Recombinant FVIIa in children with liver disease

INTRODUCTION: The aim of this study was to investigate the clinical and biochemical effects of recombinant activated factor VII (rFVIIa) in the treatment of bleeding in children with liver disease. PATIENTS AND METHODS: 12 patients (0.3-15.9 years) with chronic liver disease were included. The indication for treatment was life threatening bleeding and failing conventional therapy (group A, 7 patients) or as prophylaxis before invasive procedures (group B, 6 patients). One patient received treatment on both indications. rFVIIa was administered as intravenous bolus doses of 34-163 microg/kg (median 66 mug/kg) alone or in combination with packed red cells and/or octreotide and/or fresh frozen plasma. The follow-up included repeated INR and haemoglobin measurements as well as clinical evaluation. RESULTS: In group A rFVIIa was given on 22 occasions and bleeding decreased, was unchanged, increased or could not be evaluated on 10, 7, 2 and 3 occasions respectively. On 14 occasions rFVIIa and octreotide were administered simultaneously, in 8 of those bleeding decreased. In group B no bleeding complication was seen, interpreted as a positive effect. One thrombotic event was suspected but could not be verified by computerized tomography. CONCLUSIONS: rFVIIa may be beneficial in the short-term management of life threatening bleeding in some children with liver disease. This effect may be further enhanced with the additional use of octreotide. Furthermore, rFVIIa is useful for prophylaxis at invasive procedures, even without additional treatment with fresh frozen plasma. The possible risk of portal vein thrombosis needs to be considered.