The advent of gene therapy may dramatically change the treatment paradigm for hemophilia A. But, even if patients are interested and eligible, navigating conversations around this new treatment can be challenging. Fortunately, there are resources available to help jump-start these discussions.
The addition of gene therapy as a treatment option for hemophilia A has made disease management decisions more complex.[1] Along with its potential to positively impact clinical outcomes and quality of life comes uncertainty surrounding long-term durability and safety, requiring it to be carefully considered among the many other treatments available.[1,2]
Since gene therapy is a recent addition to the therapeutic options for severe hemophilia A, you may feel unprepared to discuss it at length with patients.[3] However, the fact that it’s a one-time only, irreversible treatment requiring a long-term monitoring commitment, means it’s essential that you know how to talk about its potential benefits, risks, and unknowns.[1]
The lack of real-world data on this new therapeutic approach makes it challenging to address patients’ concerns. Gene therapy also differs from current prophylactic treatments in its eligibility, administration, safety risks, and post-infusion requirements. Therefore, any decisions made about its use will depend highly on each person’s disease experience. It necessitates a collaborative decision-making approach where clinical options are discussed in light of patient preferences and goals.
With a lot of information to cover and annual comprehensive visits already so busy, talking to eligible patients about gene therapy can seem unattainable. But there is a way to be both thorough and efficient when having conversations about gene therapy for hemophilia A.
A team of researchers at the Hemophilia and Thrombosis Treatment Center at Rady Children's Hospital San Diego have developed a tool to prepare clinicians for gene therapy discussions.[4] The content of the tool was informed by feedback from surveys and interviews with people with hemophilia A.[5,6] Then, ten clinicians with 1-27 years’ experience in hemophilia care were interviewed to refine the tool, which is now available as a downloadable document alongside the interview results.[4]
With key information consolidated, the tool conveniently guides clinicians through interactive and systematic treatment discussions with eligible patients. It recommends a way to explain gene therapy, summarizes the clinical evidence, and provides an overview of treatment characteristics relevant to common patient concerns (Table 1).
Having the tool handy makes it easy to provide patients with an evidence-backed idea of what they can expect if they undertake gene therapy, as well as discuss how it may affect their future care and daily life. Potential benefits, such as experiencing fewer bleeds and stopping prophylactic treatment, should be balanced with uncertainties like unpredictable individual response and unknown duration of treatment effect.[7,8] By being transparent about known and unknown safety risks, including the likelihood of needing immunosuppressive treatment, you can build trust with patients and ensure that they understand what their decision may entail.
The tool also helps you gauge a patient’s willingness to commit to gene therapy by outlining the required post-infusion lifestyle changes and monitoring tests. For example, the need to limit alcohol and attend regular check-ups after gene therapy may affect the timing of treatment, if pursued. Clarifying these obligations is vital for setting realistic expectations and assessing suitability alongside current and alternative treatment options.
To ensure completeness, the tool includes a checklist of all the key information you should go over with each patient to help them make an informed decision. Additional resources are also provided, should you or your patient need further guidance.
You will likely need multiple conversations to ensure patients feel fully informed. So, rather than waiting for an annual visit, it’s advisable to encourage scheduling gene therapy-specific discussions. Some of these discussions can be led by other healthcare team members like nurses and social workers, as patients value their input while making their decision.[6] Group discussions with a few patients together can save you time, and allows them to hear perspectives from others considering gene therapy too.[9]
Although the clinician tool will evolve with growing experience in gene therapy and the approval of new products, it’s a great starting point to help you engage people with hemophilia A in their treatment journey in a collaborative manner.
Implement shared decision-making for gene therapy in your practice using this evidence-based clinician tool.
References
Fletcher S, Jenner K, Khair K. Shared decision-making for gene therapy in haemophilia care. J Haemoph Pract. 2023;10(1):62-67. doi:10.2478/jhp-2023-0009
Woollard L, Gorman R, Rosenfelt DJ. Improving patient informed consent for haemophilia gene therapy: the case for change. Ther Adv Rare Dis. 2021;2. doi:10.1177/26330040211047244
Peyvandi F, Lillicrap D, Mahlangu J, et al. Hemophilia gene therapy knowledge and perceptions: results of an international survey. Res Pract Thromb Haemost. 2020;4(4):644-651. doi:10.1002/rth2.12326
Limjoco J, Thornburg CD. Development of a haemophilia A gene therapy shared decision-making tool for clinicians. Haemophilia. 2023;29(5):1184-1190. doi:10.1111/hae.14822
Limjoco J, Calatroni A, Aristizabal P, Thornburg CD. Gene therapy preferences and informed decision-making: results from a National Hemophilia Foundation Community Voices in Research survey. Haemophilia. 2023;29(1):51-60. doi:10.1111/hae.14706
Limjoco J, Thornburg CD. Gene therapy for hemophilia A: a mixed methods study of patient preferences and shared decision-making. Patient Prefer Adherence. 2023;17:1093-1105. doi:10.2147/PPA.S406894
Mahlangu J, Kaczmarek R, Von Drygalski A, et al. Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A. N Engl J Med. 2023;388(8):694-705. doi:10.1056/NEJMoa2211075
European Medicines Agency. ROCTAVIAN, INN-valoctocogene roxaparvovec. Summary of product characteristics. Last updated: December 6, 2023. Accessed April 28, 2024. https://www.ema.europa.eu/en/medicines/human/EPAR/roctavian
International Society on Thrombosis and Haemostasis. Shared decision-making: the journey with gene therapy in hemophilia (session 1). Streamed live: October 30, 2023. Accessed April 4, 2024. https://www.youtube.com/watch?v=jX4KD5eFGNY
World Federation of Hemophilia. World Federation of Hemophilia Shared Decision Making Tool. Last updated: August 1, 2023. Accessed April 28, 2024. https://sdm.wfh.org/