PB0679 - Clinical Characteristics and Disease Outcomes in Previously Treated Patients with Haemophilia: Results from a Real-World Study in Europe and United States

Background: Previously treated patients with haemophilia who later stopped treatment are under-represented in existing real-world literature.

Aims: To describe clinical characteristics and disease outcomes of previously treated patients with haemophilia.

Methods: Data were drawn from the Adelphi Haemophilia Disease Specific Programme™, a real-world, point-in-time survey conducted in France, Germany, Italy, Spain, United Kingdom, and United States, February 2020 - May 2021. Haematologists provided demographics, clinical characteristics, treatment history and disease outcomes data for up to 20 consecutively consulting male patients with haemophilia A or B. Descriptive analyses were conducted on previously treated patients with haemophilia who were untreated for ≥365 days prior to and at data collection.

Results: Haematologists (n=39) provided data for 69 previously treated patients with haemophilia, of which 61% had Haemophilia A. Mean [standard deviation, SD] age was 30.5 [16.6] years. Baseline clotting factor activity was 1-5% in 46% of patients and < 1% in 3%. Haematologist-reported reasons for discontinuation of previous treatment included efficacy concerns (52% of patients), safety/side effects (35%), and administration/dosing difficulties (34%).
Overall, 36% of patients experienced ≥1 bleeds in the 12 months prior to data collection with a mean [SD] of 1.7 [0.8] bleeds, 0.6 [0.8] of which occurred in joints. Most common bleed types were joint (40%), nose (40%), and small cuts/grazes (24%). Physicians reported joint problems in 17% of patients, use of a mobility aid in 12% and anxiety in 10% due to haemophilia. Table 1 shows Haemophilia A and B delineations.

Conclusion(s): Untreated haemophilia leaves patients at risk of uncontrolled bleeding episodes and progressive joint damage. With previously treated patients with haemophilia experiencing multiple bleeds and/or joint damage, effective, safe, and easy to administer treatments continue to be an unmet need. Further research is necessary to determine the impact of prolonged untreated periods on long-term haemophilia outcomes.