Plasma half-life, therapeutic target levels, available treatment, and therapeutic dosages for each RCD (on demand and prophylaxis)
| Deficient factor . | Plasma half-life . | Trough levels . | Available treatment . | On-demand dosages . | Long-term prophylaxis dosages . | |
|---|---|---|---|---|---|---|
| Previously reported . | EN-RBD* . | |||||
| Fibrinogen | 2-4 d | 0.5-1 g/L | 1 g/L | Cryoprecipitate | 15-20 mL/kg | 1 bag/10 kg/7-10 d |
| FFP† | 15-30 mL/kg | — | ||||
| Fibrinogen concentrate | 50-100 mg/kg | 20-30 mg/kg/wk | ||||
| Prothrombin | 3-4 d | 20%-30% | >10% | FFP† | 15-25 mL/kg | — |
| PCC | 20-40 U/kg | 20-40 U/kg once/wk | ||||
| FV | 36 h | 10%-20% | 10% | FFP† | 15-25 mL/kg | 20 mL/kg 2 times/wk |
| Platelet transfusions could be considered, with particular attention on alloimmunization | — | |||||
| FV and FVIII | FVIII 10-14 h | 10%-15% | 40% | FV deficiency: (see above) mild FVIII deficiency: DDAVP moderate and severe FVIII deficiency: pd- or rFVIII concentrates | Usually no need for prophylaxis | |
| FVII | 4-6 h | 10%-15% | >20% | FFP† | — | 10-15 mL/kg 2 times/wk |
| pd-FVII concentrate | 30-40 U/kg | 30-40 U/kg 3 times/wk | ||||
| rFVIIa | 15-30 µg/kg every 4-6 h | 20-40 mg/kg 2-3 times/wk | ||||
| FX | 40-60 h | 10%-20% | >40% | FFP† | 10-20 mL/kg | — |
| PCC | 20-30 U/kg | 20-40 U/kg 2 times/wk | ||||
| pd-FX/FIX concentrate | 10-20 U/kg | 20 U/kg/weekly | ||||
| pd-FX | 25 U/kg | 25 U weekly | ||||
| FXI | 50 h | 15%-20% | — | FFP† | 15-20 mL/kg | Not indicated |
| pd-FXI concentrate | 15-20 U/kg | |||||
| FXIII | 9-12 d | 2%-5% | 30% | Cryoprecipitate | 2-3 bags | 1 bag/10 kg/3 wk |
| FFP† | 3-5 mL/kg | — | ||||
| pd-FXIII concentrate | 20-40 U/kg | 20-40 U/kg/4 wk‡ | ||||
| rFXIII-A | 35 U/kg | 35 U/kg/4 wk (2-3 wk in pregnant women) | ||||
| Vitamin K dependent | Prothrombin, FVII, FIX, FX (see specific factors) | Vitamin K1 | 10 mg for minor bleeding | 5-20 mg/daily (orally) 5-20 mg/wk (parenteral) | ||
| 4-factor PCC | 20-30 U/kg | — | ||||
| FFP† | 15-25 mL/kg | — | ||||
| Deficient factor . | Plasma half-life . | Trough levels . | Available treatment . | On-demand dosages . | Long-term prophylaxis dosages . | |
|---|---|---|---|---|---|---|
| Previously reported . | EN-RBD* . | |||||
| Fibrinogen | 2-4 d | 0.5-1 g/L | 1 g/L | Cryoprecipitate | 15-20 mL/kg | 1 bag/10 kg/7-10 d |
| FFP† | 15-30 mL/kg | — | ||||
| Fibrinogen concentrate | 50-100 mg/kg | 20-30 mg/kg/wk | ||||
| Prothrombin | 3-4 d | 20%-30% | >10% | FFP† | 15-25 mL/kg | — |
| PCC | 20-40 U/kg | 20-40 U/kg once/wk | ||||
| FV | 36 h | 10%-20% | 10% | FFP† | 15-25 mL/kg | 20 mL/kg 2 times/wk |
| Platelet transfusions could be considered, with particular attention on alloimmunization | — | |||||
| FV and FVIII | FVIII 10-14 h | 10%-15% | 40% | FV deficiency: (see above) mild FVIII deficiency: DDAVP moderate and severe FVIII deficiency: pd- or rFVIII concentrates | Usually no need for prophylaxis | |
| FVII | 4-6 h | 10%-15% | >20% | FFP† | — | 10-15 mL/kg 2 times/wk |
| pd-FVII concentrate | 30-40 U/kg | 30-40 U/kg 3 times/wk | ||||
| rFVIIa | 15-30 µg/kg every 4-6 h | 20-40 mg/kg 2-3 times/wk | ||||
| FX | 40-60 h | 10%-20% | >40% | FFP† | 10-20 mL/kg | — |
| PCC | 20-30 U/kg | 20-40 U/kg 2 times/wk | ||||
| pd-FX/FIX concentrate | 10-20 U/kg | 20 U/kg/weekly | ||||
| pd-FX | 25 U/kg | 25 U weekly | ||||
| FXI | 50 h | 15%-20% | — | FFP† | 15-20 mL/kg | Not indicated |
| pd-FXI concentrate | 15-20 U/kg | |||||
| FXIII | 9-12 d | 2%-5% | 30% | Cryoprecipitate | 2-3 bags | 1 bag/10 kg/3 wk |
| FFP† | 3-5 mL/kg | — | ||||
| pd-FXIII concentrate | 20-40 U/kg | 20-40 U/kg/4 wk‡ | ||||
| rFXIII-A | 35 U/kg | 35 U/kg/4 wk (2-3 wk in pregnant women) | ||||
| Vitamin K dependent | Prothrombin, FVII, FIX, FX (see specific factors) | Vitamin K1 | 10 mg for minor bleeding | 5-20 mg/daily (orally) 5-20 mg/wk (parenteral) | ||
| 4-factor PCC | 20-30 U/kg | — | ||||
| FFP† | 15-25 mL/kg | — | ||||
Plasma half-life, therapeutic target levels, available treatment, and therapeutic dosages for each RCD (on demand and prophylaxis).12,48
—, no data; DDAVP, 1-deamino-8-d-arginine vasopressin; EN-RBD, European Network of Rare Bleeding Disorders; pd, plasma derived.
EN-RBD retrospective study (2007-2010).48
Virus-inactivated plasma preferable.
In countries with low income and a high number of patients with FXIII deficiency, a prophylaxis program of 10 U/kg every 4 or 6 wk was shown to be cost-beneficial and acceptable for prevention of severe bleeding symptoms.95