Haemophilia Treatment Preferences Survey

(HaemPref Survey)

How do individuals make decisions about treatment?
What factors encourage or discourage patients from treatments?
What are individuals’ treatment goals?

Results presented are based on a study conducted by Community and Patient Preference Research (CaPPRe) in partnership with Haemophilia Foundation Australia (HFA) in 2024.

What we did

The survey

We conducted a 30 minute online survey where we asked participants to indicate their treatment preferences through a Discrete Choice Experiment (DCE) and their treatment goals through a Best-Worst Scaling (BWS) exercise.

The DCE

Participants were shown several DCE scenarios where they were asked to choose their most preferred hypothetical (‘imagined’) treatment, labelled as:

• IV infusion at home
• Subcutaneous injection at home
• Gene therapy (IV infusion at hospital/clinic)
• Or the opt-out option: Stay on current treatment.

The treatments were described using seven key attributes that varied between each scenario:

1. Chance the treatment will work (shown for gene therapy only)
2. Frequency of administration
3. Number of bleeds per two years
4. Improvement in activity of daily life/physical activity
5. Risk of mild-to-moderate side effects
6. Risk of severe side effects
7. Lifetime risk of developing inhibitors or anti-drug antibodies

Who we spoke to

We surveyed 38 adults living with haemophilia and 34 parents/carers of children under the age of 18 years living with haemophilia (total sample 72). Most patients (both adult and child) had a diagnosis of haemophilia A and were classed as having ‘severe’ haemophilia when first diagnosed.

The BWS

Over another series of scenarios, participants were asked to choose the most and least important treatment outcomes out of a list of 11:

1. Less time in hospital
2. Having minor and major surgeries safely
3. Living longer
4. Having less pain and stiffness
5. Being independent
6. Participating in social activities
7. Participating in sport, exercise, and personal interests/hobbies
8. Participating at school and work
9. Travelling overseas
10. Feeling well emotionally
11. Having a ‘haemophilia-free mind’

What we found

Most important treatment features

• Overall, participants prioritised subcutaneous injections that were given less often and that posed a low risk of inhibitors/anti-drug antibodies.
• Improvements in daily activity, the risk of mild to moderate side effects, and the number of bleeds were also important, but these attributes were relatively less important in treatment decisions.

What we found

Gene therapy preferences

• Many participants expressed concerns about potential side effects of currently available gene therapies and the lack of long-term data regarding their risks and benefits.
• Gene therapy was not the most preferred treatment in the DCE overall, however many participants did choose it when it had a high chance of success, long lasting benefits, and low risk of side effects. This suggests that if the haemophilia community’s current concerns about gene therapy are addressed through ongoing long term research, gene therapy could become the more preferred treatment option.
• The most crucial factor that determined whether people would choose gene therapy in the DCE was its chance of working (e.g., “80% chance it will work for you/your child” vs. “50% chance it will work for you/your child”).

Priority treatment outcomes

• Participants prioritised independent and pain-free physical activity, social engagement, and maintaining physical and emotional wellbeing.
• Parents/carers placed more importance on their children’s emotional wellbeing than adult patients did on their own.

Conclusion

Results of this study indicate that while the Australian haemophilia community currently prefers subcutaneous injections that are given less often and have a low risk of inhibitors/anti-drug antibodies, its members will consider gene therapy as an alternative, provided concerns they have about its risks and benefits are addressed.

These findings underscore the need for ongoing long term research into gene therapy and effective health communication.

Shared decision making will also ensure treatment teams respond effectively to the individual preferences and goals of people living with haemophilia in Australia.

Results presented above are based on a study conducted by Community and Patient Preference Research (CaPPRe) in partnership with Haemophilia Foundation Australia (HFA) in 2024.