PB0664 - Treatment adherence in young people with hemophilia: perceptions of UK healthcare professionals

Background: UK guidelines for prophylactic treatment in hemophilia were first developed in 2010 and were subsequently updated in 2020. The primary goal of prophylaxis in hemophilia is to prevent bleeding and, in children, to reach maturity with normal joints.

Aims: To provide a current, real-world understanding of UK healthcare professional (HCP) perceptions of prophylactic treatment adherence in hemophilia patients transitioning from adolescence to young adulthood.

Methods: Over twelve months, ten virtual interviews were conducted with UK-based consultant hematologists, hemophilia specialist nurses, and hemophilia physiotherapists to explore their perceptions of prophylactic treatment adherence among their patient cohort.

Results: The interviews found that HCPs believe increased support is required to maintain treatment adherence in this population. An established relationship between patients and their hemophilia center, plus a well-planned transition to adult services using a multidisciplinary approach, involving doctors, nurses, physiotherapists and psychologists, was considered essential for maintaining treatment adherence and a trusted patient-provider relationship.
It was noted that patients with reduced disease awareness and lack of support face additional challenges. Interviewed HCPs recommended patients should have sufficient disease knowledge and an understanding of the consequences of missed doses from a young age. Among families with generational hemophilia, treatment adherence was perceived as less of an issue versus those without.
It was recognized that this population has unique needs and balancing the requirement for independence with the necessary surveillance can be difficult. Maintaining a normalized lifestyle is crucial and treatment should remain flexible within a defined regimen. Strategies for achieving individualized treatment goals should be developed alongside patients and their families, which may require adaptation for different life phases to support lifestyle needs.

Conclusion(s): Good treatment adherence in young people with hemophilia requires multidisciplinary support and parental collaboration from an early age, along with an understanding that this group requires unique and individualized care.