Pediatric Factor VII Deficiency Treatment & Management
- Author: Helge Hartung, MD; Chief Editor: Max J Coppes, MD, PhD, MBA more...
Medical Care
Acute bleeds: Management of acute hemorrhage primarily consists of factor VII (FVII) replacement therapy to treat bleeding. levels of more than 10% are usually hemostatic, although higher levels may be advisable in the event of a severe bleeding episode. Because factor VII has a short half-life (3-4 h), repeat treatment may be necessary in all except minor bleeding episodes. Treatment alternatives include the following:
- Fresh frozen plasma is the least effective because of the volume required to provide adequate factor VII replacement. No viral attenuation of this product means that a risk of viral transmission is present.
- Prothrombin complex concentrates contain factors II, IX, and X in addition to factor VII. These concentrates have undergone viral attenuation during manufacturing. Determining the appropriate dosage for treatment of factor VII deficiency can be difficult. These agents carry a risk of thrombogenic complications, particularly with repeated administration.
- Factor VII concentrates are purified plasma–derived preparations that have undergone a vapor-heat viral-inactivation process. If available, factor VII concentrates are preferred over untreated plasma.[4] When given at high doses, these concentrates carry a risk of thrombosis, likely because of other vitamin K-dependent factors that are present in significant concentrations.
- Recombinant activated factor VII (rFVIIa) was originally developed to treat patients with hemophilia and inhibitors, but it can be used at lower doses for patients with congenital factor VII deficiency. With increasing experience and evaluation of rFVIIa for treatment and prophylaxis in factor VII deficiency, the benefits and safety profile in this setting are becoming clearer. Arterial thromboembolic events, a concern in adult patients treated with high doses of rFVIIa,[5] have not been found at an increased rate.
Prophylaxis: The decision to embark on a program of prophylaxis is determined by the patient's clinical presentation and the number of clinically significant bleeding episodes requiring intervention. Consider prophylaxis for patients with recurrent hemarthrosis or intracranial hemorrhage. Beneficial results have been reported with regimens that vary from twice daily to twice weekly treatment.[6]
Surgical Care
Maintaining factor VII levels of at least 15-25% provides adequate hemostasis levels for most surgical procedures.
Preoperative factor VII replacement and monitoring of factor VII levels is essential for major surgical interventions.
Because of the short half-life (3-4 h), replacement therapy should continue postoperatively; the period of therapy is determined by the nature and extent of the procedure.
Consultations
Consult a hematologist and/or hemostasis specialist for patients who require factor VII replacement therapy.
Genetic counseling and family studies are part of a complete evaluation.
Activity
In patients with severe factor VII deficiency and a history of clinical bleeding, consider the risk of bleeding when choosing activities.
Individuals should stay fit because good muscle strength protects joints.
Patients are encouraged to avoid contact sports, wear appropriate protective gear, and choose activities, such as swimming, that promote muscle strength and flexibility with a low risk of joint injury.
Mandhyan R, Tiwari A, Cherian G. Congenital factor VII deficiency. Br J Anaesth. Feb 2010;104(2):267-8. [Medline].
Mariani G, Herrmann FH, Dolce A, et al. Clinical phenotypes and factor VII genotype in congenital factor VII deficiency. Thromb Haemost. Mar 2005;93(3):481-7. [Medline].
Peyvandi F, Mannucci PM. Rare coagulation disorders. Thromb Haemost. Oct 1999;82(4):1207-14. [Medline].
Mariani G, Bernardi F. Factor VII Deficiency. Semin Thromb Hemost. Jun 2009;35(4):400-6. [Medline].
Levi M, Levy JH, Andersen HF, Truloff D. Safety of recombinant activated factor VII in randomized clinical trials. N Engl J Med. Nov 4 2010;363(19):1791-800. [Medline].
Todd T, J Perry D. A review of long-term prophylaxis in the rare inherited coagulation factor deficiencies. Haemophilia. Nov 11 2009;[Medline].
Di Paola J, Nugent D, Young G. Current therapy for rare factor deficiencies. Haemophilia. Jan 2001;7 Suppl 1:16-22. [Medline].
Giansily-Blaizot M, Schved JF. Potential predictors of bleeding risk in inherited factorVII deficiency. Clinical, biological and molecular criteria. Thromb Haemost. Nov 2005;94(5):901-6. [Medline].
Mariani G, Dolce A, Marchetti G, Bernardi F. Clinical picture and management of congenital factor VII deficiency. Haemophilia. Oct 2004;10 Suppl 4:180-3. [Medline].
Mariani G, Lapecorella M, Dolce A. Steps towards an effective treatment strategy in congenital factor VII deficiency. Semin Hematol. Jan 2006;43(1 Suppl 1):S42-7. [Medline].
Perry DJ. Factor VII Deficiency. Br J Haematol. Sep 2002;118(3):689-700. [Medline].
Tuddenham EG, Pemberton S, Cooper DN. Inherited factor VII deficiency: genetics and molecular pathology. Thromb Haemost. Jul 1995;74(1):313-21. [Medline].
Print
