Patient-reported Outcomes

Enabling your patients to make the most of their lives^1-7

•  Efficacy that is established for prophylaxis, on-demand and surgery^1-4
• A favourable safety profile with low inhibitor formation^5-7,*
• Dosing flexibility through the broad range of doses (250, 500, 1000, 2000 and 3000 IU) enables FIX coverage for your patients in their everyday life⁵
• Convenient storage options: Storage flexibility through room temperature storage (<30 ⁰C). Do not freeze⁵

Joint bleeds are associated with impairment in day-to-day activities⁸

Patients with hemophilia commonly experience recurrent bleeding into joints that may cause pain and arthropathy that can impact their quality of life.⁸

Children With Hemophilia and Sport

Prophylaxis in paediatric patients supports an active lifestyle, which may reduce the bleed frequency and improve the health-related quality of life.¹⁰

A cross-sectional, multisite, study of boys aged 6 to 17 years (n=84) with hemophilia A or B^§ that assessed physical performance, sporting activity and health-related quality of life found the following^10,¶:

Bleeding has a variable impact on the lives of adolescent boys with hemophilia, but is most obvious in those who play sport and who have to reduced or limit their participation because of bleeding. These boys recognized that prophylaxis offered them protection from bleeding, the older and more sporty boys understood the need for tailored prophylaxis around 'risk' activities such as sport or events away from home.¹⁰

Encouraging haemophilic boys to participate in sport has a direct impact on their overall health-related quality of life.¹⁰

*Results of pooled safety data from 6 prospective studies encompassing more than 11,000 infusions of [BeneFIX^®] showed that 1.2% of patients experienced inhibitors on BeneFIX^® (5/412). This was considered low by study investigators.^7
†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
§Of any severity, current or past inhibitor.
^¶Health-related quality of life was assessed using age-appropriate questionnaires including KINDL, Haemo-QoL and HEP-Test-Q.

FIX, factor IX; Haemo-QoL, Hemophilia-Specific Quality of Life; HEP-Test-Q, Hemophilia & Exercise Project-Test-Questionnaire; IU, international unit; KINDL, German questionnaire for measuring quality of life in children and adolescents; PTP, previously treated patient; PUP, previously untreated patient.