Table 4.
Comparison of treatment results on PX and OD regimens in patients with both regimens during the observed period (adult patients with severe haemophilia A)
| OD, N = 106 | PX, N = 106 | p value1 | |
|---|---|---|---|
| Age at start of treatment regimen (years) | |||
| Mean (SD) | 37.6 (13.9) | 39.6 (14.9) | |
| Median (range) | 34.5 (26.2–47.8) | 37.0 (28.0–49.0) | |
| Follow-up duration (years) | 0.003 | ||
| Mean (SD) | 3.3 (2.2) | 4.6 (2.7) | |
| Median (range) | 3 (1–9) | 4 (1–9) | |
| Sum | 350 | 485 | |
| PX dose (IU/kg per week) | |||
| Mean (SD) | NA | 50.2 (21.2) | |
| Median (IQR) | 47.4 (37.3–59.2) | ||
| ABR | < 0.001 | ||
| Mean (SD) | 11.8 (12.7) | 3.4 (5.8) | |
| Median (range) | 8.2 (0.0–57.0) | 1.9 (0.0–36.5) | |
| JABR | < 0.001 | ||
| Mean (SD) | 9.0 (12.4) | 2.3 (4.2) | |
| Median (range) | 5.0 (0.0–54.5) | 1.0 (0.0–28.3) | |
| ABR, n (%) | < 0.001 | ||
| 0–1 | 22 (26) | 41 (40) | |
| 2–5 | 12 (14) | 41 (40) | |
| > 5 | 52 (60) | 20 (20) | |
| JABR, n (%) | < 0.001 | ||
| 0–1 | 29 (37) | 50 (51) | |
| 2–5 | 12 (15) | 40 (40) | |
| > 5 | 38 (48) | 9 (9.1) | |
| Total of FVIII consumption (IU/kg per year) | < 0.001 | ||
| Mean (SD) | 1500 (1591) | 2676 (1291) | |
| Median (IQR) | 1165 (646–1692) | 2489 (1891–3091) | |
| Total of pdFVIII consumption (IU/kg per year) | 0.002 | ||
| Mean (SD) | 1595 (2619) | 2027 (1370) | |
| Median (IQR) | 1050 (620–1599) | 1837 (1273–2518) | |
| Total of rFVIII consumption (IU/kg per year) | < 0.001 | ||
| Mean (SD) | 1184 (871) | 2529 (1387) | |
| Median (IQR) | 1286 (346–1706) | 2312 (1538–3059) | |
| Total of rFVIII EHL consumption (IU/kg per year) | 0.95 | ||
| Mean (SD) | 1555 (751) | 2054 (1057) | |
| Median (IQR) | 1404 (1062–2120) | 2159 (1348–2740) |
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
1Wilcoxon signed rank test with continuity correction