Recombinant factor VIIa prophylaxis in a patient with severe congenital factor VII deficiency

Abstract

Use of recombinant factor VIIa (rFVIIa, NovoSeven in patients with congenital FVII deficiency has been reported for the prophylactic management of surgical bleeding and for the treatment of acute bleeding episodes. Because of its short half-life, the use of rFVIIa on a regular prophylactic regimen has not been routinely adopted. In this report, we describe our successful experience with rFVIIa prophylaxis in preventing recurrent target joint bleeding in a severely FVII-deficient adolescent.

Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect

“…In conclusion, our data support previous convincing clinical data (11)(12)(13)(14) published on the efficacy of prophylaxis in FVII deficiency, and provides evidence for a large volume of distribution of rFVIIa and the persistence of a clotting potential when FVII:C is no longer detectable. The rapid and substantial diffusion of rFVIIa towards the extravascular spaces would…”

“…Notwithstanding the unfavourable PK data reported (9, 10), rFVIIa has been successfully used for prophylaxis in patients with FVII deficiency (11)(12)(13)(14), both in young children and adults (14). Considering this apparent discrepancy, understanding the PK profile of rFVIIa in patients with FVII deficiency is of paramount importance.…”

Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)

“…[8][9][10][11] Clinical experience with prophylaxis in FVII deficiency has been limited to individual case reports, mostly not published in full. [12][13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29] The Seven Treatment Evaluation Registry (STER, www.targetseven.org) has collected information on prophylaxis from a worldwide series of well-characterized FVII-deficient patients. Here we report an analysis of prophylaxis in these patients, focusing…”

Newly diagnosed congenital factor VII deficiency and utilization of recombinant activated factor VII (NovoSeven®)

“…As mentioned, rFVIIa was originally developed as a bypassing agent for the treatment of bleeding in hemophilia A and hemophilia B patients with inhibitors to replacement factor VIII and factor IX 30. However, recent research has supported its use in numerous other hematological conditions 1–4,26.…”

“…However, recent research has supported its use in numerous other hematological conditions 1–4,26. Its safety and efficacy in congenital factor VII deficiency has been shown, and it has been used as prophylaxis for bleeding associated with surgery as well as used in the management of acute bleeding episodes 30. Despite this, exact dosing preoperatively has been questioned, because it appears that more than just factor VII levels play a role in bleeding 22–25…”