Steps towards an effective treatment strategy in congenital factor VII deficiency.
Congenital factor VII (FVII) deficiency is a consequence of a genetic polymorphism that can produce a wide spectrum of disease severity. Mildly affected patients may experience increased bleeding after surgery, trauma or mucosal bleeding, while spontaneous and life-threatening bleeding occurs in patients who are severely affected. Replacement therapy is the mainstay of treatment for patients with FVII deficiency. This has traditionally been achieved using fresh frozen plasma (FFP), prothrombin complex concentrates (PCCs), or plasma-derived FVII concentrates. However, recombinant activated FVII is now widely used for therapy in these patients. As cases of FVII deficiency tend to be encountered infrequently in most centers, no consolidated evidence-based therapeutic regimens have evolved and the side effects of the available treatments have not been comprehensively evaluated. Consequently, an online registry, the Seven Treatment Evaluation Registry (STER) has been set up. This is a prospective study that aims to evaluate the efficacy and safety of the different therapeutic options with which FVII-deficient patients may be treated. Recruitment of patients into the study is currently underway.
Mariani
Guglielmo
G
Department of Medicine and Public Health, L'Aquila University Medical School, L'Aquila, Italy. gmariani@cc.univaq.it
Lapecorella
Mario
M
Dolce
Alberto
A
D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D016913:Blood Component Transfusion; D016063:Blood Loss, Surgical / Q000517:prevention & control; D005069:Evaluation Studies as Topic; D005168:Factor VII Deficiency / Q000145:classification / Q000150:complications / Q000628:therapy*; D015942:Factor VIIa / Q000627:therapeutic use*; D006801:Humans; D010949:Plasma; D012042:Registries
D001779:Blood Coagulation Factors; C025667:prothrombin complex concentrates; D015942:Factor VIIa