Researchers have found that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A.
The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.
Several factors can affect the quality of life and satisfaction of patients with hemophilia A, including the development of inhibitors to FVIII (the most significant treatment complication), problems in venous access, and pain at injection site. Importantly, the increase in half-life (an indicator of how long the medication remains in the blood) of recent FVIII replacement therapies enables less-frequent dosing, which could help reduce complications.
Researchers analyzed health-related quality of life (HRQOL) in children with severe hemophilia A, who were treated with N8-GP (turoctocog alfa pegol), a novel recombinant FVIII product developed by Denmark-based Novo Nordisk.
Participants had taken part in the global Phase 3 clinical trial pathfinder 5 (NCT01731600).
A total of 68 children received N8-GP (60 IU/kg) twice weekly for 26 weeks. Participants were aged 0-12 years (20 aged 0-3, 23 aged 4-7, and 25 aged 8-12).
Children and their parents evaluated HRQOL using age-specific versions of the Hemophilia-Quality of Life (HAEMO-QOL) questionnaire. Conversely, parents evaluated satisfaction with the Hemophilia Satisfaction questionnaire (HEMO-SAT). Both questionnaires use a 0-100 scale, with a lower score indicating greater HRQOL or treatment satisfaction. Researchers analyzed results only from individuals who filed the questionnaires before and after the 26-week treatment period.
Children aged 4-7 and their parents reported a decrease in individual items and total score of HAEMO-QOL-I (thus reflecting an improvement) with N8-GP treatment. Similar improvements were reported by children aged 8-12 and their parents.
Results of HEMO-SAT showed low scores at baseline and at the end of treatment, indicating high level of satisfaction with the medication.
“Overall, children with severe hemophilia A who were treated with turoctocog alfa pegol [N8-GP] consistently reported improvement in HRQOL and maintained a high level of satisfaction by the end of the 26-week main phase of the trial,” the authors wrote.
Hemophilia A is a genetic bleeding disease caused by missing or defective factor VIII (FVIII), a clotting protein. Hemophilia affects about 20,000 people in the U.S. and is the most common type of the disease. Approximately 60 percent of patients have the severe form of hemophilia A.