Turoctocog Alfa Pegol and Its Impact on Health-Related Quality of Life
The presented work is based on the results of studies that evaluated how the administration of turoctocog alfa pegol affects the quality of life in individuals with severe hemophilia A. The obtained data also reflect the level of patient satisfaction with this treatment.
EHL as a Path to More Comfortable Prophylaxis for Hemophilia A
The standard of care for individuals with severe hemophilia A involves the administration of coagulation factor VIII (FVIII) concentrate, either during bleeding episodes or regularly as prophylaxis to prevent bleeding. Standardly used FVIII concentrates are so-called standard half-life (SHL) factors. In recent years, however, extended half-life (EHL) factors have also entered clinical practice, enabling a reduction in the number of injections required for prophylactic therapy while simultaneously improving the level of protection by increasing the minimum achievable (trough) FVIII levels.
Analyzed Data
One representative of EHL is turoctocog alfa pegol (N8-GP). Recently, research was conducted on its impact on the quality of life of hemophilia patients and their satisfaction with this form of therapy. The results of the analysis, summarized below, were published in the journal Patient Preference and Adherence.
The work is based on data obtained from two phase III studies that evaluated the safety and efficacy of N8-GP. Health-related quality of life (HRQoL) was examined using the Haemo-QoL questionnaire (for children and their parents) or Haem-A-QoL (for adults), and treatment satisfaction (TS) with the Hemo-Sat questionnaire.
Results
Average changes in HRQoL scores were examined in children and adults across four age groups: 4–7 years (n = 14), 8–11 years (n = 21), 13–16 years (n = 10), and ≥ 17 years (n = 163):
- Average changes in Haemo-QoL score values were –14.0 in the 4–7 years category, –3.6 in the 8–11 years category, and –0.1 in the 13–16 years category, where a higher negative value indicates more significant improvement.
- Average changes in Haemo-QoL score values reported by parents were –11.5 in the 4–7 years category, –8.6 in the 8–11 years category, and –4.0 in the 13–16 years category.
- Average changes in Haem-A-QoL score for 17 years and older patients were –3.1 in the subgroup treated during bleeding only and –2.3 in the subgroup using prophylaxis.
Both the parents of children and adult patients reported a high level of satisfaction with the treatment using N8-GP.
Conclusion
Most patients reported relatively good health-related quality of life at the beginning of the study. During the use of N8-GP, the quality of life was maintained or improved. Adult hemophilia patients and the parents of children and adolescents reported a high level of satisfaction with N8-GP therapy. This therapy led to good outcomes in the assessment of hemophilia-specific HRQoL in children, adolescents, and adults with severe hemophilia A.
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Source: Kearney S., Raffini L. J., Pham T. P. 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 Prefer Adherence 2019; 13: 497–513, doi: 10.2147/PPA.S196103.
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