Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice...

The expansion of haemophilia treatment to include non-factor prophylaxis provides new options with various benefits, risks, administration modes, and device types, necessitating trade-offs in decision-making.

This study evaluated prophylactic treatment preferences of people with haemophilia and their caregivers, assessing their willingness to make trade-offs among treatment attributes.

A web-based survey containing a discrete-choice experiment (DCE) was completed by adults (≥ 18 years old) and caregivers of children (aged 8-17 years) with haemophilia in the United States and United Kingdom. Participants were asked to complete 10 choice tasks, choosing their preferred prophylaxis from two hypothetical profiles defined by seven attributes. The primary analysis employed a mixed logit model, and subgroup analyses explored preference heterogeneity.

Participants were 194 adults with haemophilia (US: 150; UK: 44) and 169 caregivers (US: 150; UK: 19). Avoiding daily treatment administration had the highest attribute importance among both adults and caregivers, followed by changes in annual bleeds. Participants were willing to accept an increased risk of serious side effects or developing inhibitors, or reduced efficacy in preventing annual bleeds, to switch from intravenous to subcutaneous administration. Refrigeration requirements and the need for a second treatment for breakthrough bleeds had lower importance.

Participants highly value avoiding daily administration and reducing bleeds, and they are willing to accept increased risks or reduced efficacy to have subcutaneous rather than intravenous administration. As new treatments become available, understanding these preferences can facilitate shared decision-making in treatment selection and enhance patient-centred drug development and patient communication.