https://orcid.org/0000-0002-1372-0619
Abstract
Purpose
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 hopes for the future while taking N8-GP, such as confidence in ability to undertake physical activities and ability to plan and go on a holiday. Compared with previous treatments, patients were feeling optimistic and hopeful about N8-GP and expressed that they have started to think less about the disease.
Despite the unique advantages of taking N8-GP, patients overall still perceive their quality-of-life to be less than an average person’s, presenting an opportunity for future advancements. Overall, this study sheds light on the unique experiences of people with hemophilia A taking N8-GP and further opens up the scope for addressing the unmet needs.
Abbreviations
HA, Hemophilia A; QoL, quality of life; HRQoL, health-related quality of life; ABR, annualized bleeding rates; FVIII, factor VIII; SHL, standard half-life; RWE, real-world evidence; EHL, extended half-life; EQ-5D-5L, EuroQol-5 Dimension; EQ-VAS, EuroQol-visual analogue scales; HERO, The Hemophilia Experiences, Results and Opportunities; CHESS, Cost of Hemophilia in Europe: a Socioeconomic.
Acknowledgments
The authors would like to thank the patients and caregivers who completed the survey. This study was funded by Novo Nordisk Health Care and medical writing support was provided by Dr. Ambika Shivakant Kurbet, PhD of Novo Nordisk India Pvt Limited, Global Medical Affairs, Bengaluru, India. We also thank Ashfield MedComms GmbH (Mannheim, Germany, an Inizio company) for editorial support, which was funded by Novo Nordisk. We thank Masha Eletskaya and Florence MacIver Bulbrook from Lumanity for data collection and analysis.
Author Contributions
All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.
Disclosure
AN has received investigator-initiated grant funding from Takeda (Shire), Chugai and Bayer; and has received honoraria from Sanofi, Takeda, Chugai, Bayer, Fujimoto, KMB, Pfizer, JB, Novo Nordisk, Sekisui Medical and CSL. AS does not have any obligations towards any of the companies involved in the study or the development of the medication. HE has received research support, honoraria, or consultation fees from Bayer Vital, BioMarin, Biotest, CSL Behring, Novo Nordisk, Pfizer, Roche, and Sobi. JW is an employee of Novo Nordisk. The authors report no other conflicts of interest in this work.