Haemophilia : the official journal of the World Federation of Hemophilia
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Case Reports
Use of recombinant factor VIIa (NovoSeven) in a haemophilia A patient with inhibitor in Kuwait.
Development of inhibitors is a known complication in some haemophiliacs receiving coagulation factor replacement therapy. We report on the successful management of a young boy with haemophilia A with inhibitor using recombinant factor VIIa. ⋯ Recombinant factor VIIa is a useful 'factor VIII bypassing agent' for the control of bleeding in patients with haemophilia A and B who develop inhibitors. We suggest that severely affected haemophiliacs should be absolved of ritual circumcision as a protective measure against what might become a life-threatening haemorrhage - especially in those with inhibitors.
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Multicenter Study Clinical Trial Controlled Clinical Trial
Recombinant factor VIIa for patients with inhibitors to factor VIII or IX or factor VII deficiency.
Inhibitors to factor VIII (FVIII) or IX (FIX) in patients with haemophilia A or B create a challenging problem for the treatment of these patients. Recombinant FVIIa (rFVIIa; NovoSeven, Novo Nordisk A/S, Bagsvaerd, Denmark) is a realistic treatment option, owing to its specific mode of action and lack of immunogenicity. This was a multicentre, open-label, compassionate-use trial in patients with severe haemophilia A (FVIII:C < 1%) or B (FIX:C < 1%) with inhibitors, acquired antibodies to FVIII or FIX, or FVII deficiency (FVII:C < 5%), for whom alternative therapies had failed or were contraindicated. ⋯ Only 10 were considered as having a possible, probable, or unknown relationship with rFVIIa; of these, fever (n=2) and thrombophlebitis (n=3) were the most common. There was no evidence of disseminated intravascular coagulation. In conclusion, rFVIIa is an effective, well-tolerated treatment for serious bleeding episodes and bleeding associated with surgical procedures in patients with severe haemophilia A/B with inhibitors, acquired inhibitors, or FVII deficiency.