A survey of physicians’ treatment switching practice in people on long-term prophylaxis for hemophilia in five European countries

Abstract

Objective

Recombinant factor VIII and factor IX Fc fusion proteins (rFVIIIFc and rFIXFc) were developed with an extended half-life (EHL) to improve the management of people with hemophilia A (PwHA) and B (PwHB), respectively.

Methods

This survey gathered physician-reported treatment decisions and physician views on outcomes in PwHA or PwHB who switched to rFVIIIFc or rFIXFc in the 12 months prior to study completion.

Results

Physicians (N = 37) considered bleeds, pharmacokinetic parameters, joint health and adherence the most important factors to assess both in routine care and when deciding to switch to an EHL therapy. In the 12 months prior to study completion, 37 physicians switched 113 PwHA to rFVIIIFc and 25 physicians switched 36 PwHB to rFIXFc. Most PwH (>90%) had moderate or severe hemophilia and many (>60%) switched within 6 months of the survey. The main reason for switching PwHA to rFVIIIFc was to allow fewer injections (49%), while the main reason for switching PwHB to rFIXFc was the product becoming available for use (36%). Overall, 96% of PwHA and 89% of PwHB who were switched remained on these EHL products at the time of survey. Mean total weekly dose, injection frequency and annualized bleeding rate were reported to have reduced following switching.

Conclusion

This survey provides valuable insight into reasons for, and challenges to, the use of EHL products in clinical practice. Physicians perceived that switching to treatment with rFVIIIFc or rFIXFc can improve quality of life, treatment burden, disease control and adherence.

Keywords:

• Hemophilia
• prophylaxis
• recombinant Fc fusion protein
• switch

Transparency

Declaration of funding

The project was funded by Swedish Orphan Biovitrum AB (Sobi) and implemented by two independent customer insight companies who ran the market research study (boobook [https://www.boobook.world/] supported by Big Fish International [http://bigfish-international.com/]) who helped with questionnaire design and data interpretation.

Declaration of financial/other relationships

M. van der Sluijs is a shareholder in, and employee of, Swedish Orphan Biovitrum AB. S. Tawil is an employee of Swedish Orphan Biovitrum AB. N. Huyghe and C. Wood are employees of boobook and Big Fish International, respectively. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship. M. van der Sluijs and S. Tawil designed the study. N. Huyghe ran the survey and collected and analyzed the data. C. Wood contributed to questionnaire design and data analysis. All authors interpreted the data, contributed to drafting and revising the article, provided their final approval of all content and agree to be accountable for all aspects of the work.

Acknowledgements

Medical writing and editorial support, funded by Sobi, was provided by Andy Lockley and Jilly Hope of Bioscript Medical. Data in this manuscript have previously been presented as posters at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH), Melbourne, Australia, 6–10 July 2019.

Data availability statement

The datasets generated and/or analyzed during the current study are not publicly available due to their proprietary nature but are available from the corresponding author on reasonable request.

Ethics approval

Formal ethical approval was not required for this market research study as this was an opt-in survey, which was considered to be low risk. This market research followed the laws and guidelines related to the protection of personal data in each country included in the study (including BHBIA, MRS, EphMRA and ESOMAR). None of the findings from the research could be related back to the individual physician or their institution. All physicians provided informed consent and had the right to withdraw from the interview at any time.