Biomaterial Database

Single-dose recombinant activated factor VII therapy in hemophilia patients with inhibitors. 2008

Gili Kenet, and Uri Martinowitz

The Israeli National Hemophilia Center, Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel-Hashomer, Israel. Gili.Kenet@sheba.health.gov.il

Recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) is well established as an effective hemostatic agent for the management of hemorrhage in hemophilia patients with inhibitors. Its use in prophylaxis is currently being investigated. On-demand treatment schedules usually involve multiple bolus doses of 90 to 120 microg/kg administered every 2 to 3 hours, but recent evidence from randomized controlled trials suggests that the use of higher single doses is equally safe and effective when used for the home treatment of hemarthroses. Consequently, the use of a single dose of 270 microg/kg rFVIIa for the treatment of bleeds in inhibitor patients has recently been approved by the European Medicines Agency (EMEA). Such high-dose regimens may overcome the rapid clearance rate observed in pediatric patients, and may be more convenient for patients with poor venous access. It also has been suggested that individually tailored single-dose therapies might be beneficial in selected groups of patients, although proper monitoring of such patients is advised. Further research should contribute towards more effective dose optimization of rFVIIa in hemophilic inhibitor patients.

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)
D004305	Dose-Response Relationship, Drug	The relationship between the dose of an administered drug and the response of the organism to the drug.	Dose Response Relationship, Drug,Dose-Response Relationships, Drug,Drug Dose-Response Relationship,Drug Dose-Response Relationships,Relationship, Drug Dose-Response,Relationships, Drug Dose-Response
D004334	Drug Administration Schedule	Time schedule for administration of a drug in order to achieve optimum effectiveness and convenience.	Administration Schedule, Drug,Administration Schedules, Drug,Drug Administration Schedules,Schedule, Drug Administration,Schedules, Drug Administration
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
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
D016032	Randomized Controlled Trials as Topic	Works about clinical trials that involve at least one test treatment and one control treatment, concurrent enrollment and follow-up of the test- and control-treated groups, and in which the treatments to be administered are selected by a random process, such as the use of a random-numbers table.	Clinical Trials, Randomized,Controlled Clinical Trials, Randomized,Trials, Randomized Clinical
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