Efficacy and safety
Reliable efficacy when you and your patients need it most1–3
|Prophylactic treatment in PTPs|
|of responses were rated as excellent or effective (n=47)1,*|
|Prophylactic treatment in PUPs|
|of responses were rated as excellent† (n=42), indicating that spontaneous musculoskeletal bleeding was prevented2|
|of patients under 6 years old experienced no spontaneous bleeding episodes whilst on prophylaxis (n=22)5|
*The response to secondary prophylaxis was assessed once every 3 months by the investigator using a 3-point scale: excellent (no spontaneous bleeding of any kind, no change in dosing regimen necessary); effective (no spontaneous musculoskeletal bleeding, no change in dosing regimen necessary); or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).1
†Rated by the investigator at 3-month intervals on a 3-point scale: excellent (prophylaxis treatment completely prevented spontaneous musculoskeletal bleeding, no change in dosing regimen necessary); effective (adequately prevented spontaneous musculoskeletal bleeding episodes as demonstrated by a lower than expected incidence of spontaneous musculoskeletal bleeding episodes); or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).2
|On-demand treatment in PTPs|
|of 1,796 bleeding episodes were resolved with only 1 infusion (n=55)1|
|On-demand treatment in PUPs|
|of 997 bleeding episodes were resolved with only 1 infusion (n=54)2|
|of responses were rated excellent or good‡ (n=28)3,§|
‡ Haemostasis was rated as excellent or good in 34 out of 35 of the operative procedures. Response ratings are defined as follows: excellent if the response was as satisfactory, with as much and as rapid an improvement as the best responses with other factor IX products for similar bleeds or procedures; good if the response was as satisfactory, with as much and as rapid an improvement as most responses with other factor IX products for similar bleeds or procedures; moderate if the response was less than satisfactory and not as good as most responses seen with other factor IX products when used for similar bleeding episodes or similar procedures; or no response if no improvement at all was observed.3
§ 29% (8/28) of patients (9 operative procedures) received postoperative BeneFIX® by continuous infusion.3 BeneFIX® is not licensed for use by continuous infusion – please refer to the BeneFIX® Summary of Product Characteristics for further information.4
A favourable safety profile
The most common adverse reactions reported in clinical trials of PTPs and identified in post-marketing use were4:
- Headache, cough, pyrexia – very common (≥10%)
- Hypersensitivity, dizziness, dysgeusia, phlebitis, flushing, vomiting, nausea, rash, urticaria, chest discomfort, infusion-site reaction, infusion-site pain – common (≥1% to <10%)
Low inhibitor formation
Data from more than 11,000 infusions of BeneFIX® demonstrate that inhibitor development and allergic reactions were low6,7:
- Results of pooled safety data from 6 prospective studies showed that 1.2% of patients developed inhibitors on BeneFIX® (5/412)6
- 4 of these patients also exhibited an allergic-type manifestation
- Results from a retrospective study suggest that there is no difference in the frequency of allergic reactions or inhibitor development in patients receiving BeneFIX® versus those receiving plasma-derived factor IX concentrates7
1. Roth DA et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001;98(13):3600–3606.
2. Shapiro AD et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood 2005;105(2):518–525.
3. Ragni MV et al. Use of recombinant factor IX in subjects with haemophilia B undergoing surgery. Haemophilia 2002;8(2):91–97.
4. Benefix® (nonacog alfa) Summary of Product Characteristics BF_16 : Accessed October 2020.
5. Monahan PE et al. Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 2010;16(3):460–468.
6. Rendo P et al. Nonacog alfa: an analysis of safety data from six prospective clinical studies in different patient populations with haemophilia B treated with different therapeutic modalities. Blood Coagul Fibrinolysis 2015;26(8):912–918.
7 Recht M et al. A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B. Haemophilia 2011;17(3):494–499.
PTP = previously treated patient; PUP = previously untreated patient.
PP-BEN-GBR-0418. October 2020