The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B

Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B

“…Hypersensitivity reactions have been reported for all FIX products and have manifested as pruritus, rash, urticaria, hives, facial swelling, dizziness, hypotension, nausea, chest discomfort, cough, dyspnea, wheezing, flushing, generalized discomfort, and fatigue. 24,25 Therefore, the event observed in the present trial was not unexpected. In a trial including 56 previously treated patients, Roth et al reported 4 episodes in 4 patients with signs or symptoms of allergic reactions to rFIX.…”

Haemophilia in the first years of life

“…Older anecdotal publications suggest that the significant morbidity and mortality associated with high risk surgery may increase substantially for children who are co-afflicted with haemophilia, due to either the lack of diagnosis and/or inadequate factor replacement. Conversely, pre-and postlicensure studies of recombinant and plasma-derived factor VIII and IX concentrates, as well as the more recent anecdotal surgical literature, document that once the diagnosis is made and an appropriate prophylactic replacement strategy is implemented, complex surgery can be performed safely with a minimally increased risk of haemorrhage [3][4][5][6][7][8][9]. Minimum effective factor dosing also limits surgical haemorrhage in the developing world where resources are limited [10].…”

“…In the PUP retrospective analysis, the adjusted surgery-related RR was 2.4 (CI 1.2-4.8) for all inhibitors and 4.5 (2-10.1) for high titre antibodies [19]. Pre-and postlicensure PUP studies receiving recombinant or plasma-derived factor IX (FIX) have reported a low inhibitor risk with surgery [7,8].…”

Design and characterization of an APC-specific serpin for the treatment of hemophilia

“…The mainstream treatment of hemophilia consists of replacing the affected factor on demand when bleeds occur or by using prophylaxis. [8][9][10][11][12][13] Prophylactic treatment is not completely effective and reduces only the frequency of bleeds. Neither treatment regimen prevents hemophilic arthropathy (extravasation of blood into the joints), a major cause of morbidity associated with hemophilia.…”