Treatment and Disease Burden in a Cohort of People With Haemophilia Without Inhibitors in the United States.

introductionHaemophilia is a bleeding disorder caused by a deficiency in coagulation factors VIII or IX. Great advances in haemophilia treatment have been achieved in recent decades, with a range of products for prophylaxis now available, the ability to individualise therapy, and resultant improvements in life expectancy and quality of life. Despite these advances, there remains an unresolved burden, both from the disease and its treatment. Real-world data provide an opportunity to understand the experiences of people with haemophilia and gain insight into the areas of greatest unmet need.

aimTo assess the treatment and disease burden of haemophilia using real-world data.

methodsMedical records data and patient-reported outcomes from a self-selected cohort of 446 people with haemophilia without inhibitors across the United States were combined using an online research platform. Participants' treatment and disease burdens were assessed using the Hemophilia-Treatment Experience Measure (Hemo-TEM) and the patient-reported outcomes measurement information system (PROMIS)-29, respectively. Results were stratified by haemophilia type and disease severity.

resultsThese real-world data indicated that treatment and disease burden are experienced by people with haemophilia regardless of treatment regimen or severity of disease. The domains most affected by treatment burden were physical impact and emotional impact; the domains most affected by disease burden were physical functioning, pain interference, depression and anxiety.

conclusionThis underscores how the perspectives of people with haemophilia in real-world settings represent a valuable adjunct to clinical trial data. Such insights can potentially contribute to guide individualised treatment options and help address current unmet needs.