Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: post hoc analysis of phase III trials using patient-reported outcomes

Abstract

Background: Pain is a common symptom of hemophilia that may adversely affect patients’ quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. Objective: To describe in depth the evolution of QoL, pain- and activity-related domains and questions for pediatric, adolescent, and adult patients with hemophilia B treated with rFIXFc prophylaxis. Design: A post hoc analysis of data from a series of clinical trials. Methods: This post hoc, long-term analysis assessed patient-reported outcomes (PROs) from the Kids B-LONG (NCT01440946: pediatric) and B-LONG (NCT01027364: adults and adolescents) parent studies and the B-YOND (NCT01425723: all age groups) extension study. Results: Ninety-two adult and adolescent patients that started in the B-LONG study were assessed, with a median (range) duration of follow-up of 58.9 (0.0–78.4) months. The Haem-A-QoL total score was significantly reduced from baseline by 4.45 ( p ⩽ 0.01), as were the subdomains ‘physical health’ (9.10; p = 0.001), ‘sports and leisure’ (11.25; p ⩽ 0.01), ‘treatment’ (2.69; p = 0.05), and ‘view of self’ (5.81; p = 0.002). Thirty pediatric patients that started in the Kids B-LONG study were assessed, with a median (min–max) duration of follow-up of 36.7 (9.0–59.9) months. The high level of satisfaction demonstrated by the PROs at baseline was maintained. Conclusion: rFIXFc prophylaxis reduced perceived pain and increased levels of physical activity with sustained, long-term improvements in QoL in adult and adolescent patients with hemophilia B and maintained high QoL scores in pediatric patients.

Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes

“…In that study, significant and sustained improvements were observed in the overall Haem-A-QoL score, as well as the subdomains of ‘ physical health ’, ‘ sports and leisure ’, ‘ treatment ’, and ‘ view of self ’. 29 Similarly, a HRQoL study of patients with hemophilia A treated with turoctocog alfa pegol for 6–27 months reported improvements from baseline in both Haem-A-QoL domains ‘ physical health ’ and ‘ feeling ’, and improvements in the domain of ‘ sport ’ in patients previously treated on-demand. 30 These observations are consistent with documented benefits of extended half-life (EHL) factor treatments, namely higher trough levels for clotting factors in patients, resulting in reduced risk of both clinically evident and subclinical bleeds.…”

“… 12 , 18 – 20 The Haem-A-QoL improvements observed reflect those of a study by Astermark et al . 29 into the long-term effects of recombinant factor IX Fc fusion protein in adults and adolescents with hemophilia B. In that study, significant and sustained improvements were observed in the overall Haem-A-QoL score, as well as the subdomains of ‘ physical health ’, ‘ sports and leisure ’, ‘ treatment ’, and ‘ view of self ’.…”