Hypothesis rationale |
Physical functioning expected to improve due to stabilised factor levels and fewer joint bleedsRole functioning expected to disappear or become irrelevant in gene therapy due to nature of the therapy |
Worries expected to decrease and HRQoL to increase in the long term as factor levels expected to improve with gene therapy |
Consequences of bleeding will decrease (and therefore domain score will improve) with treatment advancement as number of bleeds are likely to decrease |
The emotional impact of treatment outcomes may either positively impact a patient (e.g., new sense of freedom, ability to partake in rigorous activities) or negatively impact a patient (e.g., loss of identity, reflection of time lost) |
Items within domain were considered dated and not fit for purpose in the context of novel treatment regimens, such as gene therapy |
Comments from expert working group |
Patients will not need to infuse prior to physical activity |
Worry may be engrained and long-lasting, therefore perhaps unlikely to change rapidly in the short termTypes of worries a PWH may have will be largely impacted by their demographics, such as their age, parental status, type of work or school stage |
Although bleeding consequences reduce with treatment advancement, PWH will remain aware/conscious of needing to be compliant with treatment in order to reduce consequencesAbility to clot and heal in gene therapy more than any other commonly used existing treatment |
There could be a deterioration in emotional wellbeing in the short term on gene therapy, but an improvement in the long term |
Advanced or novel treatments can be more costly and are not necessarily covered by insurance or national health service, which could be a potential treatment concernAssumption with gene therapy is patient will only need treatment oncePotential selection bias may occur where patients with fewer treatment concerns are more likely to enrol in clinical studiesSome uncertainties tend to exist around novel regimens, resulting in potential treatment concernsKnown complications or adverse events for commonly used existing therapies may result in treatment concerns |