Turoctocog alfa in the treatment of individuals with hemophilia A: review of quality of life data collected in Phase III trials

Abstract

Hemophilia A is an X-linked recessive hereditary bleeding disorder resulting from a deficiency in coagulation factor VIII. Difficulties due to hemophilia and its management present challenges for patient's quality of life. Turoctocog alfa, a recombinant, B-domain truncated factor VIII, is a recent US FDA-and EMA-approved replacement therapy shown to be an effective and safe option for the treatment of individuals with hemophilia A. Data collected throughout two Phase 3, multinational, open-label, non-randomized, non-comparative trials demonstrated that individuals with hemophilia A, particularly young adults experienced improvements in healthrelated quality of life when switched from an on-demand to a prophylactic regimen of turoctocog alfa.

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

“…Consistent with the substantial efficacy and tolerability previously reported in children treated with emicizumab prophylaxis from HAVEN 2, 30 improvements in HRQoL for children were observed as early as the first scheduled time point of Week 13 and were sustained throughout the treatment period across multiple domains of both the Haemo‐QoL SF II and Adapted Inhib‐QoL questionnaires. Baseline scores for ‘Physical Health’ in this population were similar to those reported in other studies using the Haemo‐QoL SF II to assess HRQoL in children with HA with FVIII inhibitors receiving episodic or prophylactic aPCC or and rFVIIa 19,35,36 . Furthermore, the domains ‘Family’ and ‘Sports & School’ were the most impacted domains for those receiving both episodic or prophylactic FVIII, 20,35,36 as was the case for HAVEN 2 participants at baseline 31…”

“…34 As such, emi- Baseline scores for 'Physical Health' in this population were similar to those reported in other studies using the Haemo-QoL SF II to assess HRQoL in children with HA with FVIII inhibitors receiving episodic or prophylactic aPCC or and rFVIIa. 19,35,36 Furthermore, the domains 'Family' and 'Sports & School' were the most impacted domains for those receiving both episodic or prophylactic FVIII, 20,35,36 as was the case for HAVEN 2 participants at baseline. 31 Following prophylaxis with emicizumab, improvements in do-…”