Health economic review of recombinant activated factor VII for treatment of bleeding episodes in hemophilia patients with inhibitors. 2007

Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
Pharmerit North America LLC, 7272 Wisconsin Ave, Suite 300, Bethesda, MD 20814-4858, USA. jstephens@pharmerit.com

Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic and economic consequences. This review provides an overview of the role of recombinant activated factor VII (rFVIIa) versus plasma-derived bypassing agents for hemophilia with inhibitors and summarizes the 13 formal economic analyses (6 burden of illness and 7 comparative studies) that have been published in this indication. The findings suggest that the economic impact of rFVIIa has occurred primarily during hospitalization to manage major bleeding episodes and to allow for elective orthopedic surgeries that would not have been attempted prior to rFVIIa. Comparative analyses for on-demand treatment suggest that the total cost of treating a bleeding episode with rFVIIa may be lower than with plasma-based agents due to faster bleeding resolution, higher initial efficacy rates and avoidance of second and third lines of treatment.

UI MeSH Term Description Entries
D011994 Recombinant Proteins Proteins prepared by recombinant DNA technology. Biosynthetic Protein,Biosynthetic Proteins,DNA Recombinant Proteins,Recombinant Protein,Proteins, Biosynthetic,Proteins, Recombinant DNA,DNA Proteins, Recombinant,Protein, Biosynthetic,Protein, Recombinant,Proteins, DNA Recombinant,Proteins, Recombinant,Recombinant DNA Proteins,Recombinant Proteins, DNA
D002836 Hemophilia B A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008) Christmas Disease,Factor IX Deficiency,Deficiency, Factor IX,F9 Deficiency,Haemophilia B,Hemophilia B Leyden,Hemophilia B(M),Plasma Thromboplastin Component Deficiency,Bs, Hemophilia,Deficiencies, F9,Deficiencies, Factor IX,Deficiency, F9,Disease, Christmas,F9 Deficiencies,Factor IX Deficiencies,Haemophilia Bs,Hemophilia Bs,Hemophilia Bs (M)
D003029 Coagulants Agents that cause clotting. Coagulant
D005167 Factor VII Heat- and storage-stable plasma protein that is activated by tissue thromboplastin to form factor VIIa in the extrinsic pathway of blood coagulation. The activated form then catalyzes the activation of factor X to factor Xa. Coagulation Factor VII,Proconvertin,Stable Factor,Blood Coagulation Factor VII,Factor 7,Factor Seven,Factor VII, Coagulation
D006467 Hemophilia A The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage. Factor VIII Deficiency,Hemophilia,Autosomal Hemophilia A,Classic Hemophilia,Deficiency, Factor VIII,Factor 8 Deficiency, Congenital,Factor VIII Deficiency, Congenital,Haemophilia,Hemophilia A, Congenital,Hemophilia, Classic,As, Autosomal Hemophilia,Autosomal Hemophilia As,Classic Hemophilias,Congenital Hemophilia A,Congenital Hemophilia As,Hemophilia A, Autosomal,Hemophilia As,Hemophilia As, Autosomal,Hemophilia As, Congenital,Hemophilias, Classic
D006470 Hemorrhage Bleeding or escape of blood from a vessel. Bleeding,Hemorrhages
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D015942 Factor VIIa Activated form of factor VII. Factor VIIa activates factor X in the extrinsic pathway of blood coagulation. Coagulation Factor VIIa,Factor VII, Activated,Blood Coagulation Factor VII, Activated,Factor 7A,Factor Seven A,Activated Factor VII,Factor VIIa, Coagulation
D016527 Drug Costs The amount that a health care institution or organization pays for its drugs. It is one component of the final price that is charged to the consumer (FEES, PHARMACEUTICAL or PRESCRIPTION FEES). Cost, Drug,Costs, Drug,Drug Cost
D019774 Blood Coagulation Factor Inhibitors Substances, usually endogenous, that act as inhibitors of blood coagulation. They may affect one or multiple enzymes throughout the process. As a group, they also inhibit enzymes involved in processes other than blood coagulation, such as those from the complement system, fibrinolytic enzyme system, blood cells, and bacteria. Blood Coagulation Factor Inhibitor,Inhibitor, Blood Coagulation Factor,Inhibitor, Coagulation Factor, Blood,Inhibitors, Blood Coagulation Factor,Inhibitors, Coagulation Factor, Blood

Related Publications

Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
April 2000, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
April 2008, Seminars in hematology,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
April 1993, Transfusion medicine reviews,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
August 2008, Clinical advances in hematology & oncology : H&O,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
August 1998, Transfusion clinique et biologique : journal de la Societe francaise de transfusion sanguine,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
March 2003, Journal of thrombosis and haemostasis : JTH,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
September 1994, American journal of hematology,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
January 2010, Journal of blood medicine,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
November 2009, Seminars in thrombosis and hemostasis,
Jennifer M Stephens, and Ashish V Joshi, and Michael Sumner, and Marc F Botteman
May 2004, Current opinion in hematology,
Copied contents to your clipboard!