Recombinant human factor vila in the management of amyloid-associated factor x deficiency

Lisa N. Boggio*, David Green

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Factor X deficiency is a dreaded complication of systemic amyloid disease, associated with frequent and serious bleeding. Replacement therapy with factor X, using either plasma or prothrombin complex concentrates, is often ineffective because of the rapid removal of factor X from the circulation. Splenectomy may produce a rapid increase in factor X levels because of the removal of factor X-binding amyloid deposits. Recombinant factor Vila (rhVIIa) is a coagulation concentrate which has proved safe and effective in the management of bleeding in patients with a variety of acquired coagulopathies. We administed rh Vila to a patient with amyloid-associated factor X deficiency, to determine whether this concentrate would provide effective hemostasis for splenectomy. A 63 year old woman with carpal tunnel syndrome lost 20 pounds over a two month period. Large ecchymoses appreared on the breast and leg after minimal trauma. The factor X level was 4%. Other studies showed a restricted band in the gamma region and a monoclonal lambda light chain in the urine. A bone marrow biopsy demonstrated the presence of amyloid. Because of persistent skin and muscle bleeding, she was treated with a prothrombin complex concentrate which neither increased the factor X level nor decreased blood loss. Splenectomy was recommended because of the patient's persistent bleeding and very low factor X levels. rhVIIa, 90 meg/kg, was given immediately pre-operatively and every 3 hours post-operatively for 48 hours. An open splenectomy was performed without any increase in operative bleeding; the estimated blood loss was 200 ml. Immediately following surgery, the prothrombin time decreased from 29 seconds to 16 seconds, and 48 hours after operation it was 10 seconds. The factor X level rose from 4% to 99%. The post-operative course was uneventful. The resected spleen weighed only 182 grams but displayed extensive amyloid deposition. We speculate that platelet-bound factor X may be protected from adsorption to amyloid fibrils. This factor X may be available for activation by the rhVIIa and participate in the generation of thrombin on the platelet surface.

Original languageEnglish (US)
JournalBlood
Volume96
Issue number11 PART II
StatePublished - Dec 1 2000

ASJC Scopus subject areas

  • Hematology

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