Abstract
Summary
This is a summary of the results from a study that looked at the treatment preferences of people with hemophilia and caregivers of children with hemophilia in the US and UK. The study was published in a research journal called Haemophilia.
People with hemophilia need regular treatments to help their blood clot properly and to prevent bleeding. Treatments to prevent bleeding are often injected into a vein. For some people, the injections can be painful or hard to use. If treatments are hard to use, people may be less likely to stick to their treatment plan. This can make it harder for them to manage their hemophilia. New treatments that work in a different way can be given as an injection under the skin instead of into a vein. These treatments may be easier for people to use. Newer treatments have different benefits and risks compared with earlier types of treatment, so researchers wanted to understand what features of treatments are most important to people with hemophilia and caregivers
Adults with hemophilia and caregivers of children with hemophilia in the US and UK took an online survey. They were asked to choose between made-up (hypothetical) treatments with different benefits, risks, and ways of administration (how the treatment is delivered into the body). The study showed that people with hemophilia and caregivers had mostly the same preferences for treatments to prevent bleeds. People cared most about having a treatment they can take less often. People were willing to accept some additional risks or lower treatment benefits if they could have injections under the skin instead of into a vein.
People with hemophilia and caregivers found some treatment features more important than others. It is important for healthcare teams to know which treatment features are most important to people. If treatments match people’s preferences, they are more likely to be satisfied. For example, if people prefer injections under the skin, they may be more likely to stick to their treatment plan if they use a treatment delivered under the skin instead of into a vein. People who stick to their treatment plan are likely to have better health outcomes.
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Acknowledgements
Pfizer thanks all the people who took part in the study, their caregivers and supporters, and all study staff. Medical writing support was provided by Courtney Cameron, PhD, Engage Scientific Solutions, and was funded by Pfizer Inc.
Ethics approval and consent to participate
The protocol was approved by an independent review board, Salus IRB, Austin, TX, USA (Salus IRB Study number: 23144). The study complied with the Declaration of Helsinki and applicable US regulatory requirements.
Author contributions
Conceptualization: H. Lu, T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, J. C. Cappelleri, S. A. Christopher, B. Hauber, N. Dunn, P. Chowdary, and J. A. Whitty. Methodology: H. Lu, T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, J. C. Cappelleri, S. A. Christopher, B. Hauber, and J. A. Whitty. Investigation: H. Lu, T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, and J. A. Whitty. Writing – original draft preparation: H. Lu, T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, J. C. Cappelleri, S. A. Christopher, B. Hauber, and J. A. Whitty. Writing – review and editing: H. Lu, T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, J. C. Cappelleri, S. A. Christopher, Á. A. Gutiérrez-Vargas, B. Hauber, N. Dunn, P. Chowdary, and J. A. Whitty. Visualization: H. Lu and Á. A. Gutiérrez-Vargas. Supervision: T. Gould, C. Colavecchia, J. Coulter, L. J. Wilcox, B. Hauber, and J. A. Whitty. Validation: H. Lu and Á. A. Gutiérrez-Vargas. Project administration: H. Lu and C. Colavecchia.
Funding
The study was funded by Pfizer Inc.
Competing interests
H. Lu and Á. A. Gutiérrez-Vargas: Employed by PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, which received funding from Pfizer to conduct this study. J. A. Whitty: Employed by PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, which received funding from Pfizer to conduct this study, and also is a minority shareholder in Thermo Fisher Scientific. N. Dunn: Shareholder in and consultant for Pfizer. P. Chowdary: Grant/research support from Bayer, CSL Behring, Freeline, Novo Nordisk, Pfizer, SOBI, Takeda; consultant for: Apcintex, Bayer, Boehringer Ingelheim, CSL Behring, Chugai, Freeline, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, SOBI, Takeda (advisory boards). C. Colavecchia, T. Gould, J. Coulter, L. J. Wilcox, J. C. Cappelleri, S. A. Christopher, and B. Hauber: Current employees and equity holders in Pfizer.
Availability of data and material
Data are available on request due to privacy and ethical restrictions.