<p>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</p>

Abstract

Background Prophylactic treatment regimens lead to improvements in health-related quality-of-life (HRQoL) among individuals with hemophilia. 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 (TS). Aim To investigate the HRQoL and TS of patients with severe hemophilia A from two phase III trials evaluating the safety and efficacy of N8-GP. Methods HRQoL was assessed using the Haemo-QoL (reported by children and their parents) and Haem-A-QoL (reported by adults). TS was assessed using Hemo-Sat. Domain and total scores for all questionnaires ranged from 0 to 100, with lower scores indicating a better HRQoL or TS. A negative change in score indicates an improvement in HRQoL/TS. Results Mean changes in HRQoL scores were reported for 14 children aged 4–7 years, 21 children aged 8–11 years, 10 adolescents aged 13–16 years, and 163 adults (17 years and above). Mean changes in children/adolescents-reported Haemo-QoL total score were -14.0 for ages 4–7 years, -3.6 for ages 8–11 years, and -0.1 for ages 13–16 years. Mean changes in parent-reported Haemo-QoL total scores were -11.5 for 4–7 years, -8.6 for ages 8–11 years, and -4.0 for 13–16 years. Adults’ mean change in Haem-A-QoL total score was -3.1 for those receiving on-demand treatment and -2.3 for those receiving prophylaxis treatment. High levels of TS with N8-GP were reported by parents of children/adolescents and the adults at the end of the trial. Conclusion While most patients reported a relatively good baseline HRQoL when entering the respective trials, the HRQoL of patients was either maintained or further improved when treated with N8-GP. Adults and parents of children and adolescents reported a high level of treatment satisfaction with N8-GP.

Measuring the impact of changing from standard half‐life (SHL) to extended half‐life (EHL) FVIII prophylaxis on health‐related quality of life (HRQoL) in boys with moderate/severe haemophilia A: Lessons learned with the CHO‐KLAT tool

“…Others have also shown significant improvements in HRQoL when boys with high rates of target joints and/or high ABRs switch to an EHL FVIII. Kearney and colleagues showed that boys (n = 68) and their parents reported improvements in most domains of the Haemo‐QoL (a Haemophilia‐specific HRQoL questionnaire) when switching from a SHL FVIII to a glycopegylated EHL FVIII (N8‐GP; Novo Nordisk, Bagsvaerd , Denmark) as part of the Pathfinder‐5 study . However, a considerable proportion (15/68; 22%) of the boys in the study had target joints at baseline prior to switching to the EHL FVIII.…”

Long‐term safety and efficacy of N8‐GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5

“…During the extension phase, PRO questionnaires were collected annually. Detailed methods of the questionnaires used and main phase results have been previously published 5 . In short, aged‐based versions of the Haemo‐QoL questionnaires were distributed to patients in the age groups 4‐7 years (Haemo‐QoL I) and 8‐12 years (Haemo‐QoL II) and their parents.…”

“…The pathfinder5 trial comprised a main phase and an extension phase. Results from the main phase, which encompassed 50 exposure days (EDs) of twice‐weekly 60 IU/kg N8‐GP treatment, showed a median annualized bleeding rate (ABR) of 1.95, with 42.6% of patients experiencing no bleeding episodes, 4 maintenance or improvement in health‐related quality of life (QoL) measure, 5 and good tolerability 4 . Following completion of the main phase, patients could continue prophylactic treatment with N8‐GP in an extension phase, totaling up to 5.5 years of treatment.…”

Health‐related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors—Results from the HAVEN 2 study

“…Haemophilia A (HA), a congenital bleeding disorder characterized by deficiency of coagulation protein factor (F)VIII, has a negative impact on the health-related quality of life (HRQoL) of affected people. [1][2][3] A clinical hallmark of severe HA is recurrent spontaneous bleeds, particularly into joints, and a substantial corresponding impact on physical health and HRQoL. [4][5][6][7][8] Approximately 30% of previously untreated persons with haemophilia A (PwHA) develop one of the most challenging complications of haemophilia treatment: neutralizing alloantibodies (inhibitors) against FVIII, [9][10][11] preventing effective FVIII prophylaxis.…”