Treatment with turoctocog alfa pegol (Esperoct, Novo Nordisk) stabilized or improved health-related quality of life (HRQoL) in patients of all ages with hemophilia A, according to a study published in Patient Preference and Adherence.
 
Turoctocog alfa pegol, also known as N8-GP, is a new recombinant extended half-life factor VIII replacement treatment that was approved by the FDA in February 2019 for adults and children with hemophilia A. It is indicated for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment, and control of bleeding episodes and perioperative management of bleeding.
 
In previous clinical trials, treatment with turoctocog alfa pegol demonstrated effective routine prophylaxis in patients through a simple, fixed dosing regimen of 1 injection every 4 days in adults and adolescents or every 3 to 4 days in children. Turoctocog alfa pegol has been evaluated across 5 prospective, multi-center clinical trials in previously-treated patients with severe hemophilia A and no history of inhibitors.
 
For the study, the researchers investigated HRQoL and treatment satisfaction (TS) in patients with severe hemophilia A from 2 phase 3 trials evaluating the safety and efficacy of turoctocog alfa pegol. Changes in HRQoL scores were measured for 14 children aged 4 to 7 years, 21 children aged 8 to 11 years, 10 adolescents aged 13 to 16 years, and 163 adults. When entering the clinical trials, most patients reported a relatively good baseline HRQoL.
 
“Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction,” the researchers wrote in the study.
 
The data showed that children/adolescent-reported mean changes on the hemophilia-specific quality of life index (Haemo-QoL) total score were:
  • -14.0 for ages 4 to 7 years.
  • -3.6 for ages 8 to 11 years.
  • -0.1 for ages 13 to 16 years. 
Mean changes in parent-reported Haemo-QoL total scores were:
  • -11.5 for ages 4 to 7 years.
  • -8.6 for ages 8 to 11 years
  • -4.0 for ages 13 to 16 years.
Overall, adults’ mean change in the Haemo-QoL total score was -3.1 for those receiving on-demand treatment and -2.3 for those receiving prophylaxis treatment, according to the study. Both parents of children/adolescents and adults also reported higher levels of TS by the end of the trial period.
 
Reference
 
Kearney S, Raffini LJ, Pham TP, et al. Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII. Patient Preference and Adherence. 2019. Doi: https://doi.org/10.2147/PPA.S196103