Table 4.
Main data on long-term prophylaxis with concentrates of von Willebrand factor/factor VIII or von Willebrand factor only products in von Willebrand disease.
| Author | Patients (n) | Median age at start of prophylaxis (range) | VWD types | Median FVIII doses (range) | Indication (n) | Main results |
|---|---|---|---|---|---|---|
| Castaman et al. 25 | 31 | NI | 9 type 1, 6 type 2, 16 type 3 | 30 IU/kg (1–169) 2–3 times weekly | GI and joint bleeding, menorrhagia | Excellent/good responses in 93% of cases |
| Berntorp and Petrini 53 | 35 | 13 years (1–61) | 1 type 1, 6 type 2, 28 type 3 | 24 IU/kg (12–50) 1–3 times weekly |
Mucocutaneous and joint bleeding | Number of bleeds reduced after prophylaxis. No arthropathy in children starting prophylaxis before 5 years of age |
| Federici 56 | 11 | NI | 1 type 1, 5 type 2, 5 type 3 | NI | GI (7) and joint (4) bleeding | Excellent/good responses in 100% of cases. Reduction of annual consumption of VWF/FVIII concentrates, number of transfused blood units and days spent in hospital |
| Holm et al. 59 | 105 | 26 years (1–81) | 13 type 1, 38 type 2, 54 type 3 | NI | Epistaxis (33%), GI (23%) and joint (23) bleeding | Reduction of ABR was statistically significant for all bleeding indications |
| Abshire et al. 60 | 11 | 34.6 years (3–80.6) | 6 type 2, 5 type 3 | 50 IU/kg 2–3 times weekly* | Epistaxis (6), GI (3)and joint bleeding (3) | Median ABR score decreased from 25.0 (IQR: 12.0–51.2) to 6.1 (IQR: 3.1–29.0) |
| Abshire et al. 62 | 59 | 22.4 years (2.3–77.2) | 5 type 1, 20 type 2, 34 type 3 | 40–60 IU/kg (30–6)* 2–3 times weekly | Epistaxis (13), GI (13) and joint bleeding (12) | Prophylaxis was effective in reducing the association bleeding rate, particularly joint bleeding |
| Halimeh et al. 63 | 32 | Children: 13 years, adolescent: 7 years, adults: 12 years | 4 type 1, 15 type 2, 13 type 3 | 40 IU/kg (20–47) 2–3 times weekly* | GI and joint bleeding | Recurrent bleeding stopped in 31 patients. Monthly bleeding frequency significant reduced (p < 0.001) |
ABR, annualized bleeding rate; FVIII, factor VIII; GI, gastrointestinal; IQR, interquartile range; NI, not indicated; VWD, von Willebrand disease.
*
VWF:RCo IU/kg.