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Recombinant FVIIa in children with liver disease
Maria Pettersson1, Björn Fischler, Pia Petrini
1Department of Paediatrics, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm S-141 86, Sweden. Maria.Petterson@klinvet.ki.se
Insights
Recombinant activated factor VII (rFVIIa) shows promise for managing severe bleeding in children with liver disease and for prophylaxis during procedures. Octreotide may enhance its effectiveness, but portal vein thrombosis is a potential risk.
Area of Science:
- Pediatric Hematology
- Hepatology
- Pharmacology
Background:
- Children with chronic liver disease often experience severe bleeding.
- Conventional therapies may be insufficient for managing bleeding in this population.
Purpose of the Study:
- To evaluate the clinical and biochemical effects of recombinant activated factor VII (rFVIIa) in pediatric liver disease patients.
- To assess rFVIIa's efficacy in treating life-threatening bleeding and as prophylaxis for invasive procedures.
Main Methods:
- A study involving 12 pediatric patients (0.3-15.9 years) with chronic liver disease.
- rFVIIa administered intravenously (median 66 µg/kg) for bleeding treatment or procedural prophylaxis.
- Follow-up included INR, hemoglobin, and clinical assessments.
Main Results:
- In bleeding episodes, rFVIIa led to decreased bleeding in 10 of 22 occasions, with enhanced effect when combined with octreotide.
- No bleeding complications occurred in patients receiving rFVIIa for prophylaxis.
- One suspected thrombotic event was noted but unconfirmed.
Conclusions:
- rFVIIa may offer short-term benefits for severe bleeding in pediatric liver disease.
- Combined use with octreotide might improve efficacy.
- rFVIIa is effective for prophylaxis in invasive procedures.
- Potential risk of portal vein thrombosis requires consideration.
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.
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