Exploring the Impact of Infusion Frequency in Hemophilia A: Exit Interviews with Patients Participating in BAY 94-9027 Extension Studies (PROTECT VIII)

Abstract

IntroductionProphylactic treatment of severe hemophilia A is burdensome, requiring frequent intravenous injections. Extended half-life (EHL) factor VIII replacement therapies offer longer intervals between infusions while still meeting efficacy and safety outcomes; however, patient perspectives following long-term use of such products in the real-world remain unknown.ObjectiveWe aimed to explore the importance of infusion frequency and the potential benefits of reduced infusion frequency among patients receiving prophylactic treatment with an EHL product (BAY 94-9027).MethodsPatients with severe hemophilia A participating in the PROTECT VIII extension study were invited to participate in a semi-structured, concept elicitation ‘exit’ interview to discuss their experiences. Participants were recruited from Israel, The Netherlands, and the US. Interview transcripts were translated into English and analyzed using thematic analysis methods.ResultsSixteen participants (29–68 years of age) infusing with BAY 94-9027 once every 7 days, once every 5 days, or twice weekly were interviewed. Participants reported infusion frequency (alongside efficacy) as the most important treatment attribute influencing their satisfaction with therapy. Patient-reported benefits of reduced infusion frequency and longer duration of factor coverage included greater ability to participate in physical activities; better vein health; less time to schedule and administer factor VIII; reduced impact on work; and improved emotional well-being.ConclusionsThis study provides rich insights into the experiences of patients with EHL products and the value of reduced infusion frequency. Such data could be of value to a range of stakeholders (e.g. regulators, payers) and facilitate patient–clinician discussions to promote tailored treatment decisions.

Real‐world prophylactic usage of recombinant factor VIII Fc in Sweden: A report from the Swedish national registry for bleeding disorders

“…3 Reducing the frequency of injections and extending the protection afforded by each injection will improve patients' quality of life, including improved ability to participate in physical activity and less time needed to administer treatment. 8 Furthermore, the need for fewer injections may improve patient adherence to treatment. 9 This study has both strengths and limitations.…”

Extended Half-life rFVIII for the Treatment of Hemophilia A: Drugs Consumption and Patients’ Perspective

“…EHL rFVIII products such as Jivi ® offer the potential for increased protection against bleeds while requiring less frequent infusions and IU consumption. A recent analysis aimed to evaluate patients reported outcomes regarding the benefit of the treatment with an EHL rFVIII therapy with Jivi ® [24,25].…”

Comparing Physician and Patient Perspectives on Prophylactic Treatment with BAY 94-9027 for Severe Haemophilia A: A Post Hoc Analysis

“…More details around the recruitment method and the conduct of the interviews with the patients has been published elsewhere [13].…”

“…In addition, physicians and study participants from the investigator sites in Israel, the Netherlands and the USA were invited to participate in qualitative exploratory interviews when exiting the extension part of the study. The findings from the patient interviews were recently published and showed that participants reported that BAY 94-9027 received during the PROTECT VIII study resulted in their increased ability to participate in physical activities, better 'vein health', less time needed to schedule and administer factor VIII, reduced impact on work and improved emotional well-being [13].…”