OBJECTIVES: Turoctocog alfa pegol (N8-GP) is an extended half-life factor VIII molecule for use in adults and children with haemophilia A (HA) for routine prophylaxis, on-demand treatment including control of bleeding episodes, and perioperative management. This study aims to understand the real-world experience of patients with HA receiving N8-GP.

METHODS: A quantitative online survey was conducted in Germany, Italy, Portugal, Spain, the UK and the US, in patients (≥16 years) with HA and currently treated with N8-GP for >3 (Portugal) or >6 months (other countries), and carers (≥18 years) of adolescents (12-17 years) meeting the same criteria. Patients were recruited by recruitment panels, referral from healthcare professionals treating HA, and patient associations. The survey comprised of questions regarding current and previous treatment perceptions across a number of metrics including satisfaction, convenience, impact on quality of life (QoL), and activities of daily living.

RESULTS: In an interim analysis of 56 patients (98% male; mean age: 30 years; severity: 23% mild, 41% moderate, 36% severe), comprising surveys from 43 patients and 13 carers, the mean time on N8-GP was 0.8 years. The proportion of patients satisfied with their treatment increased from 46% with previous management strategies to 96% after switching to N8-GP. Of the patients who previously received another treatment, 92% preferred N8-GP. Unprompted reasons for preferring N8-GP included more convenient treatment (lower frequency of administration, easier to store and use), improved treatment characteristics (efficacious, safe, and longer half-life), and improved QoL. N8-GP had a positive long-term impact on many aspects of patients’ lives, such as positive effects on their independence to live like a person without HA (63% of patients).

CONCLUSIONS: The advantages of lower frequency of injections, strong efficacy, and improved QoL offered by N8-GP drive patient satisfaction and preference for it over previous treatments among patients with HA.