Illustrative Cases from the Pathfinder Clinical Trials of Patients with Hemophilia A Treated with Turoctocog Alfa Pegol (N8-GP)

Klamroth, Robert; Hampton, K. K.; Trakymienė, Sonata Šaulytė; Korsholm, Lars; Carção, Manuel

doi:10.2147/ppa.s326282

Cited by 1 publication

(1 citation statement)

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“…The major challenges for patients with HA are their inability to undertake different physical activities and the perceived burden of the disease. Though previous illustrative cases from the pathfinder clinical trials demonstrated improved physical activity levels, bleeding rates and treatment adherence, 16 there are still unmet needs for the patients, which was also evident from this study. Less than half (44%, n=22) acknowledged that N8-GP had an impact on reducing pain (Figure S3A) and some economic factors such as days missed at school/college (31%, n=8) and ability to work in more demanding roles (19%, n=5; Figure S3B).…”

Section: Challenges and Unmet Needs Of Patientsmentioning

confidence: 57%

Real-World Experience of People with Hemophilia A Receiving Turoctocog Alfa Pegol (N8-GP): Results from a Patient Experience Survey

Eichler,

Nagao,

Waller

et al. 2023

PPA

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Turoctocog alfa pegol (N8-GP) is an extended half-life recombinant factor VIII molecule used for the treatment of hemophilia A (HA). The purpose of this study was to investigate real-world experiences of patients with HA treated with N8-GP. Patients and Methods: A 25-minute online survey was completed by adults (≥18 years) and caregivers of adolescents (12-16 years) with HA receiving N8-GP across six countries (Germany, Italy, Portugal, Spain, UK and US). Patients were recruited using a multichannel approach through recruitment panels, referrals from healthcare professionals and patient associations. The survey comprised a questionnaire with metrics including satisfaction and preferences for N8-GP, quality of life (QoL) and long-term impact. Results: A total of 62 participants (98% male [n=61], mean age 29 years) comprising 46 patients and 16 caregivers completed the survey. Patients (60% non-severe [n=37] and 40% severe [25]) were on N8-GP for a mean period of 1.4 years. Patients expressed satisfaction (95% vs 42%, p<0.001) and preference (91% vs 9%, p<0.001) for N8-GP vs their previous treatments. Most patients with severe HA (87%, p=0.038) and patients on prophylaxis (84%, p<0.001) stated lower frequency of injections as their main reason for satisfaction, while improved QoL drove satisfaction for non-severe patients (81%, p=0.053). Overall, patients perceived that QoL score improved (74.8 vs 65.9, p=0.01) with N8-GP treatment compared with previous treatments. Flexibility to store at room temperature was one of the key convenience factors driving satisfaction. Patients believed that N8-GP can offer a long-term impact in areas such as ability to perform day-to-day activities (68%), independence to live like a person without hemophilia (63%), ability to travel (60%) with a feeling of optimism and hopefulness (82%). Conclusion: Lower frequency of injections, storage flexibility and improved QoL drove satisfaction and preference for N8-GP over previous treatments among patients with HA.Plain language summary: Hemophilia A is a rare bleeding disorder that often runs in families. Although there is no cure, several therapeutic options are available to help control bleeding in people with hemophilia A. However, most treatments require intravenous (directly into a vein) or subcutaneous (directly under the skin) injections, which is a significant burden for all patients. In this study, adults and caregivers of adolescents, with hemophilia A answered a survey about their treatment experience with a medicine called N8-GP (turoctocog alfa pegol) compared with previous treatments. This is the first real-world evidence study focused on satisfaction with and preference for N8-GP compared with previous treatments. Survey results showed that adults and adolescents with hemophilia A were very satisfied with N8-GP compared with previous medicines. The main reasons for satisfaction included less frequent injections, the flexibility to store at room temperature, and improved quality-of-life.In addition, many patients expressed hop...

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Section: Challenges and Unmet Needs Of Patientsmentioning

confidence: 57%