What is the role of recombinant activated factor VII (rFVIIa) in the treatment of hemophilia?

Updated: Feb 05, 2020
  • Author: Bishnu Prasad Devkota, MD, MHI, FRCS(Edin), FRCS(Glasg), FACP; Chief Editor: Eric B Staros, MD  more...
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Recombinant FVIIa has also been used to control severe obstetrical bleeding in women without hemophilia. [6] In that series, bleeding was diminished or arrested in over 80% of patients with no complications due to rFVIIa. However, Lewis et al mention that rFVIIa administration will not be effective if plasma fibrinogen is depleted, particularly if the levels are decreased to 50 mg/dL or less. [7]

A concern in rFVIIa use is the specter of thrombosis. [5] Thrombotic events may affect as many as 7% of treated patients but appear so far to be uncommon in obstetric patients. [8]  (Using Mendelian randomization analysis, de Vries et al found evidence that factor VII activity can be tied to the etiology of ischemic stroke. [9] )

Evidence differs, however, regarding the likelihood of thrombosis. A retrospective study by Rajpurkar et al indicated that in the FDA-approved indications for rFVIIa, the risk of thrombotic events is lower than that stated above. For congenital hemophilia with inhibitors, Glanzmann thrombasthenia with refractoriness to platelets, congenital factor VII deficiency, and acquired hemophilia the risk was, respectively, 0.11%, 0.19%, 0.82%, and 1.77%. Risk factors for thrombotic events in rFVIIa use were found to include age of 65 years or above, as well as concomitant cardiac or vascular disease and activated prothrombin complex concentrate use. [10]

An intranasal form of desmopressin is also an option for use in hemophilia and is more frequently used at home than the subcutaneous route. A single spray in a single nostril (150 micrograms total dose) is adequate for children more than 5 years old (< 50 kg). A single spray in each nostril is used (300 micrograms total dose) for adolescents (>50 kg) and adults. This dose of intranasal desmopressin increases the factor VIII level by 2-3 times. This therapy can be repeated every 8-12 hours; however, with repetitive use, the patient's stores of factor VIII will be depleted, and, subsequently, the effect will be reduced significantly. Desmopressin is an antidiuretic agent, and fluid restriction may be needed during use. [11]

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