USE OF RECOMBINANT FACTOR VIIA IN PAEDIATRIC PATIENTS WITH LIVER FAILURE AND SEVERE COAGULOPATHY

P701 Aims: The only approved indications for recombinant Factor VIIa (rFVIIa, NiaStase, Novo Nordisk) in Canada are patients with inhibitors in haemophilia A or B and severe congenital factor VII deficiency. There have been a number of small trials studying its uses in non haematological indications, including liver disease and transplantation. A recent study reported improvement in PT’s in children with liver failure (Brown et al J.Ped. Gastroenterol. Nutr. 37:268, 2003).; however, there are few reports of its use in children with liver failure. Recently, we have used rFVIIa in 3 patients with liver failure, severe coagulopathy with bleeding who demonstrated signifcant laboratory and clinical improvement and no side effects. Patients and Methods: Patient 1 was an 11-y old boy with TPN-induced liver failure from short bowel who had 2 to 3 bleeds daily from his duodenostomy in addition to frequent nose bleeds. He was receiving: fresh frozen plasma (ffp) 20-30 ml/kg/d, a continuous octreotide infusion (20 to 80 μg/hour) aprotonin, thrombostatin and platelets to try and control his bleeding with minimal success. He received 0.2mg/kg/dose of rFVIIa on 5 separate occasions for acute bleeds which had not resolved within 3 hours after his usual treatment. His INR (normal 0.9 to 1.1) improved from 1.9 +/- 0.15 to 1.3 +/-0.15 and his factor VII level (normal 0.42 to 1.58 U/ml) increased from 0.44 U/ml to 1.4 U/ml. More importantly each of his bleeding episodes stopped within 10 minutes of administering the rFVIIa. Patient 2 was a 7 mo old girl with biliary atresia who developed acute on chronic liver failure with an INR ranging from 3.4 to 5.8 and bleeding from multiple sites despite 20 to 30 ml/kg/d of ffp. At the time of transplant she received 0.3 mg/kg of rFVIIa at the start of her liver transplant. One hour post infusion, her INR decreased from 3.4 to1.2, her factor VII level increased from 0.06 to 15.1 and her bleeding stopped. Patient 3 was a 3 mo old boy with fulminant liver failure of unknown etiology, bleeding from multiple sites and an INR ranging from 3.2 to 5.3. He was given 0.13 mg/kg of rFVIIa 1 hour prior to a liver biopsy and a second dose 2 hours later. His INR improved from 3.2 to 1.0, his factor VII level increased from 0.05 to 1.57 and there was no bleeding or other complications from the biopsy or the rFVIIa. Twenty hours later his INR was 2.3 and his factor VII level was 0.1 U/ml. Conclusions: Consistent with other reports, rFVIIa provided significant benefit in these children with liver failure and severe coagulopathy, in terms of clinical and laboratory improvement in their bleeding and coagulation profiles. There were no obvious side effects from the rFVIIa. This drug may be an important tool in the treatment of children with liver failure and more study is needed to define the optimal dosing for children.

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