5 (3.5–8.3) | 4.9 (4.1–8.5) | 5.7 (4.0–12) | 6.1 (4.3–18) |
| Median annual n. of bleeds | 2.9 (1.9–7.8) | 3.7 (1.8–7.7) | 1.9 (0.8–3.6) | 1.2 (0.5–3.1) | 1.2 (0.2–2.1) |
| Median annual n. of joint bleeds | 1.0 (0.3–3.1) | 1.3 (0.4–2.5) | 0 (0–1.2) | 0 (0–0.96) | 0 (0–0.73) |
| No joint bleeds in last 5 years | 2 (40%) | 7 (32%) | 14 (54%) | 8 (62%) | 3 (38%) |
| History of prophylaxis | 2 (40%) | 11 (44%) | 5 (17%) | 3 (19%) | 1 (13%) |
| Annual factor consumption (IU/kg/year) | 119 (48–1,215) | 264 (119–1,044) | 217 (34–347) | 22 (0–244) | 46 (12–277) |
| Without treatment | 0 | 2 (8%) | 0 | 2 (15%) | 2 (25%) |
|
| |||||
| Orthopaedic outcome | |||||
| HJHS (max. 128 points) | 0 | 5 (2–10) | 4 (0–9) | 1 (0–4) | 2 (0–6) |
| History of orthopaedic surgery | 0 | 5 (22%) | 4 (15%) | 4 (31%) | 0 |
| HAL sum score (max 100 points) | 97 | 96 (81–100) | 96 (85–100) | 92 (79–97) | 86 (70–100) |
| Utility (EQ5D, max 1.0) | 0.80 | 0.90 (0.72–1) | 1 (0.78–1) | 0.80 (0.73–1) | 0.76 (0.62–0.81) |
Values are medians (IQR) or n (%).