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The efficacy and safety of recombinant factor VIIA (rFVIIa) in adult patients: the experience of the hemophilia and thrombosis research society (HTRS) registry (2004–2008)
Abstract number: PP-MO-598
Kessler1 C.M., Cooper2 D.L., Carr2 M.E.
11Lombardi Cancer Center, Georgetown University Medical Center, Washington 22Clinical, Medical and Regulatory Affairs, Novo Nordisk Inc., Princeton, US
How-to-cite Kessler CM, Cooper DL, Carr ME. The efficacy and safety of recombinant factor VIIA (rFVIIa) in adult patients: the experience of the hemophilia and thrombosis research society (HTRS) registry (2004–2008). Journal of Thrombosis and Haemostasis 2009; Volume 7, Supplement 2: Abstract PP-MO-598
Background: For persons with congenital hemophilia (PWH) complicated by alloantibody inhibitors (CHwI), the advent of bypassing agents, such as rFVIIa, has allowed for effective treatment of acute bleeding episodes. Few adverse events have been reported with rFVIIa use in PWH; however, most of the experience has been accrued in children. This study examines the efficacy and safety of rFVIIa for older PWH within the HTRS registry database.
Methods: Data from 2,041 rFVIIa-treated bleeds in the HTRS registry (January 2004–November 2008) were analyzed to identify bleeds in adults (age >18 years) with CHwI.
Results: Of those 429 CHwI patients in the registry with bleeding episodes, 145 (CHA:132, CHB:13) were > 18 years of age (median 32 years; range 19–83 years). The majority were non-Hispanic whites (68%) and non-Hispanic blacks (21%). Median highest human anti-FVIII titer was 69 BU (range 0.5–4800 BU). Patient reported functional status was unrestricted in only 26%. 329 bleeding episodes were treated with bolus rFVIIa (first line treatment:307 episodes, second line:22 episodes). Most bleeds were spontaneous (69%) or traumatic (12%) and occurred commonly in joints (65%) and muscles (14%). Median (range) total rFVIIa dose per bleeding episode (one or more injections), was 270 mcg/kg (46–21392 mcg/kg), with a median 3 injections (1–234) of 90 mcg/kg (46–400 mcg/kg) over 1 day (1–36 days). This included 89 bleeds treated with a single dose of rFVIIa. For the majority of episodes, bleeding stopped (89%) or slowed (9%) with no rebleeds reported within 48 h. Overall, there was one serious adverse event (bruising/pain at port-a-cath placement site) and no thromboembolic events.
Conclusion: As treatment options for PWH with inhibitors expand, the proportion of older patients who may be at greater risk for adverse events increases. The HTRS data support the efficacy and safety of rFVIIa in adult PWH. Thus far, the thrombogenic risks that have been observed for rFVIIa use in elderly patients with normal coagulation, have not been realized in adult PWH with inhibitors. Longitudinal surveillance in the aging hemophilia population is indicated.
Disclosure of interest: C. Kessler, Novo Nordisk:Research/Consultant.
D. Cooper, Novo Nordisk:Employee.
M. Carr, Novo Nordisk:Employee.
To cite this abstract use the following format:
Journal of Thrombosis and Haemostasis 2007; Volume 5, Supplement 2: abstract number
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