Use of recombinant factor VIIa in severe acquired coagulopathies in childhood

Abstract number: P0065

Hassenpflug^* W., Rischewski^* J., Hellwege† H. H., Zander‡ A. R., Schneppenheim^* R.

‡Department of Bone Marrow Transplantation †Intensive Care Unit; ^*University Children's Hospital, Hamburg, Germany;

Introduction  

Recombinant factor VIIa (rFVIIa) is an effective treatment for hemophilic patients with inhibitors. Besides this well recognized therapy rFVIIa is used to control bleedings caused by a variety of diseases like thrombocytopathies, liver disease, during liver transplantation or cardiac surgery. In contrast to rising experience obtained by ongoing clinical trials in adults, poor data exit for the use of rFVIIa in acquired coagulopathies in childhood.

Patients  

We report on six children, 1 month to 13 years old, with severe bleeding episodes treated by recombinant factor VIIa. Two patients underwent bone marrow transplantation for SCID, lymphoma leukemia and neuroblastoma respectively. In one patient diagnosis was hemophagocytic lymphohistiocytosis, two patients suffered from severe liver insufficiency and received liver transplantation and one patient had a transposition of the great arteries to be corrected by heart surgery. Manifestation of bleeding included mucosal/gastrointestinal bleeding, intra-abdominal bleeding intracerebral hemorrhage and pulmonary bleeding. Recombinant factor VIIa was used as bolus therapy. Dose regimen was calculated individually with doses of 60–180 µg kg^-1 bw every 2–12 h. We monitored the response to the therapy by a simple bleeding score, an estimation of blood loss and by coagulation tests.

Results  

In all patients, bleeding symptoms could be markedly reduced or stopped by application of rFVIIa. The bleeding score and coagulation tests normalized. In one patient with thrombocytopenia (13 nL^-1) mucosal bleeding did not stop after rFVIIa but resolved after transfusion of a platelet concentrate and local application of fibrin glue. No adverse effects were observed, especially no thrombembolic complications. Nevertheless five of the nine patients died for underlying disease.

Conclusion  

The use of recombinant factor VIIa may be a valuable therapeutic option in children with severe acquired coagulopathies since it provides a good hemostatic efficacy and probably few adverse side-effects. A minimal platelet count seems to be necessary for its mode of action.